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Switch Over Study of Biosimilar AGA for Fabry Disease (SMILE)

Primary Purpose

Fabry Disease

Status
Recruiting
Phase
Phase 3
Locations
Argentina
Study Type
Interventional
Intervention
Recombinant human alpha galactosidase A (agalsidase beta)
Recombinant human alpha-galactosidase A (agalsidase beta)
Sponsored by
Bio Sidus SA
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fabry Disease

Eligibility Criteria

18 Years - 60 Years (Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Sex and Age Male or female participant with ≥18 and ≤60 years of age at the time of signing the informed consent form (ICF). Reproduction Female participants who are not pregnant, breastfeeding, donating eggs (ova, oocytes), or considering becoming pregnant during the study and for 3 months after the last dose of study treatment. All women of childbearing potential (WOCBP) must have a negative urine pregnancy test at the Screening visit and at Baseline visit (prior to the first dose of experimental intervention). WOCBP must use one highly effective form of birth control contraception through the study and for 3 months after the last dose of study treatment (refer to Appendix 1 in Section 10.1). Male participants who are not considering fathering a child during the study and for 3 months after the last dose of study treatment. Male sexually active participant with female partner(s) of childbearing potential must agree to use male condoms during the study and for 3 months after the last dose of study treatment or have documented successful surgical sterilization. Informed Consent Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. Type of Participant and Characteristics Confirmed previous diagnosis of FD. Women: preferably present genetic testing showing pathogenic GLA mutation consistent with FD at screening. Men: preferably present leukocyte α-Gal A activity below normal range and/ or pathogenic GLA mutation consistent with FD at screening. At least 50% of the participants will be male with classic FD phenotype. The remaining percentage will consist of male late onset and classic women FD phenotype. Participants who have been on stable Fabrazyme® treatment for at least 6 months prior to Baseline visit. Patients that in the last 3 months before the baseline visit have been receiving ≥80% of Fabrazyme®'s labeled dose/kg, this calculation includes both infusions provided by Biosidus during the Lead in period. Disease status considered clinically stabilized, at Investigators' discretion. Estimated glomerular filtration rate (eGFR) ≥45 mL/minute/1.73 m2 by CKD-EPI equation at Screening visit. If receiving pain killers, angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs), participants must be in a stable dose for ≥ 4 weeks. Exclusion Criteria: Medical Conditions Chronic kidney disease in stage 3b, 4, or 5. History of dialysis, kidney transplant or participants who are on the waiting list for a kidney transplant. Proteinuria ≥1 g/day at screening. Participants who have suffered a clinical cardiovascular event (such as but not limited to myocardial infarction, transient ischemic attack) within 6 months prior to Screening visit. Participants who have clinically significant unstable cardiac disease (such as but not limited to uncontrolled symptomatic arrhythmia, unstable angina, congestive heart failure New York Heart Association class III or IV). Participants who have suffered a clinical cerebrovascular event (such as but not limited to stroke, transient ischemic attack) within 6 months prior to Screening visit. History of anaphylaxis or other type I hypersensitivity reactions to agalsidase beta. History of acute kidney injury in the 12 months prior to Screening visit (such as but not limited to acute interstitial nephritis, acute renal failure of glomerular origin or caused by vasculitis). Presence of any medical, emotional, behavioral, or psychological condition that, according to the Investigator, would interfere with the participant's compliance with the requirements of the study. Prior/Concomitant Therapy Treatment initiation or change of dose of ACE inhibitors or ARBs in the 4 weeks before the screening. Prior/Concurrent Clinical Trial Experience Current participation in an interventional study, in which the participant received any drug within 90 days before the Screening visit.

Sites / Locations

  • Instituto de Nefrología Pergamino S.R.LRecruiting
  • Centro Médico Santa María de la SaludRecruiting
  • Instituto de Investigaciones Clínicas QuilmesRecruiting
  • Clínica Universitaria Reina FabiolaRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

AGA BETA BS

Arm Description

The study has a single-arm. First, all participants will receive 2 doses of Fabrazyme® with approximately 14 days between them, and afterwards all participants will switch treatment and receive AGA BETA BS for 54 weeks

Outcomes

Primary Outcome Measures

Equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment
• The primary objective of the study is to evaluate the equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment in participants with Fabry disease previously stabilized with Fabrazyme, by measuring disease biomarker. Endpoint: • Mean plasma Lyso-Gb3 marker ratio after 26 weeks of treatment, defined as plasma level of the marker Lyso-Gb3 after 26 weeks (6 months) divided by plasma level of the marker Lyso-Gb3 at baseline.

Secondary Outcome Measures

Difference in efficacy between AGA BETA BS and Fabrazyme® after 1 year of treatment
• To evaluate the difference in efficacy between AGA BETA BS and Fabrazyme® after 1 year of treatment in participants with Fabry disease previously stabilized with Fabrazyme®, by measuring disease biomarker. Endpoint: • Mean plasma Lyso-Gb3 marker ratio after 54 weeks of treatment, defined as plasma level of the marker Lyso-Gb3 after 54 weeks (12 months) divided by plasma level of the marker Lyso-Gb3 at baseline.
Compare the pain severity before and after 26 weeks of treatment with AGA BETA BS
• To compare the pain severity before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 26 of treatment.
Compare the pain severity before and after 54 weeks of treatment with AGA BETA BS
• To compare the pain severity before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 54 weeks of treatment.
Compare the impact of pain on daily functions before and after 26 weeks of treatment with AGA BETA
• To compare the impact of pain on daily functions before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain interference as assessed by BPI-short form pain interference items scores after 26 weeks of treatment
Compare the impact of pain on daily functions before and after 54 weeks of treatment with AGA BETA
• To compare the impact of pain on daily functions before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain interference as assessed by BPI-short form pain interference items scores after 54 weeks of treatment
Compare the participants' perception of their own health before and after 26 weeks of treatment with AGA BETA BS
• To compare the participants' perception of their own health before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the SF (Short Form)-36. Endpoint: • Change from baseline in SF-36 scores after 26 weeks of treatment.
Compare the participants' perception of their own health before and after 54 weeks of treatment with AGA BETA BS
• To compare the participants' perception of their own health before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the SF (Short Form)-36. Endpoint: • Change from baseline in SF-36 scores after 54 weeks of treatment.
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count at baseline.
Platelet count at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count after 14 weeks of treatment.
Platelet count at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count after 26 weeks of treatment.
Platelet count at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count after 54 weeks of treatment.
Platelet count at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count at baseline.
Red blood cell count at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count after 14 weeks of treatment
Red blood cell count at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count after 26 weeks of treatment
Red blood cell count at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count after 54 weeks of treatment
Red blood cell count at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin at baseline
Hemoglobin at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin after 14 weeks of treatment
Hemoglobin at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin after 26 weeks of treatment
Hemoglobin at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin after 54 weeks of treatment
Hemoglobin at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit
Hematocrit at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit
Hematocrit at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit
Hematocrit at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit
Hematocrit at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume
Mean corpuscular volume at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume
Mean corpuscular volume at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume
Mean corpuscular volume at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume
Mean corpuscular volume at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin
Mean corpuscular hemoglobin at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin
Mean corpuscular hemoglobin at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin
Mean corpuscular hemoglobin at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin
Mean corpuscular hemoglobin at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration
Mean cell hemoglobin concentration at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration
Mean cell hemoglobin concentration at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration
Mean cell hemoglobin concentration at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration
Mean cell hemoglobin concentration at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes
%reticulocytes at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes
%reticulocytes at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes
%reticulocytes at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes
%reticulocytes at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count
White blood cell count at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count
White blood cell count at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count
White blood cell count at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count
White blood cell count at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils
Neutrophils at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils
Neutrophils at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils
Neutrophils at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils
Neutrophils at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes
Lymphocytes at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes
Lymphocytes at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes
Lymphocytes at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes
Lymphocytes at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes
Monocytes at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes
Monocytes at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes
Monocytes at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes
Monocytes at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils
Eosinophils at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils
Eosinophils at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils
Eosinophils at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils
Eosinophils at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils
Basophils at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils
Basophils at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils
Basophils at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils
Basophils at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen
Blood urea nitrogen at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen
Blood urea nitrogen at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen
Blood urea nitrogen at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen
Blood urea nitrogen at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate
Phosphate at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate
Phosphate at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate
Phosphate at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate
Phosphate at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine
Creatinine at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine
Creatinine at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine
Creatinine at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine
Creatinine at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol
Total cholesterol at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol
Total cholesterol at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol
Total cholesterol at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol
Total cholesterol at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL
Cholesterol LDL at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL
Cholesterol LDL at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL
Cholesterol LDL at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL
Cholesterol LDL at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL
Cholesterol HDL at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL
Cholesterol HDL at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL
Cholesterol HDL at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL
Cholesterol HDL at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides
Triglycerides at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides
Triglycerides at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides
Triglycerides at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides
Triglycerides at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia
Glycemia at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia
Glycemia at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia
Glycemia at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia
Glycemia at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin
Total bilirrubin at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin
Total bilirrubin at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin
Total bilirrubin at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin
Total bilirrubin at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin
Direct bilirrubin at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin
Direct bilirrubin at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin
Direct bilirrubin at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin
Direct bilirrubin at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase
Aspartate aminotransferase at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase
Aspartate aminotransferase at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase
Aspartate aminotransferase at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase
Aspartate aminotransferase at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase
Alanine aminotransferase at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase
Alanine aminotransferase at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase
Alanine aminotransferase at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase
Alanine aminotransferase at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium
Sodium at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium
Sodium at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium
Sodium at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium
Sodium at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium
Potassium at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium
Potassium at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium
Potassium at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium
Potassium at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine
Chlorine at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine
Chlorine at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine
Chlorine at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine
Chlorine at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate
Bicarbonate at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate
Bicarbonate at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate
Bicarbonate at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate
Bicarbonate at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium
Magnesium at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium
Magnesium at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium
Magnesium at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium
Magnesium at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium
Calcium at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium
Calcium at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium
Calcium at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium
Calcium at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase
Alkaline phosphatase at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase
Alkaline phosphatase at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase
Alkaline phosphatase at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase
Alkaline phosphatase at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins
Total proteins at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins
Total proteins at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins
Total proteins at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins
Total proteins at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin
Albumin at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin
Albumin at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin
Albumin at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin
Albumin at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase
Gamma glutamyl transferase at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase
Gamma glutamyl transferase at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase
Gamma glutamyl transferase at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase
Gamma glutamyl transferase at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate
estimated Glomerular Filtration Rate at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate
estimated Glomerular Filtration Rate at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate
estimated Glomerular Filtration Rate at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate
estimated Glomerular Filtration Rate at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio
Urine albumin-creatinine ratio at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio
Urine albumin-creatinine ratio at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio
Urine albumin-creatinine ratio at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio
Urine albumin-creatinine ratio at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine
First morning urine at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine
First morning urine at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine
First morning urine at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine
First morning urine at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of electrocardiograms.
General evaluation of cardiac function based on the analysis of electrocardiogram exams performed at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of electrocardiograms.
General evaluation of cardiac function based on the analysis of electrocardiogram exam performed after 26 weeks of treatment.
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of electrocardiograms.
General evaluation of cardiac function based on the analysis of electrocardiogram exam performed after 54 weeks of treatment.
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of echocardiograms.
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exam performed at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of echocardiograms.
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed after 26 weeks of treatment.
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of echocardiograms.
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed after 54 weeks of treatment.
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples.
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples.
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples.
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples.
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the analysis of physical assessments, adverse events and infusion-related reactions.
Analysis of data obtained from clinical and physical assessments, and from reported adverse events and infusion-related reactions throughout the clinical trial. This outcome will be measured in terms of Presence/Absence of relevant clinical findings.

Full Information

First Posted
November 28, 2022
Last Updated
July 18, 2023
Sponsor
Bio Sidus SA
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1. Study Identification

Unique Protocol Identification Number
NCT05843916
Brief Title
Switch Over Study of Biosimilar AGA for Fabry Disease
Acronym
SMILE
Official Title
Phase III, Open-label, Switch Over Trial of the Efficacy and Safety of Agalsidase Beta Biosidus (AGA BETA BS) in Fabry Disease Patients Previously Stabilized With Fabrazyme®
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
December 13, 2022 (Actual)
Primary Completion Date
December 1, 2023 (Anticipated)
Study Completion Date
December 1, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bio Sidus SA

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No

5. Study Description

Brief Summary
BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and previously stabilized with Fabrazyme®.
Detailed Description
BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and previously stabilized with Fabrazyme®. The study will be conducted in 2 parts: a 5-week Lead-in period (period 1) and 54 week treatment period (period 2). During period 1 all participants will receive 2 intravenous (IV) infusions of Fabrazyme®, provided by Biosidus. After that, in period 2 all participants will switch treatment to AGA BETA BS. A total of up to 20 participants are planned for the study. •The primary objective of the study is to evaluate the equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment in participants with Fabry disease previously stabilized with Fabrazyme, by measuring disease biomarker (mean plasma Lyso-Gb3 marker ratio after 26 weeks of treatment, defined as plasma level of the marker Lyso-Gb3 after 26 weeks (6 months) divided by plasma level of the marker Lyso-Gb3 at baseline).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and previously stabilized with Fabrazyme®.
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
AGA BETA BS
Arm Type
Experimental
Arm Description
The study has a single-arm. First, all participants will receive 2 doses of Fabrazyme® with approximately 14 days between them, and afterwards all participants will switch treatment and receive AGA BETA BS for 54 weeks
Intervention Type
Drug
Intervention Name(s)
Recombinant human alpha galactosidase A (agalsidase beta)
Other Intervention Name(s)
Fabrazyme
Intervention Description
All participants will receive 2 doses of Fabrazyme® with approximately 14 days between them. The dose will be 1 mg/kg of body mass every 2 weeks
Intervention Type
Drug
Intervention Name(s)
Recombinant human alpha-galactosidase A (agalsidase beta)
Other Intervention Name(s)
AGA BETA BS
Intervention Description
All participants will receive AGA BETA BS for 54 weeks. The dose will be 1 mg/kg of body mass every 2 weeks
Primary Outcome Measure Information:
Title
Equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment
Description
• The primary objective of the study is to evaluate the equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment in participants with Fabry disease previously stabilized with Fabrazyme, by measuring disease biomarker. Endpoint: • Mean plasma Lyso-Gb3 marker ratio after 26 weeks of treatment, defined as plasma level of the marker Lyso-Gb3 after 26 weeks (6 months) divided by plasma level of the marker Lyso-Gb3 at baseline.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Difference in efficacy between AGA BETA BS and Fabrazyme® after 1 year of treatment
Description
• To evaluate the difference in efficacy between AGA BETA BS and Fabrazyme® after 1 year of treatment in participants with Fabry disease previously stabilized with Fabrazyme®, by measuring disease biomarker. Endpoint: • Mean plasma Lyso-Gb3 marker ratio after 54 weeks of treatment, defined as plasma level of the marker Lyso-Gb3 after 54 weeks (12 months) divided by plasma level of the marker Lyso-Gb3 at baseline.
Time Frame
1 year
Title
Compare the pain severity before and after 26 weeks of treatment with AGA BETA BS
Description
• To compare the pain severity before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 26 of treatment.
Time Frame
26 weeks
Title
Compare the pain severity before and after 54 weeks of treatment with AGA BETA BS
Description
• To compare the pain severity before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 54 weeks of treatment.
Time Frame
54 weeks
Title
Compare the impact of pain on daily functions before and after 26 weeks of treatment with AGA BETA
Description
• To compare the impact of pain on daily functions before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain interference as assessed by BPI-short form pain interference items scores after 26 weeks of treatment
Time Frame
26 weeks
Title
Compare the impact of pain on daily functions before and after 54 weeks of treatment with AGA BETA
Description
• To compare the impact of pain on daily functions before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain interference as assessed by BPI-short form pain interference items scores after 54 weeks of treatment
Time Frame
54 weeks
Title
Compare the participants' perception of their own health before and after 26 weeks of treatment with AGA BETA BS
Description
• To compare the participants' perception of their own health before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the SF (Short Form)-36. Endpoint: • Change from baseline in SF-36 scores after 26 weeks of treatment.
Time Frame
26 weeks
Title
Compare the participants' perception of their own health before and after 54 weeks of treatment with AGA BETA BS
Description
• To compare the participants' perception of their own health before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the SF (Short Form)-36. Endpoint: • Change from baseline in SF-36 scores after 54 weeks of treatment.
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count at baseline.
Description
Platelet count at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count after 14 weeks of treatment.
Description
Platelet count at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count after 26 weeks of treatment.
Description
Platelet count at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count after 54 weeks of treatment.
Description
Platelet count at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count at baseline.
Description
Red blood cell count at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count after 14 weeks of treatment
Description
Red blood cell count at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count after 26 weeks of treatment
Description
Red blood cell count at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count after 54 weeks of treatment
Description
Red blood cell count at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin at baseline
Description
Hemoglobin at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin after 14 weeks of treatment
Description
Hemoglobin at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin after 26 weeks of treatment
Description
Hemoglobin at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin after 54 weeks of treatment
Description
Hemoglobin at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit
Description
Hematocrit at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit
Description
Hematocrit at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit
Description
Hematocrit at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit
Description
Hematocrit at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume
Description
Mean corpuscular volume at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume
Description
Mean corpuscular volume at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume
Description
Mean corpuscular volume at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume
Description
Mean corpuscular volume at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin
Description
Mean corpuscular hemoglobin at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin
Description
Mean corpuscular hemoglobin at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin
Description
Mean corpuscular hemoglobin at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin
Description
Mean corpuscular hemoglobin at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration
Description
Mean cell hemoglobin concentration at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration
Description
Mean cell hemoglobin concentration at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration
Description
Mean cell hemoglobin concentration at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration
Description
Mean cell hemoglobin concentration at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes
Description
%reticulocytes at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes
Description
%reticulocytes at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes
Description
%reticulocytes at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes
Description
%reticulocytes at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count
Description
White blood cell count at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count
Description
White blood cell count at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count
Description
White blood cell count at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count
Description
White blood cell count at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils
Description
Neutrophils at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils
Description
Neutrophils at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils
Description
Neutrophils at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils
Description
Neutrophils at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes
Description
Lymphocytes at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes
Description
Lymphocytes at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes
Description
Lymphocytes at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes
Description
Lymphocytes at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes
Description
Monocytes at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes
Description
Monocytes at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes
Description
Monocytes at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes
Description
Monocytes at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils
Description
Eosinophils at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils
Description
Eosinophils at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils
Description
Eosinophils at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils
Description
Eosinophils at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils
Description
Basophils at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils
Description
Basophils at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils
Description
Basophils at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils
Description
Basophils at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen
Description
Blood urea nitrogen at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen
Description
Blood urea nitrogen at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen
Description
Blood urea nitrogen at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen
Description
Blood urea nitrogen at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate
Description
Phosphate at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate
Description
Phosphate at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate
Description
Phosphate at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate
Description
Phosphate at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine
Description
Creatinine at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine
Description
Creatinine at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine
Description
Creatinine at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine
Description
Creatinine at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol
Description
Total cholesterol at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol
Description
Total cholesterol at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol
Description
Total cholesterol at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol
Description
Total cholesterol at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL
Description
Cholesterol LDL at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL
Description
Cholesterol LDL at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL
Description
Cholesterol LDL at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL
Description
Cholesterol LDL at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL
Description
Cholesterol HDL at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL
Description
Cholesterol HDL at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL
Description
Cholesterol HDL at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL
Description
Cholesterol HDL at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides
Description
Triglycerides at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides
Description
Triglycerides at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides
Description
Triglycerides at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides
Description
Triglycerides at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia
Description
Glycemia at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia
Description
Glycemia at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia
Description
Glycemia at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia
Description
Glycemia at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin
Description
Total bilirrubin at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin
Description
Total bilirrubin at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin
Description
Total bilirrubin at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin
Description
Total bilirrubin at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin
Description
Direct bilirrubin at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin
Description
Direct bilirrubin at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin
Description
Direct bilirrubin at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin
Description
Direct bilirrubin at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase
Description
Aspartate aminotransferase at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase
Description
Aspartate aminotransferase at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase
Description
Aspartate aminotransferase at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase
Description
Aspartate aminotransferase at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase
Description
Alanine aminotransferase at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase
Description
Alanine aminotransferase at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase
Description
Alanine aminotransferase at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase
Description
Alanine aminotransferase at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium
Description
Sodium at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium
Description
Sodium at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium
Description
Sodium at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium
Description
Sodium at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium
Description
Potassium at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium
Description
Potassium at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium
Description
Potassium at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium
Description
Potassium at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine
Description
Chlorine at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine
Description
Chlorine at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine
Description
Chlorine at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine
Description
Chlorine at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate
Description
Bicarbonate at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate
Description
Bicarbonate at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate
Description
Bicarbonate at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate
Description
Bicarbonate at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium
Description
Magnesium at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium
Description
Magnesium at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium
Description
Magnesium at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium
Description
Magnesium at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium
Description
Calcium at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium
Description
Calcium at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium
Description
Calcium at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium
Description
Calcium at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase
Description
Alkaline phosphatase at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase
Description
Alkaline phosphatase at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase
Description
Alkaline phosphatase at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase
Description
Alkaline phosphatase at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins
Description
Total proteins at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins
Description
Total proteins at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins
Description
Total proteins at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins
Description
Total proteins at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin
Description
Albumin at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin
Description
Albumin at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin
Description
Albumin at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin
Description
Albumin at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase
Description
Gamma glutamyl transferase at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase
Description
Gamma glutamyl transferase at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase
Description
Gamma glutamyl transferase at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase
Description
Gamma glutamyl transferase at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate
Description
estimated Glomerular Filtration Rate at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate
Description
estimated Glomerular Filtration Rate at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate
Description
estimated Glomerular Filtration Rate at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate
Description
estimated Glomerular Filtration Rate at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio
Description
Urine albumin-creatinine ratio at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio
Description
Urine albumin-creatinine ratio at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio
Description
Urine albumin-creatinine ratio at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio
Description
Urine albumin-creatinine ratio at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine
Description
First morning urine at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine
Description
First morning urine at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine
Description
First morning urine at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine
Description
First morning urine at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of electrocardiograms.
Description
General evaluation of cardiac function based on the analysis of electrocardiogram exams performed at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of electrocardiograms.
Description
General evaluation of cardiac function based on the analysis of electrocardiogram exam performed after 26 weeks of treatment.
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of electrocardiograms.
Description
General evaluation of cardiac function based on the analysis of electrocardiogram exam performed after 54 weeks of treatment.
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of echocardiograms.
Description
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exam performed at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of echocardiograms.
Description
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed after 26 weeks of treatment.
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of echocardiograms.
Description
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed after 54 weeks of treatment.
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples.
Description
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at baseline
Time Frame
Baseline
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples.
Description
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 14 weeks
Time Frame
14 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples.
Description
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 26 weeks
Time Frame
26 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples.
Description
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 54 weeks
Time Frame
54 weeks
Title
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the analysis of physical assessments, adverse events and infusion-related reactions.
Description
Analysis of data obtained from clinical and physical assessments, and from reported adverse events and infusion-related reactions throughout the clinical trial. This outcome will be measured in terms of Presence/Absence of relevant clinical findings.
Time Frame
54 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Sex and Age Male or female participant with ≥18 and ≤60 years of age at the time of signing the informed consent form (ICF). Reproduction Female participants who are not pregnant, breastfeeding, donating eggs (ova, oocytes), or considering becoming pregnant during the study and for 3 months after the last dose of study treatment. All women of childbearing potential (WOCBP) must have a negative urine pregnancy test at the Screening visit and at Baseline visit (prior to the first dose of experimental intervention). WOCBP must use one highly effective form of birth control contraception through the study and for 3 months after the last dose of study treatment (refer to Appendix 1 in Section 10.1). Male participants who are not considering fathering a child during the study and for 3 months after the last dose of study treatment. Male sexually active participant with female partner(s) of childbearing potential must agree to use male condoms during the study and for 3 months after the last dose of study treatment or have documented successful surgical sterilization. Informed Consent Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. Type of Participant and Characteristics Confirmed previous diagnosis of FD. Women: preferably present genetic testing showing pathogenic GLA mutation consistent with FD at screening. Men: preferably present leukocyte α-Gal A activity below normal range and/ or pathogenic GLA mutation consistent with FD at screening. At least 50% of the participants will be male with classic FD phenotype. The remaining percentage will consist of male late onset and classic women FD phenotype. Participants who have been on stable Fabrazyme® treatment for at least 6 months prior to Baseline visit. Patients that in the last 3 months before the baseline visit have been receiving ≥80% of Fabrazyme®'s labeled dose/kg, this calculation includes both infusions provided by Biosidus during the Lead in period. Disease status considered clinically stabilized, at Investigators' discretion. Estimated glomerular filtration rate (eGFR) ≥45 mL/minute/1.73 m2 by CKD-EPI equation at Screening visit. If receiving pain killers, angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs), participants must be in a stable dose for ≥ 4 weeks. Exclusion Criteria: Medical Conditions Chronic kidney disease in stage 3b, 4, or 5. History of dialysis, kidney transplant or participants who are on the waiting list for a kidney transplant. Proteinuria ≥1 g/day at screening. Participants who have suffered a clinical cardiovascular event (such as but not limited to myocardial infarction, transient ischemic attack) within 6 months prior to Screening visit. Participants who have clinically significant unstable cardiac disease (such as but not limited to uncontrolled symptomatic arrhythmia, unstable angina, congestive heart failure New York Heart Association class III or IV). Participants who have suffered a clinical cerebrovascular event (such as but not limited to stroke, transient ischemic attack) within 6 months prior to Screening visit. History of anaphylaxis or other type I hypersensitivity reactions to agalsidase beta. History of acute kidney injury in the 12 months prior to Screening visit (such as but not limited to acute interstitial nephritis, acute renal failure of glomerular origin or caused by vasculitis). Presence of any medical, emotional, behavioral, or psychological condition that, according to the Investigator, would interfere with the participant's compliance with the requirements of the study. Prior/Concomitant Therapy Treatment initiation or change of dose of ACE inhibitors or ARBs in the 4 weeks before the screening. Prior/Concurrent Clinical Trial Experience Current participation in an interventional study, in which the participant received any drug within 90 days before the Screening visit.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Viridiana Berstein, MD
Phone
+5491159597061
Email
v.berstein@biosidus.com.ar
First Name & Middle Initial & Last Name or Official Title & Degree
Roberto A Diez, MD
Phone
+541149098000
Email
r.diez@biosidus.com.ar
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alberto A Fernández, MD
Organizational Affiliation
Instituto de Investigaciones Clínicas Quilmes
Official's Role
Principal Investigator
Facility Information:
Facility Name
Instituto de Nefrología Pergamino S.R.L
City
Pergamino
State/Province
Buenos Aires
ZIP/Postal Code
2700
Country
Argentina
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Norberto R Antongiovanni, MD
Phone
+54 2477-430597
Email
nantongiovanni1@gmail.com
First Name & Middle Initial & Last Name & Degree
Norberto R Antongiovanni, MD
Facility Name
Centro Médico Santa María de la Salud
City
San Isidro
State/Province
Buenos Aires
ZIP/Postal Code
1642
Country
Argentina
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Gustavo H Cabrera, MD
Phone
+54 114742-3598
Email
gustavo.h.cabrera@hotmail.com
First Name & Middle Initial & Last Name & Degree
Gustavo H Cabrera, MD
Facility Name
Instituto de Investigaciones Clínicas Quilmes
City
Buenos Aires
Country
Argentina
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alberto Fernandez
Phone
60634883
Email
afernan59@hotmail.com
Facility Name
Clínica Universitaria Reina Fabiola
City
Córdoba
ZIP/Postal Code
X5004
Country
Argentina
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Norberto B Guelbert, MD
Phone
(0351)4142121
Email
gguelbert@gmail.com
First Name & Middle Initial & Last Name & Degree
Norberto B Guelbert, MD

12. IPD Sharing Statement

Learn more about this trial

Switch Over Study of Biosimilar AGA for Fabry Disease

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