Back to resultsA Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease
Primary Purpose
Fabry Disease
Status
Recruiting
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
REPLAGAL
Sponsored by
About this trial
This is an interventional treatment trial for Fabry Disease
Eligibility Criteria
Inclusion Criteria:
- Participant and/or legally authorized representative must voluntarily sign an Institutional Review Board/Independent Ethics Committee approved written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the participant. For the participants less than (<) 18 years old, participants will give assent AND their parents/legally authorized representative should sign the ICF accordingly.
The participant has confirmed diagnosis of Fabry disease as determined by the investigator, according to medical record including:
- For male participant, Fabry disease is confirmed by a deficiency of α-galactosidase A (GLA) activity and a mutation in the GLA gene
- For female participant, Fabry disease is confirmed by a mutation in the GLA gene.
- The participant is 7 to 65 years of age, inclusive, at screening.
- Female participants of childbearing potential must have a negative pregnancy test at screening.
- Female participants of childbearing potential must agree to use a medically acceptable method of contraception at all times during the study and for at least 14 days after the final investigational product infusion.
- The participant is deemed, as determined by the investigator, to have adequate general health to undergo the specified protocol-related procedures and to have no safety or medical contraindications for participation.
- The participant has not received any treatment (approved or investigational) specific to Fabry disease, such as ERT, chaperone therapy, or substrate reduction therapy.
- The adult participant (greater than or equal to [>=] 18 years old) must have an estimated glomerular filtration rate (eGFR) of 45 to 120 milliliter per minute per 1.73 meter square (mL/min/1.73 m^2). Serum creatinine is tested and the eGFR is calculated by central laboratory using the Chronic Kidney Disease Epidemiology (CKD-EPI) equation.
Exclusion Criteria:
- In the opinion of the investigator, the participant's life expectancy is less than or equal to (<=) 5 years.
- The participant has undergone or is scheduled to undergo kidney transplantation or is currently on dialysis, or has any signs or symptoms of end stage renal disease.
- The participant has a urine protein/creatinine ratio of greater than (>) 500 milligram per gram (mg/g).
- The participant has a clinically relevant history of allergy or signs or symptoms of severe hypersensitivity, which in the investigator's judgment, will substantially increase the participant's risk if he or she participates in the study.
- In the opinion of the investigator, the participant has non-Fabry disease-related cause of end organ (renal, cardiovascular, central nervous system) dysfunction/failure or is receiving medications that may affect the rate of disease progression, as assessed by renal measures.
- The participant has a positive test result at screening for hepatitis B surface antigen with detectable hepatitis B viral deoxyribonucleic acid (DNA) load, hepatitis C virus (HCV) antibody with confirmation by HCV ribonucleic acid polymerase chain reaction testing, or human immunodeficiency virus antibody.
The participant has received prior treatment with any of the following medications, with the exception of non-systemic use:
- Chloroquine
- Amiodarone
- Monobenzone
- Gentamicin
- The participant is pregnant or lactating.
- The participant has a body mass index >35 kilogram per square meter (kg/m^2).
- The participant is treated or has been treated with any investigational drug for indication other than Fabry disease within 30 days of study start.
- The participant and/or the participant's parent(s) or legal guardians are unable to understand the nature, scope, and possible consequences of the study.
- The participant is unable to comply with the protocol, example, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for evaluations, or is otherwise unlikely to complete the study, as determined by the investigator.
Sites / Locations
- Peking Union Medical College Hospital
- Xiangya Hospital, Central South UniversityRecruiting
- West China Hospital, Sichuan UniversityRecruiting
- The Children's Hospital of Zhejiang University School of MedicineRecruiting
- Shandong Provincial HospitalRecruiting
- Ruijin Hospital, Shanghai Jiaotong Uni. School of Med.Recruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
REPLAGAL
Arm Description
Participants will receive REPLAGAL 0.2 milligram per kilogram (mg/kg) body weight, infusion, intravenously every other week (EOW) for 52 weeks.
Outcomes
Primary Outcome Measures
Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. Serious AE is any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality/birth defect, and is an important medical event. A TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with an investigational product or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the investigational product or medicinal product until the end of the safety follow-up period.
Secondary Outcome Measures
Number of Participants With TEAEs
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. A TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with an investigational product or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the investigational product or medicinal product until the end of the safety follow-up period. Number of participants with TEAEs will be reported.
Number of Participants With Infusion-related Reactions (IRRs)
An IRR will be defined as an event that: occurs within 12 hours after the start of the infusion, begins either during or after the infusion, and is judged as possibly or probably related to treatment with the investigational product. An IRR can be serious or non-serious. Other AEs which occur prior to the infusion, along with AEs associated with protocol-defined testing and assessments (example, laboratory testing and physical examinations), which were performed prior to the infusion, will not be considered as IRRs. Number of participants with IRRs will be reported.
Number of Participants With Positive Anti-drug Antibody (ADA) to REPLAGAL
Number of participants with positive ADA to REPLAGAL will be reported.
Number of Participants With Positive Neutralizing Antibody (NAb) to REPLAGAL
Number of participants with positive NAb to REPLAGAL will be reported.
Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters
Laboratory assessment will include serum chemistry, hematology, and urinalysis. Number of participants with clinically significant abnormalities in laboratory parameters will be reported.
Number of Participants With Clinically Significant Abnormalities in Vital Signs
Vital sign assessment will include pulse, blood pressure, respiratory rate, and temperature. Number of participants with clinically significant abnormalities in vital signs will be reported.
Number of Participants With Clinically Significant Abnormalities in 12-lead Electrocardiogram (ECG)
ECG parameters will include PR, QRS, QT, QTc intervals, and heart rate. Number of participants with clinically significant abnormalities in 12-lead ECG will be reported.
Change From Baseline in Renal Function at Week 52
Renal function is assessed by estimated glomerular filtration rate (eGFR) using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula for greater than or equal to (>=) 18 years participants, eGFR = 141 x min (Serum Creatinine [Scr]/κ,1)^(α) x max(Scr/κ,1)^(-1.209) x 0.993^(Age) x 1.018 (if female) x 1.159 (if black) where: Scr is serum creatinine (milligram per deciliter [mg/dL]); κ is 0.7 for females and 0.9 for males; α is -0.329 for females and -0.411 for males; min indicates the minimum of Scr/κ or 1; max indicates the maximum of Scr /κ or 1. For less than (<) 18 years participants, Counahan-Barratt equation will be used for calculation of eGFR. eGFR = (0.43 × height in centimeter [cm])/Scr where, Scr is serum creatinine (mg/dL). Change from baseline in renal function at Week 52 will be reported.
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) Values at Weeks 8, 16, 28 and 40
The eGFR will be calculated by CKD-EPI formula for >=18 years participants. eGFR = 141 x min (Serum Creatinine [Scr]/κ,1)^(α) x max(Scr/κ,1)^(-1.209) x 0.993^(Age) x 1.018 (if female) x 1.159 (if black) where: Scr is serum creatinine (mg/dL); κ is 0.7 for females and 0.9 for males; α is -0.329 for females and -0.411 for males; min indicates the minimum of Scr/κ or 1; max indicates the maximum of Scr /κ or 1. For <18 years participants, Counahan-Barratt equation will be used for calculation of eGFR. eGFR = (0.43 × height in cm)/Scr where, Scr is serum creatinine (mg/dL). Change from baseline in eGFR values at Weeks 8, 16, 28 and 40 will be reported.
Change From Baseline in Left Ventricular Mass Index (LVMI)
Change from baseline in LVMI will be measured by echocardiography at Weeks 16 and 52.
Change From Baseline in Left Ventricular Ejection Fraction (LVEF)
Change from baseline in LVEF will be measured by echocardiography at Weeks 16 and 52.
Change From Baseline in Urine Protein/Creatinine Ratio at Weeks 8, 16, 28, 40 and 52
Change from baseline in urine protein/creatinine ratio at Weeks 8, 16, 28, 40 and 52 will be reported.
Change From Baseline in Pain as Assessed by Brief Pain Inventory Short Form (BPI-short Form) at Weeks 8, 16, 28, 40 and 52
BPI-short form is a validated self-report measure to assess the severity of pain and the impact of pain on daily functions. BPI-short form has 4 questions that assess pain intensity (worst, least, average, right now) on 10-point rating scales (0=No pain to 10=Pain as bad as you can imagine) and 7 questions that assess impact of pain on daily functions (general activity, mood, walking ability, normal work, relations with other people, sleep, enjoyment of life) on 10-point rating scales as (0=Does not interfere to 10=Completely interferes). Total score is reported as sum of individual questions score ranging from 0 to 10 with higher numbers indicating worse outcomes. Change from baseline in pain as assessed by BPI-short form at Weeks 8, 16, 28, 40 and 52 will be reported.
Change From Baseline in Plasma Globotriaosylsphingosine (Lyso-Gb3) Level at Weeks 8, 16, 28, 40 and 52
Change from baseline in plasma Lyso-Gb3 levels at Weeks 8, 16, 28, 40 and 52 will be reported.
Change From Baseline in Audiology Testing Values
Audiology testing will include pure tone conduction and bone conduction for each ear using 4 different pure tone frequencies (500 hertz [Hz], 1000 Hz, 2000 Hz, and 4000 Hz). Any changes in threshold will be categorized as conductive, sensorineural, or unknown. As planned, only participants <18 years of age will assessed for this outcome measure. Change from baseline in audiology testing values will be reported.
Area Under Serum Concentration-time Curve From the Time of Dosing to the Last Measurable concentration (AUC0-last) of REPLAGAL
AUC0-last will be reported.
Area Under Serum Concentration-time Curve From Time Zero Extrapolated to Infinity (AUC0-inf) of REPLAGAL
AUC0-inf will be reported.
Serum Clearance of Administered Dose (CL) of REPLAGAL
Clearance is defined as a quantitative measure of the rate at which a drug substance is removed from the body. CL will be reported.
Serum Clearance of Administered Dose Normalized Based on Body Weight of REPLAGAL
Clearance is defined as a quantitative measure of the rate at which a drug substance is removed from the body. CL will be reported.
Maximum Observed Serum Concentration (Cmax) of REPLAGAL
Cmax will be reported.
Terminal Elimination Half-life (T1/2) of REPLAGAL
T1/2 is defined as the natural log of 2 divided by the terminal rate constant (ƛz). T1/2 will be reported.
Time to Reach Maximum Observed Serum Concentration (Tmax) of REPLAGAL
Tmax will be reported.
Volume of Distribution at Steady State (Vss) of REPLAGAL
Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired serum concentration of a drug. Vss will be reported.
Volume of Distribution at Steady State Normalized Based on Body Weight of REPLAGAL
Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired serum concentration of a drug. Vss will be reported.
Dose Normalized Area Under Serum Concentration-time Curve From Time Zero to the Last Sampling Time (AUClast/Dose) of REPLAGAL
AUClast/Dose will be reported.
Dose Normalized Area Under the Serum Concentration-time Curve From Time Zero Extrapolated to Infinity (AUC0-inf/Dose) of REPLAGAL
AUC0-inf/Dose will be reported.
Dose Normalized Maximum Observed Serum Concentration (Cmax/Dose) of REPLAGAL
Cmax/Dose will be reported.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04974749
Brief Title
A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease
Official Title
An Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of REPLAGAL® in Treatment-naïve Chinese Subjects With Fabry Disease
Study Type
Interventional
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
Recruiting
Study Start Date
May 6, 2022 (Actual)
Primary Completion Date
May 28, 2024 (Anticipated)
Study Completion Date
May 28, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Takeda
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The main aim of the study is to assess the safety of REPLAGAL. Study participants will receive REPLAGAL as an intravenous infusion every other week for 52 weeks. Participants will visit their study clinic many times throughout the study.
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
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
REPLAGAL
Arm Type
Experimental
Arm Description
Participants will receive REPLAGAL 0.2 milligram per kilogram (mg/kg) body weight, infusion, intravenously every other week (EOW) for 52 weeks.
Intervention Type
Biological
Intervention Name(s)
REPLAGAL
Other Intervention Name(s)
Agalsidase Alfa, TAK-675
Intervention Description
Participants will receive REPLAGAL infusion intravenously.
Primary Outcome Measure Information:
Title
Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs)
Description
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. Serious AE is any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality/birth defect, and is an important medical event. A TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with an investigational product or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the investigational product or medicinal product until the end of the safety follow-up period.
Time Frame
From start of study drug administration up to 14 days after end of treatment (EOT) period (up to Week 54)
Secondary Outcome Measure Information:
Title
Number of Participants With TEAEs
Description
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. A TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with an investigational product or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the investigational product or medicinal product until the end of the safety follow-up period. Number of participants with TEAEs will be reported.
Time Frame
From start of study drug administration up to 14 days after EOT period (up to Week 54)
Title
Number of Participants With Infusion-related Reactions (IRRs)
Description
An IRR will be defined as an event that: occurs within 12 hours after the start of the infusion, begins either during or after the infusion, and is judged as possibly or probably related to treatment with the investigational product. An IRR can be serious or non-serious. Other AEs which occur prior to the infusion, along with AEs associated with protocol-defined testing and assessments (example, laboratory testing and physical examinations), which were performed prior to the infusion, will not be considered as IRRs. Number of participants with IRRs will be reported.
Time Frame
From start of study drug administration up to 14 days after EOT period (up to Week 54)
Title
Number of Participants With Positive Anti-drug Antibody (ADA) to REPLAGAL
Description
Number of participants with positive ADA to REPLAGAL will be reported.
Time Frame
Baseline up to Week 52
Title
Number of Participants With Positive Neutralizing Antibody (NAb) to REPLAGAL
Description
Number of participants with positive NAb to REPLAGAL will be reported.
Time Frame
Baseline up to Week 52
Title
Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters
Description
Laboratory assessment will include serum chemistry, hematology, and urinalysis. Number of participants with clinically significant abnormalities in laboratory parameters will be reported.
Time Frame
Baseline up to Week 52
Title
Number of Participants With Clinically Significant Abnormalities in Vital Signs
Description
Vital sign assessment will include pulse, blood pressure, respiratory rate, and temperature. Number of participants with clinically significant abnormalities in vital signs will be reported.
Time Frame
Baseline up to Week 52
Title
Number of Participants With Clinically Significant Abnormalities in 12-lead Electrocardiogram (ECG)
Description
ECG parameters will include PR, QRS, QT, QTc intervals, and heart rate. Number of participants with clinically significant abnormalities in 12-lead ECG will be reported.
Time Frame
Baseline up to Week 52
Title
Change From Baseline in Renal Function at Week 52
Description
Renal function is assessed by estimated glomerular filtration rate (eGFR) using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula for greater than or equal to (>=) 18 years participants, eGFR = 141 x min (Serum Creatinine [Scr]/κ,1)^(α) x max(Scr/κ,1)^(-1.209) x 0.993^(Age) x 1.018 (if female) x 1.159 (if black) where: Scr is serum creatinine (milligram per deciliter [mg/dL]); κ is 0.7 for females and 0.9 for males; α is -0.329 for females and -0.411 for males; min indicates the minimum of Scr/κ or 1; max indicates the maximum of Scr /κ or 1. For less than (<) 18 years participants, Counahan-Barratt equation will be used for calculation of eGFR. eGFR = (0.43 × height in centimeter [cm])/Scr where, Scr is serum creatinine (mg/dL). Change from baseline in renal function at Week 52 will be reported.
Time Frame
Baseline, Week 52
Title
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) Values at Weeks 8, 16, 28 and 40
Description
The eGFR will be calculated by CKD-EPI formula for >=18 years participants. eGFR = 141 x min (Serum Creatinine [Scr]/κ,1)^(α) x max(Scr/κ,1)^(-1.209) x 0.993^(Age) x 1.018 (if female) x 1.159 (if black) where: Scr is serum creatinine (mg/dL); κ is 0.7 for females and 0.9 for males; α is -0.329 for females and -0.411 for males; min indicates the minimum of Scr/κ or 1; max indicates the maximum of Scr /κ or 1. For <18 years participants, Counahan-Barratt equation will be used for calculation of eGFR. eGFR = (0.43 × height in cm)/Scr where, Scr is serum creatinine (mg/dL). Change from baseline in eGFR values at Weeks 8, 16, 28 and 40 will be reported.
Time Frame
Baseline, Weeks 8, 16, 28 and 40
Title
Change From Baseline in Left Ventricular Mass Index (LVMI)
Description
Change from baseline in LVMI will be measured by echocardiography at Weeks 16 and 52.
Time Frame
Baseline, Weeks 16 and 52
Title
Change From Baseline in Left Ventricular Ejection Fraction (LVEF)
Description
Change from baseline in LVEF will be measured by echocardiography at Weeks 16 and 52.
Time Frame
Baseline, Weeks 16 and 52
Title
Change From Baseline in Urine Protein/Creatinine Ratio at Weeks 8, 16, 28, 40 and 52
Description
Change from baseline in urine protein/creatinine ratio at Weeks 8, 16, 28, 40 and 52 will be reported.
Time Frame
Baseline, Weeks 8, 16, 28, 40 and 52
Title
Change From Baseline in Pain as Assessed by Brief Pain Inventory Short Form (BPI-short Form) at Weeks 8, 16, 28, 40 and 52
Description
BPI-short form is a validated self-report measure to assess the severity of pain and the impact of pain on daily functions. BPI-short form has 4 questions that assess pain intensity (worst, least, average, right now) on 10-point rating scales (0=No pain to 10=Pain as bad as you can imagine) and 7 questions that assess impact of pain on daily functions (general activity, mood, walking ability, normal work, relations with other people, sleep, enjoyment of life) on 10-point rating scales as (0=Does not interfere to 10=Completely interferes). Total score is reported as sum of individual questions score ranging from 0 to 10 with higher numbers indicating worse outcomes. Change from baseline in pain as assessed by BPI-short form at Weeks 8, 16, 28, 40 and 52 will be reported.
Time Frame
Baseline, Weeks 8, 16, 28, 40 and 52
Title
Change From Baseline in Plasma Globotriaosylsphingosine (Lyso-Gb3) Level at Weeks 8, 16, 28, 40 and 52
Description
Change from baseline in plasma Lyso-Gb3 levels at Weeks 8, 16, 28, 40 and 52 will be reported.
Time Frame
Baseline, Weeks 8, 16, 28, 40 and 52
Title
Change From Baseline in Audiology Testing Values
Description
Audiology testing will include pure tone conduction and bone conduction for each ear using 4 different pure tone frequencies (500 hertz [Hz], 1000 Hz, 2000 Hz, and 4000 Hz). Any changes in threshold will be categorized as conductive, sensorineural, or unknown. As planned, only participants <18 years of age will assessed for this outcome measure. Change from baseline in audiology testing values will be reported.
Time Frame
Baseline, Weeks 8, 16, 28, 40 and 52
Title
Area Under Serum Concentration-time Curve From the Time of Dosing to the Last Measurable concentration (AUC0-last) of REPLAGAL
Description
AUC0-last will be reported.
Time Frame
At Weeks 0 and 28
Title
Area Under Serum Concentration-time Curve From Time Zero Extrapolated to Infinity (AUC0-inf) of REPLAGAL
Description
AUC0-inf will be reported.
Time Frame
At Weeks 0 and 28
Title
Serum Clearance of Administered Dose (CL) of REPLAGAL
Description
Clearance is defined as a quantitative measure of the rate at which a drug substance is removed from the body. CL will be reported.
Time Frame
At Weeks 0 and 28
Title
Serum Clearance of Administered Dose Normalized Based on Body Weight of REPLAGAL
Description
Clearance is defined as a quantitative measure of the rate at which a drug substance is removed from the body. CL will be reported.
Time Frame
At Weeks 0 and 28
Title
Maximum Observed Serum Concentration (Cmax) of REPLAGAL
Description
Cmax will be reported.
Time Frame
At Weeks 0 and 28
Title
Terminal Elimination Half-life (T1/2) of REPLAGAL
Description
T1/2 is defined as the natural log of 2 divided by the terminal rate constant (ƛz). T1/2 will be reported.
Time Frame
At Weeks 0 and 28
Title
Time to Reach Maximum Observed Serum Concentration (Tmax) of REPLAGAL
Description
Tmax will be reported.
Time Frame
At Weeks 0 and 28
Title
Volume of Distribution at Steady State (Vss) of REPLAGAL
Description
Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired serum concentration of a drug. Vss will be reported.
Time Frame
At Weeks 0 and 28
Title
Volume of Distribution at Steady State Normalized Based on Body Weight of REPLAGAL
Description
Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired serum concentration of a drug. Vss will be reported.
Time Frame
At Weeks 0 and 28
Title
Dose Normalized Area Under Serum Concentration-time Curve From Time Zero to the Last Sampling Time (AUClast/Dose) of REPLAGAL
Description
AUClast/Dose will be reported.
Time Frame
At Weeks 0 and 28
Title
Dose Normalized Area Under the Serum Concentration-time Curve From Time Zero Extrapolated to Infinity (AUC0-inf/Dose) of REPLAGAL
Description
AUC0-inf/Dose will be reported.
Time Frame
At Weeks 0 and 28
Title
Dose Normalized Maximum Observed Serum Concentration (Cmax/Dose) of REPLAGAL
Description
Cmax/Dose will be reported.
Time Frame
At Weeks 0 and 28
10. Eligibility
Sex
All
Minimum Age & Unit of Time
7 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Participant and/or legally authorized representative must voluntarily sign an Institutional Review Board/Independent Ethics Committee approved written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the participant. For the participants less than (<) 18 years old, participants will give assent AND their parents/legally authorized representative should sign the ICF accordingly.
The participant has confirmed diagnosis of Fabry disease as determined by the investigator, according to medical record including:
For male participant, Fabry disease is confirmed by a deficiency of α-galactosidase A (GLA) activity and a mutation in the GLA gene
For female participant, Fabry disease is confirmed by a mutation in the GLA gene.
The participant is 7 to 65 years of age, inclusive, at screening.
Female participants of childbearing potential must have a negative pregnancy test at screening.
Female participants of childbearing potential must agree to use a medically acceptable method of contraception at all times during the study and for at least 14 days after the final investigational product infusion.
The participant is deemed, as determined by the investigator, to have adequate general health to undergo the specified protocol-related procedures and to have no safety or medical contraindications for participation.
The participant has not received any treatment (approved or investigational) specific to Fabry disease, such as ERT, chaperone therapy, or substrate reduction therapy.
The adult participant (greater than or equal to [>=] 18 years old) must have an estimated glomerular filtration rate (eGFR) of 45 to 120 milliliter per minute per 1.73 meter square (mL/min/1.73 m^2). Serum creatinine is tested and the eGFR is calculated by central laboratory using the Chronic Kidney Disease Epidemiology (CKD-EPI) equation.
Exclusion Criteria:
In the opinion of the investigator, the participant's life expectancy is less than or equal to (<=) 5 years.
The participant has undergone or is scheduled to undergo kidney transplantation or is currently on dialysis, or has any signs or symptoms of end stage renal disease.
The participant has a urine protein/creatinine ratio of greater than (>) 500 milligram per gram (mg/g).
The participant has a clinically relevant history of allergy or signs or symptoms of severe hypersensitivity, which in the investigator's judgment, will substantially increase the participant's risk if he or she participates in the study.
In the opinion of the investigator, the participant has non-Fabry disease-related cause of end organ (renal, cardiovascular, central nervous system) dysfunction/failure or is receiving medications that may affect the rate of disease progression, as assessed by renal measures.
The participant has a positive test result at screening for hepatitis B surface antigen with detectable hepatitis B viral deoxyribonucleic acid (DNA) load, hepatitis C virus (HCV) antibody with confirmation by HCV ribonucleic acid polymerase chain reaction testing, or human immunodeficiency virus antibody.
The participant has received prior treatment with any of the following medications, with the exception of non-systemic use:
Chloroquine
Amiodarone
Monobenzone
Gentamicin
The participant is pregnant or lactating.
The participant has a body mass index >35 kilogram per square meter (kg/m^2).
The participant is treated or has been treated with any investigational drug for indication other than Fabry disease within 30 days of study start.
The participant and/or the participant's parent(s) or legal guardians are unable to understand the nature, scope, and possible consequences of the study.
The participant is unable to comply with the protocol, example, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for evaluations, or is otherwise unlikely to complete the study, as determined by the investigator.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Takeda Contact
Phone
+1-877-825-3327
Email
medinfoUS@takeda.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Peking Union Medical College Hospital
City
Beijing
ZIP/Postal Code
Dongcheng District
Country
China
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+86 13371628442
Email
Zhengqingqiu33@aliyun.com
First Name & Middle Initial & Last Name & Degree
Qiu Zhengqing, MD
Facility Name
Xiangya Hospital, Central South University
City
Changsha
ZIP/Postal Code
Kaifu District
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+86 13548966986
Email
pengjing4346@163.com
First Name & Middle Initial & Last Name & Degree
Peng Jing, MD
Facility Name
West China Hospital, Sichuan University
City
Chengdu
ZIP/Postal Code
Wuhou District
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+86 18980602149
Email
chenyucheng2003@126.com
First Name & Middle Initial & Last Name & Degree
Chen Yucheng, MD
Facility Name
The Children's Hospital of Zhejiang University School of Medicine
City
Hangzhou
ZIP/Postal Code
Xiacheng District
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+86 13516819071
Email
maojh88@zju.edu.cn
First Name & Middle Initial & Last Name & Degree
Mao Jianhua, MD
Facility Name
Shandong Provincial Hospital
City
Jinan
ZIP/Postal Code
Huaiyin District
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+86 13791082272
Email
wrjlsd@126.com
First Name & Middle Initial & Last Name & Degree
Wang Rong, MD
Facility Name
Ruijin Hospital, Shanghai Jiaotong Uni. School of Med.
City
Shanghai
ZIP/Postal Code
Huangpu District
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
+86 13601638963
Email
cnrj100@126.com
First Name & Middle Initial & Last Name & Degree
Chen Nan, MD
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
IPD Sharing Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
IPD Sharing URL
https://vivli.org/ourmember/takeda/
Links:
URL
https://clinicaltrials.takeda.com/study-detail/61031f84beb21d002a926063
Description
To obtain more information on the study, click here/on this link
Learn more about this trial
A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease
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