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Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease (RISE)

Primary Purpose

Fabry Disease

Status
Recruiting
Phase
Phase 2
Locations
Japan
Study Type
Interventional
Intervention
PRX-102 1 mg/kg every 2 weeks
PRX-102 2 mg/kg every 4 weeks
Sponsored by
Chiesi Farmaceutici S.p.A.
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 (all subjects) Must have been born in Japan and have their biological parents and all 4 grandparents of Japanese descent A documented diagnosis of Fabry disease, as determined by the following: Males: Plasma and/or leukocyte alpha-galactosidase-A activity (by activity assay) that is ≤ 5% of mean normal laboratory levels or, if the enzymatic activity is above the 5% limit but still under the normal level, a confirmed disease-causing mutation of the GLA gene Females: Historical genetic test results consistent with Fabry mutations or, in the case of novel mutations, a first-degree male relative with Fabry disease All subjects: At least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma Estimated glomerular filtration rate (eGFR) at screening ≥40 mL/min/1.73 m2. For adults, this will be calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Creatinine equation (2009); and for adolescents, it will be calculated using the Creatinine Cystatin C-based Chronic Kidney Disease in Children (CKiD) equation. Clinical condition that in the opinion of the Investigator requires treatment with ERT Additional inclusion criteria for subjects in Cohort A Aged ≥18 to ≤60 years Treatment with agalsidase beta for at least the last 12 months, with the dose stable (defined as having received at least 80% of the labelled dose) for at least the last 6 months Diagnosis of kidney impairment, defined as a linear slope of eGFR more negative or equal to -2 mL/min/1.73 m2/year. The historical eGFR slope will be calculated based on at least 3 serum creatinine values obtained over the past 9 to 24 months prior to screening, using the CKD-EPI Creatinine equation (2009). This criterion will be confirmed at screening by calculating the screening eGFR slope using historical and screening serum creatinine values. Both historical and screening eGFR slopes will be used for the diagnosis of kidney impairment. Additional inclusion criterion for subjects in Cohort B - Aged ≥18 to ≤60 years Additional inclusion criteria for subjects in Cohort C Aged ≥13 to <18 years If they previously received or are currently receiving ERT treatment, the subjects must be negative for anti-drug antibodies for PRX-102 Exclusion Criteria: Administration of ERT for Fabry disease within 14 days before baseline, or chaperone therapy for Fabry disease within 3 days before baseline History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or to any component of the study drug Cohort A only: eGFR value of >90 to ≤120 mL/min/1.73 m2 at screening and a historical eGFR value >120 mL/min/1.73 m2 in the past 9 to 24 months before screening, indicating absence of renal impairment Urine protein to creatinine ratio (UPCR) >0.5 g/g (0.5 mg/mg or 500 mg/g) if not treated with an ACE inhibitor or ARB Initiation of treatment, or a change in dose to ongoing treatment, with an angiotensin-converting-enzyme inhibitor (ACEI) or angiotensin II receptor blocker (ARB) in the 4 weeks prior to screening. Currently taking another investigational drug for any condition Known non-pathogenic Fabry mutations History of renal dialysis or kidney transplantation History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and renal vasculitis); non-specific conditions (e.g., ischemia, toxic injury); as well as extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive nephropathy History of (or current) malignancy requiring treatment; the one exception is a prior history of resected basal cell carcinoma Severe cardiomyopathy or significant unstable cardiac disease within 6 months prior to screening A positive test for Severe Acute Respiratory Syndrome-Coronavirus 2 (SARS-CoV-2) within 3 months prior to screening, using a validated molecular assay or validated antigen assay Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until 30 days after the last study treatment Presence of any medical, emotional, behavioral, or psychological condition that in the judgment of the Investigator could interfere with the subject's compliance with the requirements of the study

Sites / Locations

  • Fukuoka University Chikushi Hospital
  • Tohoku University Hospital
  • University of the Ryukyu HospitalRecruiting
  • Osaka University Hospital
  • Juntendo University Hospital, 3-1-3 Hongo, Bunkyo-ku, Tokyo
  • Tokyo Jikei University HospitalRecruiting
  • Keio University Hospital
  • Niigata University Medical & Dental HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PRX-102 1 mg/kg every 2 weeks or PRX-102 2 mg/kg every 4 weeks

Arm Description

PRX-102 1 mg/kg every 2 weeks or PRX-102 2 mg/kg every 4 weeks (available only in the optional extension part)

Outcomes

Primary Outcome Measures

Incidence of Treatment Emergent Adverse Events (TEAEs)
Incidence of Infusion Related Reactions (IRRs)
Incidence of Injection site reactions (ISRs)
Change of laboratory tests' results
Change in in body weight in kilograms
Change in height in centimeters
Change in Tanner stage
Tanner Staging of Sexual Development will be used to assess sexual development (i.e. breast development (B1 to B5) and pubic hair development (Ph-1 to Ph-5) in females and pubic hair and genetical development (G1-G5) in males.
Change from baseline of 12-lead ECG quantitative parameters: Mean Heart Rate, PR Interval, QRS Duration, QT Interval, QTc Interval, and ST Segment
Quantitative ECG parameters will be summarized by cohort and overall
Incidence of treatment-emergent Anti-Drug Antibodies (ADAs)
ADA status change from baseline
Incidence of premedication use at each visit and change of infusion premedications from baseline
Change from baseline of Maximum plasma concentration (Cmax), pharmacokinetic parameter
Change from baseline of Time to maximum plasma concentration (tmax), pharmacokinetic parameter
Change from baseline of Area under the plasma concentration-time curve from time 0 to time t (AUC0 t), pharmacokinetic parameter
Change from baseline of Area under the curve from time 0 to 2 weeks (AUC0-2wk), pharmacokinetic parameter
Change from baseline of Area under the curve from time 0 to infinity (AUC0-∞), pharmacokinetic parameter
Change from baseline of Terminal half-life (t1/2), pharmacokinetic parameter
Change from baseline of Area under the curve over a dosing interval (AUCτ), pharmacokinetic parameter
Change from baseline of Observed drug concentration at the end of the dosing interval (Cτ), pharmacokinetic parameter
Change from baseline of Clearance (Cl), pharmacokinetic parameter
Change from baseline of Volume of distribution (Vz), pharmacokinetic parameters

Secondary Outcome Measures

Full Information

First Posted
December 13, 2022
Last Updated
September 22, 2023
Sponsor
Chiesi Farmaceutici S.p.A.
Collaborators
ICON plc
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1. Study Identification

Unique Protocol Identification Number
NCT05710692
Brief Title
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
Acronym
RISE
Official Title
A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Pegunigalsidase Alfa (PRX-102) in Japanese Patients With Fabry Disease (RISE)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 1, 2023 (Actual)
Primary Completion Date
March 2026 (Anticipated)
Study Completion Date
March 2028 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Chiesi Farmaceutici S.p.A.
Collaborators
ICON plc

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
The aim of this study is to evaluate the safety and efficacy of pegunigalsidase alfa in Japanese patients (adults and adolescents) affected by Fabry disease. It is planned of a total of approximately 18-20 male and female Fabry disease patients between the ages of 13 and 60 years to be part of the study. The study is conducted in Japan.
Detailed Description
Investigators are doing this study to find out if treatment with pegunigalsidase alfa will prevent or reduce the development of health problems caused by Fabry disease and thereby improve patients' health and quality of life. pegunigalsidase alfa (PRX-102) is a drug made using genetic engineering techniques and manufactured using cultured tobacco cells. It is given by intravenous infusion every 2 weeks, at a dosage of 1 milligram per kilogram (mg/kg) of body weight. The study consists of a main study that is divided into two stages, each of which will last one year, followed by an optional extension study. In the optional extension stage, the participants may receive PRX-102 intravenous infusion every 2 weeks, at a dosage of 1 milligram per kilogram (mg/kg) of body weight or every 4 weeks at a dosage of 2 milligrams per kilogram (mg/kg) of body weight. There are three groups (cohorts) in this study, with adults enrolled in either Cohort A or B and adolescents in Cohort C. Whether an adult is assigned to Cohort A or Cohort B depends on their kidney function and treatment history. This study will start with a screening visit of up to 4 weeks. It will be followed up by infusion visits every 2 weeks or 4 weeks. For subjects not continuing in the extension stage, a follow-up call is to be made 30 days after the last study drug infusion.

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 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
18 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
PRX-102 1 mg/kg every 2 weeks or PRX-102 2 mg/kg every 4 weeks
Arm Type
Experimental
Arm Description
PRX-102 1 mg/kg every 2 weeks or PRX-102 2 mg/kg every 4 weeks (available only in the optional extension part)
Intervention Type
Drug
Intervention Name(s)
PRX-102 1 mg/kg every 2 weeks
Other Intervention Name(s)
pegunigalsidase alfa, Recombinant human alpha galactosidase-A
Intervention Description
PRX-102 1 mg/kg every 2 weeks
Intervention Type
Drug
Intervention Name(s)
PRX-102 2 mg/kg every 4 weeks
Other Intervention Name(s)
pegunigalsidase alfa, Recombinant human alpha galactosidase-A
Intervention Description
PRX-102 2 mg/kg every 4 weeks
Primary Outcome Measure Information:
Title
Incidence of Treatment Emergent Adverse Events (TEAEs)
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Incidence of Infusion Related Reactions (IRRs)
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Incidence of Injection site reactions (ISRs)
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change of laboratory tests' results
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change in in body weight in kilograms
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change in height in centimeters
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change in Tanner stage
Description
Tanner Staging of Sexual Development will be used to assess sexual development (i.e. breast development (B1 to B5) and pubic hair development (Ph-1 to Ph-5) in females and pubic hair and genetical development (G1-G5) in males.
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change from baseline of 12-lead ECG quantitative parameters: Mean Heart Rate, PR Interval, QRS Duration, QT Interval, QTc Interval, and ST Segment
Description
Quantitative ECG parameters will be summarized by cohort and overall
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Incidence of treatment-emergent Anti-Drug Antibodies (ADAs)
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
ADA status change from baseline
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Incidence of premedication use at each visit and change of infusion premedications from baseline
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change from baseline of Maximum plasma concentration (Cmax), pharmacokinetic parameter
Time Frame
Assessments will be done over four two-week periods, starting at Baseline (Week 0), V7 (Week 12), V14 (Week 26), and V27 (Week 52)
Title
Change from baseline of Time to maximum plasma concentration (tmax), pharmacokinetic parameter
Time Frame
Assessments will be done over four two-week periods, starting at Baseline (Week 0), V7 (Week 12), V14 (Week 26), and V27 (Week 52)
Title
Change from baseline of Area under the plasma concentration-time curve from time 0 to time t (AUC0 t), pharmacokinetic parameter
Time Frame
Assessments will be done over four two-week periods, starting at Baseline (Week 0), V7 (Week 12), V14 (Week 26), and V27 (Week 52)
Title
Change from baseline of Area under the curve from time 0 to 2 weeks (AUC0-2wk), pharmacokinetic parameter
Time Frame
Assessments will be done over four two-week periods, starting at Baseline (Week 0), V7 (Week 12), V14 (Week 26), and V27 (Week 52)
Title
Change from baseline of Area under the curve from time 0 to infinity (AUC0-∞), pharmacokinetic parameter
Time Frame
Assessments will be done over four two-week periods, starting at Baseline (Week 0), V7 (Week 12), V14 (Week 26), and V27 (Week 52)
Title
Change from baseline of Terminal half-life (t1/2), pharmacokinetic parameter
Time Frame
Assessments will be done over four two-week periods, starting at Baseline (Week 0), V7 (Week 12), V14 (Week 26), and V27 (Week 52)
Title
Change from baseline of Area under the curve over a dosing interval (AUCτ), pharmacokinetic parameter
Time Frame
Assessments will be done over four two-week periods, starting at Baseline (Week 0), V7 (Week 12), V14 (Week 26), and V27 (Week 52)
Title
Change from baseline of Observed drug concentration at the end of the dosing interval (Cτ), pharmacokinetic parameter
Time Frame
Assessments will be done over four two-week periods, starting at Baseline (Week 0), V7 (Week 12), V14 (Week 26), and V27 (Week 52)
Title
Change from baseline of Clearance (Cl), pharmacokinetic parameter
Time Frame
Assessments will be done over four two-week periods, starting at Baseline (Week 0), V7 (Week 12), V14 (Week 26), and V27 (Week 52)
Title
Change from baseline of Volume of distribution (Vz), pharmacokinetic parameters
Time Frame
Assessments will be done over four two-week periods, starting at Baseline (Week 0), V7 (Week 12), V14 (Week 26), and V27 (Week 52)
Other Pre-specified Outcome Measures:
Title
Change in eGFR
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change in annualized eGFR slope
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change in urine albumin levels
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change in urine protein levels
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Incidence of changes in echocardiogram results
Description
Systolic and diastolic heart function and structure is assessed by ultrasound of the heart. Echocardiogram parameters include left ventricular mass index (LVMi), ejection fraction, fractional shortening, left ventricular mass, valve abnormalities and thickness.
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Incidence of changes in Holter ECG
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change of cardiac biomarkers
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Adults only: Response of the heart to external stress induced by exercise measured with Stress test (Bruce protocol)
Description
Qualitative evaluation (yes/no) of symptoms (chest pain, shortness of breath, dizziness, palpitations, and other) and the overall impression: normal stress test (yes/no) will be summarized. For overall impression only, a shift from baseline will be presented: normal stress test (yes / no).
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Adults only: change of Cardiac MRI
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Adults only: change of Brain MRI
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change in plasma level of Gb3 concentration (nM)
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change in plasma level of lyso-Gb3 (nM)
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change in urine level of lyso-Gb3 (nM)
Time Frame
12 Months, 24 Months and through study completion (an average of 4.5 years)
Title
Change from baseline of Mainz Severity Score Index (MSSI) scores
Description
Domains (general, neurological, cardiovascular, renal dysfunction)
Time Frame
12 Months, 24 Months and at the end of study
Title
Incidence of change from baseline in the number of different pain medications
Time Frame
12 Months, 24 Months and at the end of study
Title
Incidence of Fabry Clinical Events
Description
FCEs are classified into four categories: renal, cardiac, cerebrovascular and death due to non-cardiac reasons
Time Frame
12 Months, 24 Months and at the end of study
Title
Adults only: change in Gastrointestinal Symptom Rating Scale (GSRS) scores
Description
To measure common symptoms of gastrointestinal disorders.
Time Frame
12 Months, 24 Months and at the end of study
Title
Adults only: change in Brief Pain Inventory - Short Form (BPI-SF) scores
Time Frame
12 Months, 24 Months and at the end of study
Title
Adults only: change of quality of life assessed using EQ-5D-5L questionnaire
Time Frame
12 Months, 24 Months and at the end of study
Title
Cohort C only: Change in Gastrointestinal Symptoms (PedsQL-GI) Questionnaire scores
Description
To measure common symptoms of gastrointestinal disorders.
Time Frame
12 Months, 24 Months and at the end of study
Title
Cohort C only: change in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ) scores
Time Frame
12 Months, 24 Months and at the end of study
Title
Cohort C only: change in PedsQL Pediatric Pain Questionnaire (PedsQL-PPQ) scores
Time Frame
12 Months, 24 Months and at the end of study
Title
Cohort C only: change of quality of life assessed using EQ-5D-Y Questionnaire
Time Frame
12 Months, 24 Months and at the end of study

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 (all subjects) Must have been born in Japan and have their biological parents and all 4 grandparents of Japanese descent A documented diagnosis of Fabry disease, as determined by the following: Males: Plasma and/or leukocyte alpha-galactosidase-A activity (by activity assay) that is ≤ 5% of mean normal laboratory levels or, if the enzymatic activity is above the 5% limit but still under the normal level, a confirmed disease-causing mutation of the GLA gene Females: Historical genetic test results consistent with Fabry mutations or, in the case of novel mutations, a first-degree male relative with Fabry disease All subjects: At least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma Estimated glomerular filtration rate (eGFR) at screening ≥40 mL/min/1.73 m2. For adults, this will be calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Creatinine equation (2009); and for adolescents, it will be calculated using the Creatinine Cystatin C-based Chronic Kidney Disease in Children (CKiD) equation. Clinical condition that in the opinion of the Investigator requires treatment with ERT Additional inclusion criteria for subjects in Cohort A Aged ≥18 to ≤60 years Treatment with agalsidase beta for at least the last 12 months, with the dose stable (defined as having received at least 80% of the labelled dose) for at least the last 6 months Diagnosis of kidney impairment, defined as a linear slope of eGFR more negative or equal to -2 mL/min/1.73 m2/year. The historical eGFR slope will be calculated based on at least 3 serum creatinine values obtained over the past 9 to 24 months prior to screening, using the CKD-EPI Creatinine equation (2009). This criterion will be confirmed at screening by calculating the screening eGFR slope using historical and screening serum creatinine values. Both historical and screening eGFR slopes will be used for the diagnosis of kidney impairment. Additional inclusion criterion for subjects in Cohort B - Aged ≥18 to ≤60 years Additional inclusion criteria for subjects in Cohort C Aged ≥13 to <18 years If they previously received or are currently receiving ERT treatment, the subjects must be negative for anti-drug antibodies for PRX-102 Exclusion Criteria: Administration of ERT for Fabry disease within 14 days before baseline, or chaperone therapy for Fabry disease within 3 days before baseline History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or to any component of the study drug Cohort A only: eGFR value of >90 to ≤120 mL/min/1.73 m2 at screening and a historical eGFR value >120 mL/min/1.73 m2 in the past 9 to 24 months before screening, indicating absence of renal impairment Urine protein to creatinine ratio (UPCR) >0.5 g/g (0.5 mg/mg or 500 mg/g) if not treated with an ACE inhibitor or ARB Initiation of treatment, or a change in dose to ongoing treatment, with an angiotensin-converting-enzyme inhibitor (ACEI) or angiotensin II receptor blocker (ARB) in the 4 weeks prior to screening. Currently taking another investigational drug for any condition Known non-pathogenic Fabry mutations History of renal dialysis or kidney transplantation History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and renal vasculitis); non-specific conditions (e.g., ischemia, toxic injury); as well as extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive nephropathy History of (or current) malignancy requiring treatment; the one exception is a prior history of resected basal cell carcinoma Severe cardiomyopathy or significant unstable cardiac disease within 6 months prior to screening A positive test for Severe Acute Respiratory Syndrome-Coronavirus 2 (SARS-CoV-2) within 3 months prior to screening, using a validated molecular assay or validated antigen assay Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until 30 days after the last study treatment Presence of any medical, emotional, behavioral, or psychological condition that in the judgment of the Investigator could interfere with the subject's compliance with the requirements of the study
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Chiesi Clinical Trial
Phone
+3905212791
Email
clinicaltrials_info@chiesi.com
Facility Information:
Facility Name
Fukuoka University Chikushi Hospital
City
Chikushino
State/Province
Fukuoka
ZIP/Postal Code
818-8502
Country
Japan
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Takahito Inoue
Facility Name
Tohoku University Hospital
City
Sendai
State/Province
Miyagi
ZIP/Postal Code
980-8574
Country
Japan
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Saori Yamamoto
Facility Name
University of the Ryukyu Hospital
City
Nishihara
State/Province
Okinawa
ZIP/Postal Code
903-0125
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Koichi Nakanishi
Facility Name
Osaka University Hospital
City
Suita
State/Province
Osaka
ZIP/Postal Code
565-0871
Country
Japan
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tomoko Namba
Facility Name
Juntendo University Hospital, 3-1-3 Hongo, Bunkyo-ku, Tokyo
City
Bunkyo-ku
State/Province
Tokyo
ZIP/Postal Code
113-0033
Country
Japan
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Takao Kato
Facility Name
Tokyo Jikei University Hospital
City
Minato-ku
State/Province
Tokyo
ZIP/Postal Code
105-8461
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Masahisa Kobayashi
Facility Name
Keio University Hospital
City
Shinjuku-ku
State/Province
Tokyo
ZIP/Postal Code
160-8582
Country
Japan
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hiroyuki Yamakawa
Facility Name
Niigata University Medical & Dental Hospital
City
Niigata
ZIP/Postal Code
951-8520
Country
Japan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hirofumi Watanabe

12. IPD Sharing Statement

Learn more about this trial

Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease

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