Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

Study Overview
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Brief Summary
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The objective of CLI-06657AA1-04 (formerly PB-102-F60) is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who have successfully completed studies PB-102-F03, PB-102-F20 or PB-102-F30.

Detailed Description
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This is an open-label study. Patients will be enrolled to receive 1 mg/kg pegunigalsidase alfa as intravenous infusions every 2 weeks (±3 days). The duration of treatment is until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor. For the analysis, available efficacy and safety parameters will be summarized using descriptive statistics.

Official Title
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Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease

Conditions
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Fabry Disease

Intervention / Treatment
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* Drug: pegunigalsidase alfa


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Completion of study PB-102-F20, PB-102-F03, or PB-102-F30 The patient signs informed consent Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination. Exclusion Criteria: Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Open-label extension study
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Experimental open label<br>pegunigalsidase alfa | Drug: pegunigalsidase alfa<br>* Recombinant human alpha galactosidase A<br>* Other names: PRX-102;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluation of treatment-related adverse events | CTCAE v4.03 | Throughout the study, 364 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Kidney function 1 | Estimated glomerular filtration rate (eGFRCKD-EPI) | Every 6 months throughout trial, 364 weeks | 
| Cardiac assessment | Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI) and Echocardiograph, and exercise tolerance (Stress Test) | Every 12 months to end of the study, 7 years | 
| Biomarkers for Fabry disease | plasma Lyso-Gb3 and Gb3 | Every 12 months to end of the study, 7 years. For patients from PB-102-F20 at 3 and 6 months. | 
| Record of pain medication use | Frequency of pain medication use, or pre-infusion medication | Every two weeks for 7 years | 
| Kidney function 2 | Protein/Creatinine ratio, spot urine test (UPCR) | Every 6 months to the end of the study, 7 years | 
| Pain assessment | short form Brief Pain Inventory (BPI) | Every 6 months up to the end of the study, 7 years | 
| Symptom assessment | Mainz Severity Score Index (MSSI) | Every 12 months up to the end of the study, 7 years | 
| Quality of life assessment | quality of life (EQ-5D-5L) | Every 6 months up to the end of the study, 7 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Glomerular filtration rate, Proteinuria, PRX-102, pegunigalsidase alfa, Fabry disease