Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

Study Overview
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Brief Summary
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The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.

Detailed Description
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This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of treatment will be until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor.

Official Title
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Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult 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-F50. The patient signs informed consent. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, effective contraception method. 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

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 the duration of the study, 364 weeks | 
| Cardiac assessment | Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test | Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364 | 
| Biomarkers for Fabry disease | Plasma Lyso-Gb3 and Gb3 | Every 6 months throughout the duration of the study, 364 weeks | 
| Kidney function 2 | Protein/Creatinine ratio (UPCR), spot urine test | Every 6 months throughout the duration of the study, 364 weeks | 
| Clinical assessment | Record of pain medication and pre-medication use | Every four weeks throughout the duration of the study, 364 weeks | 
| Pain assessment | Short form Brief Pain Inventory (BPI) | Every 6 months throughout the duration of the study, 364 weeks | 
| Symptom assessment | Mainz Severity Score Index (MSSI) | Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364 | 
| Quality of life assessment | Quality of life (EQ-5D-5L) | Every 6 months throughout the duration of the study, 364 weeks | 

 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