Extension Study of PRX-102 for up to 60 Months

Phase 1

Completed

• Conditions: Fabry Disease
• Interventions: Biological: PRX-102 (pegunigalsidase alfa)

• Registration Number: NCT01981720
• Lead Sponsor: Protalix

Brief Summary

To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02.

Detailed Description

An open-label study to evaluate the ongoing safety, tolerability and efficacy parameters of PRX-102 in adult Fabry patients (≥18 years of age). Patients enrolled received 1.0 mg/kg of PRX-102 as an intravenous infusion every 2 weeks (+/- 3 days) for up to 60 months and no less than 36 months.

Study Timeline

• Study First Submitted: 2013-11-05
• Study First Submitted QC: 2013-11-05
• Study First Posted: 2013-11-11
• Study Start: 2014-01-16
• Primary Completion: 2020-08-26
• Study Completion: 2021-11-09
• Results First Submitted: 2023-01-15
• Results First Submitted QC: 2023-01-15
• Results First Posted: 2023-02-08
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-10
• Last Update Posted: 2023-09-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

• Completion of study PB-102-F02
• The patient signs informed consent
• Female patients and male patients whose co-partners were of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms. Contraception should be used throughout the duration of the study and for 3 months after termination of treatment.

Exclusion Criteria

• Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient's compliance with the requirements of the study

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
PRX-102 (pegunigalsidase alfa)	PRX-102 (pegunigalsidase alfa)	PRX-102 (pegunigalsidase alfa) 1.0 mg/kg IV every 2 weeks (+/- 3 days)

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03	Every two weeks up to 60 months	Results represent the number of treatment-emergent adverse events (TEAE) that were considered definitely, probably or possibly related to study treatment.

Trial Locations

Locations (8)

Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C); 🇵🇾 Asunción, Paraguay

Hospital de Dia Quiron Zaragoza; 🇪🇸 Zaragoza, Spain

The Royal Free Hospital; 🇬🇧 London, United Kingdom

University of Florida; 🇺🇸 Gainesville, Florida, United States

Johns Hopkins University School of Medicine; 🇺🇸 Baltimore, Maryland, United States

Institute of Metabolic Disease; 🇺🇸 Dallas, Texas, United States

O & O Alpan; 🇺🇸 Fairfax, Virginia, United States

University of Iowa Hospitals and Clinics; 🇺🇸 Iowa City, Iowa, United States