Extension Study of PRX-102 for up to 60 Months

• ClinicalTrials.gov Identifier: NCT01981720
• Recruitment Status: Completed
• First Posted: November 11, 2013
• Results First Posted: February 8, 2023
• Last Update Posted: February 8, 2023
• Sponsor: Protalix
• Information provided by (Responsible Party): Protalix

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Fabry Disease	Biological: PRX-102 (pegunigalsidase alfa)	Phase 1; Phase 2

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 15 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for up to 60 Months to Adult Fabry Patients
• Actual Study Start Date: January 16, 2014
• Actual Primary Completion Date: August 26, 2020
• Actual Study Completion Date: November 9, 2021

Arms and Interventions

Arm	Intervention/treatment
Experimental: PRX-102 (pegunigalsidase alfa); PRX-102 (pegunigalsidase alfa) 1.0 mg/kg IV every 2 weeks (+/- 3 days)	Biological: PRX-102 (pegunigalsidase alfa); PRX-102 1 mg/kg every 2 weeks; Other Names: pegunigalsidase alfa; Recombinant human alpha galactosidase-A

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03 [ Time Frame: 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.

Other Outcome Measures:

1. Plasma Lyso-Gb3 Concentration [ Time Frame: Baseline and month 60 ]
   Globotriaosylsphingosine (Lyso-Gb3) is Fabry disease specific biomarker, which was measured at Baseline, every 3 months up to 24 months and then every 6 months up to the end of the study. Baseline and Month 60 and change from Baseline reported.
2. Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: Baseline and Month 60 ]
   eGFR was calculated based on the serum creatinine values according to the CKD-EPI equation. The absolute change in eGFR from baseline measurement at visit 1 to Month 60 was summarized using descriptive statistics. Baseline and month 60 reported.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Locations

United States, Florida

University of Florida

Gainesville, Florida, United States, 32611

United States, Iowa

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States, 52242

United States, Maryland

Johns Hopkins University School of Medicine

Baltimore, Maryland, United States, 21205

United States, Texas

Institute of Metabolic Disease

Dallas, Texas, United States, 75226

United States, Virginia

O & O Alpan

Fairfax, Virginia, United States, 22030

Paraguay

Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)

Asunción, Paraguay

Spain

Hospital de Dia Quiron Zaragoza

Zaragoza, Spain, 50012

United Kingdom

The Royal Free Hospital

London, United Kingdom, NW3 2QG

Sponsors and Collaborators

Protalix

Investigators

• Study Director: Raul Chertkoff, MD; Protalix Ltd.

  Study Documents (Full-Text)

Documents provided by Protalix:

Study Protocol  [PDF] January 5, 2016

Statistical Analysis Plan  [PDF] June 2, 2021

More Information

• Responsible Party: Protalix
• ClinicalTrials.gov Identifier: NCT01981720
• Other Study ID Numbers: PB-102-F03
• First Posted: November 11, 2013
• Results First Posted: February 8, 2023
• Last Update Posted: February 8, 2023
• Last Verified: January 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes

Keywords provided by Protalix:

PRX-102; pegunigalsidase alfa; Fabry Disease

Additional relevant MeSH terms:

Fabry Disease; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Lipid Metabolism Disorders