Extension Study of PRX-102 for up to 60 Months

Fabry Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01981720
• Other study ID #: PB-102-F03
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: January 16, 2014
• Est. completion date: November 9, 2021

Study information

• Verified date: September 2023
• Source: Protalix
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: November 9, 2021
• Est. primary completion date: August 26, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Biological:
• PRX-102 (pegunigalsidase alfa)
PRX-102 1 mg/kg every 2 weeks

Locations

Country	Name	City	State
Paraguay	Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)	Asunción
Spain	Hospital de Dia Quiron Zaragoza	Zaragoza
United Kingdom	The Royal Free Hospital	London
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 Florida	Gainesville	Florida
United States	University of Iowa Hospitals and Clinics	Iowa City	Iowa

Sponsors (2)

Lead Sponsor	Collaborator
Protalix	Chiesi Farmaceutici S.p.A.

Countries where clinical trial is conducted

United States,  Paraguay,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Plasma Lyso-Gb3 Concentration	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.	Baseline and month 60
Other	Estimated Glomerular Filtration Rate (eGFR)	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.	Baseline and Month 60
Primary	Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03	Results represent the number of treatment-emergent adverse events (TEAE) that were considered definitely, probably or possibly related to study treatment.	Every two weeks up to 60 months