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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01981720
Other study ID # PB-102-F03
Secondary ID
Status Completed
Phase Phase 1/Phase 2
First received
Last updated
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
Health authority
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

Study Design


Related Conditions & MeSH terms


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
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