An Extension of a Phase 1/2, Open Label, Dose Ranging Study of PRX-102 in Adult Fabry Patients

Fabry Disease Clinical Trial

Official title:

An Extension of Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 38 Weeks to Adult Fabry Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01769001
• Other study ID #: PB-102-F02
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: January 14, 2013
• Last updated: January 9, 2018
• Start date: March 2013
• Est. completion date: March 6, 2016

Study information

• Verified date: January 2018
• Source: Protalix
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Patients will be enrolled into one of three PRX-102 dosing groups (0.2 mg/kg, 1.0 mg/kg, 2.0 mg/kg), to receive the same dose they had received in Phase 1/2 study PB-102-F01, and will continue to receive PRX-102 as an intravenous infusion every 2 weeks for 38 weeks.

Description:

Patients will be enrolled into one of three PRX-102 dosing groups (0.2 mg/kg, 1.0 mg/kg, 2.0 mg/kg) to receive the same dose they had received in the Phase 1/2 study, PB-102-F01, and will continue to receive intravenous infusions of PRX-102 every 2 weeks for 38 weeks (9 months).

All exploratory endpoints that were evaluated during the Phase 1/2 study PB-102-F01 will continue to be assessed in the extension protocol (study PB-102-F02).

The additional exploratory efficacy parameters that were evaluated at baseline of the Phase 1/2 study PB-102-F01 as a reference point, before the initiation of treatment, will be evaluated after 3 months into the extension study (total treatment of 6 months). These parameters include Gb3 concentration in renal and skin tissues, LVM, MSSI and cardiac function tests (echocardiography and stress test). Cerebrovascular disease (clinical and MRI) will be evaluated at last infusion (total treatment of 12 months).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: March 6, 2016
• Est. primary completion date: March 6, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Completion of Phase 1/2 study PB-102-F01

• The patient signs informed consent

• Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method

Exclusion Criteria:

• Pregnant or nursing

• 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

Drug:
• PRX-102
Comparison of different doses of drug

Locations

Country	Name	City	State
Australia	Royal Melbourne Hospital	Parkville	Victoria
Paraguay	Hematology and Clinical Research Private Institute	Asuncion
Spain	Hospital de Dia Quiron Zaragoza	Zaragoza
United Kingdom	The Royal Free Hospital	London
United States	Department of Human Genetics, Emory University School of Medicine	Atlanta	Georgia
United States	Johns Hopkins University School of Medicine	Baltimore	Maryland
United States	Research Baylor Institute of Metabolic Disease	Dallas	Texas
United States	Duke University Medical Center	Durham	North Carolina
United States	O & O Alpan LLC	Fairfax	Virginia
United States	University of Iowa Health Clinics	Iowa City	Iowa
United States	University of Kansas Medical Center	Kansas City	Kansas

Sponsors (1)

Lead Sponsor	Collaborator
Protalix

Countries where clinical trial is conducted

United States,  Australia,  Paraguay,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with adverse events as a measure of safety	Adverse events reported by the patient and from monitoring with clinical laboratory, physical examination, ECG	38 weeks
Secondary	Gb3 concentrations	Gb3 concentrations in plasma and urine sediment	38 weeks
Secondary	Glomerular filtration rate	Measurement of glomerular filtration	38 weeks
Secondary	Pain	Short term brief pain inventory	38 weeks
Secondary	Proteinuria	Measure of kidney function	38 weeks