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

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT01769001
First received: January 14, 2013
Last updated: September 26, 2014
Last verified: September 2014
  Purpose

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.


Condition Intervention Phase
Fabry Disease
Drug: PRX-102
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Protalix:

Primary Outcome Measures:
  • Number of participants with adverse events as a measure of safety [ Time Frame: 38 weeks ] [ Designated as safety issue: Yes ]
    Adverse events reported by the patient and from monitoring with clinical laboratory, physical examination, ECG


Secondary Outcome Measures:
  • Gb3 concentrations [ Time Frame: 38 weeks ] [ Designated as safety issue: No ]
    Gb3 concentrations in plasma and urine sediment

  • Glomerular filtration rate [ Time Frame: 38 weeks ] [ Designated as safety issue: No ]
    Measurement of glomerular filtration

  • Pain [ Time Frame: 38 weeks ] [ Designated as safety issue: No ]
    Short term brief pain inventory

  • Proteinuria [ Time Frame: 38 weeks ] [ Designated as safety issue: No ]
    Measure of kidney function


Estimated Enrollment: 18
Study Start Date: March 2013
Estimated Study Completion Date: September 2015
Estimated Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 mg/kg
PRX-102 1 mg/kg every 2 weeks
Drug: PRX-102
Comparison of different doses of drug
Other Name: plant cell expressed recombinant human alpha-galactosidase-A
Experimental: 2 mg/kg
PRX-102 2 mg/kg every 2 weeks
Drug: PRX-102
Comparison of different doses of drug
Other Name: plant cell expressed recombinant human alpha-galactosidase-A
Experimental: 0.2 mg/kg
PRX-102 0.2 mg/kg every 2 weeks
Drug: PRX-102
Comparison of different doses of drug
Other Name: plant cell expressed recombinant human alpha-galactosidase-A

Detailed 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).

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01769001

Locations
United States, Georgia
Department of Human Genetics, Emory University School of Medicine
Atlanta, Georgia, United States, 30322
United States, Iowa
University of Iowa Health Clinics
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21205
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Texas
Research Baylor Institute of Metabolic Disease
Dallas, Texas, United States, 75226
United States, Virginia
O & O Alpan LLC
Fairfax, Virginia, United States, 22030
Australia
Royal Melbourne Hospital
Victoria, Australia, 3050
Paraguay
Hematology and Clinical Research Private Institute
Asuncion, 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: Einat Almon, PhD Protalix Biotherapeutics
  More Information

No publications provided

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01769001     History of Changes
Other Study ID Numbers: PB-102-F02
Study First Received: January 14, 2013
Last Updated: September 26, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Protalix:
Fabry disease
Alpha galactosidase deficiency
Metabolic storage disease

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

ClinicalTrials.gov processed this record on October 20, 2014