We updated the design of this site on September 25th. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

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:
ClinicalTrials.gov Identifier:
NCT01769001
First Posted: January 16, 2013
Last Update Posted: December 9, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Protalix
  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
Intervention Model: Parallel Assignment
Masking: None (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 ]
    Adverse events reported by the patient and from monitoring with clinical laboratory, physical examination, ECG


Secondary Outcome Measures:
  • Gb3 concentrations [ Time Frame: 38 weeks ]
    Gb3 concentrations in plasma and urine sediment

  • Glomerular filtration rate [ Time Frame: 38 weeks ]
    Measurement of glomerular filtration

  • Pain [ Time Frame: 38 weeks ]
    Short term brief pain inventory

  • Proteinuria [ Time Frame: 38 weeks ]
    Measure of kidney function


Estimated Enrollment: 18
Study Start Date: March 2013
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2016 (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

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01769001     History of Changes
Other Study ID Numbers: PB-102-F02
First Submitted: January 14, 2013
First Posted: January 16, 2013
Last Update Posted: December 9, 2015
Last Verified: December 2015

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