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Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT01678898
First received: August 31, 2012
Last updated: April 18, 2016
Last verified: April 2016
  Purpose
This is the first human treatment with PRX-102, an enzyme being developed as a long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (alpha galactosidase deficiency). The safety, tolerability, and exploratory efficacy will be evaluated in this study of increasing doses. Patients will be treated with infusions every two weeks for 12 weeks (7 infusions).

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: A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Weeks to Adult Fabry Patients

Resource links provided by NLM:


Further study details as provided by Protalix:

Primary Outcome Measures:
  • Adverse events [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
    Reportings of adverse events reported by the patient and from monitoring with clinical laboratory, physical examination and ECG


Secondary Outcome Measures:
  • Gb3 concentrations [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Gb3 concentrations in plasma and urine sediment

  • Kidney function [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Measurement of glomerular filtration and proteinuria

  • Pain [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
    Short term brief pain inventory


Enrollment: 18
Study Start Date: October 2012
Study Completion Date: January 2016
Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 0.2 mg/kg
PRX-102 0.2 mg/kg every 2 weeks
Drug: PRX-102
Other Name: plant cell expressed recombinant human alpha-galactosidase-A
Experimental: 1 mg/kg
PRX-102 1 mg/kg every 2 weeks
Drug: PRX-102
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
Other Name: plant cell expressed recombinant human alpha-galactosidase-A

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Symptomatic adult Fabry patients (≥18 yrs)
  • Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN in plasma=3.2 nmol/hr/ml, LLN in leucocytes=32 nmol/hr/mg/protein)
  • Females: historical genetic test results consistent with Fabry mutations
  • Globotriaosylceramide (Gb3) concentration in urine > 1.5 times upper normal limit
  • Patients who have never received enzyme replacement therapy (ERT) in the past, or patients who have not received ERT in the past 6 months and have a negative anti alpha galactosidase antibody test
  • eGFR ≥ 60 mL/min/1.73m2
  • 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:

  • Participation in any trial of an investigational drug within 30 days prior to study screening
  • Chronic kidney disease stages 3-5 (CKD 3-5) (Appendix 7)
  • History of dialysis or renal transplantation
  • Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
  • Severe myocardial fibrosis by MRI (≥2 late-enhancement [LE] positive left ventricular segments) (Weidemann et al. 2009)
  • History of clinical stroke
  • Pregnant or nursing
  • Presence of HIV and/or HBsAg and/or Hepatitis C infections
  • Known allergies to ERT
  • Known allergy to Gadolinium based contrast agents
  • 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: NCT01678898

Locations
United States, California
UC Davis Medical Center, MIND Institute Department of Pediatrics, Section of Genetics
Sacramento, California, United States, 95817
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, Pennsylvania
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
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
Serbia
Clinical Center of Serbia
Belgrade, Serbia
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
  More Information

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01678898     History of Changes
Other Study ID Numbers: PB-102-F01 
Study First Received: August 31, 2012
Last Updated: April 18, 2016
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on September 29, 2016