Text Size

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

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT01678898
First received: August 31, 2012
Last updated: September 4, 2012
Last verified: September 2012
History of Changes
  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:

U.S. FDA Resources

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


Estimated Enrollment: 18
Study Start Date: October 2012
Estimated Study Completion Date: July 2013
Estimated Primary Completion Date: March 2013 (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
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
  • Chronic kidney disease - stages 1 or 2 (CKD1 or 2) (Appendix 7) with proteinuria > 300 mg/g protein-to-creatinine ratio or equivalent, measured in a Spot urine sample, demonstrated in 2 separate samples (1 sample at screening visit and the other from historical data)
  • 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01678898

Sponsors and Collaborators
Protalix
Investigators
Study Director: Einat Almon, PhD Protalix
  More Information

No publications provided

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: September 4, 2012
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
 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 May 23, 2013