Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

This study has been terminated.
(The trial was terminated early due to inadequate study design.)
Sponsor:
Collaborator:
CRL/Medinet
Information provided by:
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00837824
First received: October 23, 2008
Last updated: August 11, 2009
Last verified: October 2008
  Purpose

This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.


Condition Intervention Phase
Fabry Disease
Severe Renal Disease
Biological: Fabrazyme (agalsidase beta)
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Open Label, Dose-Ranging, Multiple Dose Study of Fabrazyme® In Patients With Fabry Disease and With Severe Renal Disease

Resource links provided by NLM:


Further study details as provided by Genzyme, a Sanofi Company:

Primary Outcome Measures:
  • Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease [ Time Frame: 7 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Plasma Globotriaosylceramide (GL-3) [ Time Frame: Evaluated at Baseline, Month 3, and Final Visit ] [ Designated as safety issue: No ]

Enrollment: 20
Study Start Date: December 2002
Study Completion Date: August 2003
Estimated Primary Completion Date: August 2003 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Fabrazyme 1mg/kg every 2 weeks
Fabrazyme 1.0 mg/kg every 2 weeks
Biological: Fabrazyme (agalsidase beta)
1.0 mg/kg every 2 weeks
Other Name: r-hαGAL
Experimental: Fabrazyme 3mg/kg every 2 weeks
Fabrazyme 3.0 mg/kg every 2 weeks
Biological: Fabrazyme (agalsidase beta)
3.0 mg/kg every 2 weeks
Other Name: r-hαGAL

  Eligibility

Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • provided written informed consent prior to any study-related procedures being performed.
  • be ≥16 years old.
  • have a current diagnosis of Fabry disease (defined as abnormal α-galactosidase (α GAL) enzyme levels or Fabry genotype).
  • have one of the following clinical conditions present at enrollment: serum creatinine level greater than 3.0 mg/dL (an average of two values at least one week apart), or be currently on dialysis, or be status post kidney transplant by greater than 3 months.
  • have the ability to comply with the requirements of the protocol
  • have a negative pregnancy test, if a female patient of childbearing potential. In addition, all female patients of childbearing potential must use a medically accepted method of contraception throughout the study.

Exclusion Criteria:

  • if they did not meet the specific inclusion criteria.
  • if they had participated in a study employing an investigational drug within 30 days of the start of their participation in this trial.
  • had previously received enzyme replacement therapy (ERT) for their Fabry disease.
  • had diabetic nephropathy.
  • were pregnant or lactating.
  • were unwilling to comply with the requirements of the protocol.
  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: NCT00837824

Locations
United States, Connecticut
Farmington, Connecticut, United States
United States, Florida
Coral Springs, Florida, United States
United States, Kansas
Kansas City, Kansas, United States
United States, Massachusetts
Boston, Massachusetts, United States
United States, New York
New York, New York, United States
Rochester, New York, United States
United States, Pennsylvania
Pittsburgh, Pennsylvania, United States
United States, Texas
Houston, Texas, United States
United States, Wisconsin
Milwaukee, Wisconsin, United States
Sponsors and Collaborators
Genzyme, a Sanofi Company
CRL/Medinet
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00837824     History of Changes
Other Study ID Numbers: AGAL-022-02
Study First Received: October 23, 2008
Results First Received: December 5, 2008
Last Updated: August 11, 2009
Health Authority: United States: Food and Drug Administration

Keywords provided by Genzyme, a Sanofi Company:
Fabry Disease patients with Severe Renal Disease

Additional relevant MeSH terms:
Kidney Diseases
Fabry Disease
Urologic Diseases
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 October 19, 2014