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Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

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.
ClinicalTrials.gov Identifier: NCT00837824
Recruitment Status : Terminated (The trial was terminated early due to inadequate study design.)
First Posted : February 5, 2009
Results First Posted : February 5, 2009
Last Update Posted : April 7, 2015
Information provided by:

Brief Summary:
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 or disease Intervention/treatment Phase
Fabry Disease Chronic Kidney Disease, Stage IV (Severe) Biological: Fabrazyme (agalsidase beta) Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (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
Study Start Date : December 2002
Primary Completion Date : August 2003
Study Completion Date : August 2003

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Kidney Diseases
U.S. FDA Resources

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease [ Time Frame: 7 months ]
    The trial was terminated early due to inadequate study design. During the study period of 7 months, only 1 patient had a clinical event, a stroke, in the Fabrazyme 1 mg/kg treatment arm. The time to event was determined from first dose of Fabrazyme to the date of event.

Secondary Outcome Measures :
  1. Plasma Globotriaosylceramide (GL-3) [ Time Frame: Evaluated at Baseline, Month 3, and Final Visit ]
    This outcome measure evaluated the mean plasma GL-3 values for all patients to see if it decreased while on Fabrazyme. Normal plasma GL-3 level is defined as ≤ 7.03 µg/mL.

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

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): NCT00837824

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
Study Director: Medical Monitor Genzyme, a Sanofi Company

Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00837824     History of Changes
Other Study ID Numbers: AGAL-022-02
First Posted: February 5, 2009    Key Record Dates
Results First Posted: February 5, 2009
Last Update Posted: April 7, 2015
Last Verified: March 2015

Keywords provided by Sanofi:
Fabry Disease patients with Severe Renal Disease

Additional relevant MeSH terms:
Kidney Diseases
Renal Insufficiency, Chronic
Fabry Disease
Urologic Diseases
Renal Insufficiency
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
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders