Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

A Phase 2, Randomized, Open Label, Dose-Ranging, Multiple Dose Study of Fabrazyme® In Patients With Fabry Disease and With Severe Renal Disease

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.

Study Overview

• Status: Terminated
• Conditions: Fabry Disease; Chronic Kidney Disease, Stage IV (Severe)
• Intervention / Treatment: Biological: Fabrazyme (agalsidase beta); Biological: Fabrazyme (agalsidase beta)

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Connecticut
    • Farmington, Connecticut, United States
  • Florida
    • Coral Springs, Florida, United States
  • Kansas
    • Kansas City, Kansas, United States
  • Massachusetts
    • Boston, Massachusetts, United States
  • New York
    • New York, New York, United States
    • Rochester, New York, United States
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States
  • Texas
    • Houston, Texas, United States
  • Wisconsin
    • Milwaukee, Wisconsin, United States

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / 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 Names: r-hαGAL; Biological: Fabrazyme (agalsidase beta); 3.0 mg/kg every 2 weeks; Other Names: r-hαGAL
Experimental: Fabrazyme 3mg/kg every 2 weeks; Fabrazyme 3.0 mg/kg every 2 weeks	Biological: Fabrazyme (agalsidase beta); 1.0 mg/kg every 2 weeks; Other Names: r-hαGAL; Biological: Fabrazyme (agalsidase beta); 3.0 mg/kg every 2 weeks; Other Names: r-hαGAL

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease	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.	7 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Plasma Globotriaosylceramide (GL-3)	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.	Evaluated at Baseline, Month 3, and Final Visit

Collaborators and Investigators

• Sponsor: Genzyme, a Sanofi Company
• Collaborators: CRL/Medinet

Study record dates

Study Major Dates

• Study Start: December 1, 2002
• Primary Completion (Actual): August 1, 2003
• Study Completion (Actual): August 1, 2003

Study Registration Dates

• First Submitted: October 23, 2008
• First Submitted That Met QC Criteria: February 4, 2009
• First Posted (Estimate): February 5, 2009

Study Record Updates

• Last Update Posted (Estimate): April 7, 2015
• Last Update Submitted That Met QC Criteria: March 19, 2015
• Last Verified: March 1, 2015

More Information

Terms related to this study

• Keywords: Fabry Disease patients with Severe Renal Disease
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Metabolic Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Urologic Diseases; Renal Insufficiency; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Cerebral Small Vessel Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Kidney Diseases; Renal Insufficiency, Chronic; Fabry Disease
• Other Study ID Numbers: AGAL-022-02