Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Phase 2

Terminated

• Conditions: Fabry Disease; Chronic Kidney Disease, Stage IV (Severe)
• Interventions: Biological: Fabrazyme (agalsidase beta)

• Registration Number: NCT00837824
• Lead Sponsor: Genzyme, a Sanofi Company

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2002-12
• Primary Completion: 2003-08
• Study Completion: 2003-08
• Study First Submitted: 2008-10-23
• Results First Submitted: 2008-12-05
• Study First Submitted QC: 2009-02-04
• Results First Submitted QC: 2009-02-04
• Study First Posted: 2009-02-05
• Results First Posted: 2009-02-05
• Status Verified: 2015-03
• Last Update Submitted: 2015-03-19
• Last Update Posted: 2015-04-07

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 20

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Fabrazyme 1mg/kg every 2 weeks	Fabrazyme (agalsidase beta)	Fabrazyme 1.0 mg/kg every 2 weeks
Fabrazyme 3mg/kg every 2 weeks	Fabrazyme (agalsidase beta)	Fabrazyme 3.0 mg/kg every 2 weeks

Primary Outcome Measures

Name	Time	Method
Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease	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

Name	Time	Method
Plasma Globotriaosylceramide (GL-3)	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.