Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Phase 2TerminatedNCT00837824

Genzyme, a Sanofi Company20 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Fabry Disease Chronic Kidney Disease, Stage IV (Severe)

Interventions

Fabrazyme (agalsidase beta)BIOLOGICAL

1.0 mg/kg every 2 weeks

Fabrazyme (agalsidase beta)BIOLOGICAL

3.0 mg/kg every 2 weeks

Eligibility

Sex: ALLMin age: 16 Years

Medical Language ↔ Plain English

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.

Locations (9)

Unnamed facility

Farmington, Connecticut, United States

Unnamed facility

Coral Springs, Florida, United States

Unnamed facility

Kansas City, Kansas, United States

Unnamed facility

Boston, Massachusetts, United States

Outcomes

Primary Outcomes

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.

Time frame: 7 months

Secondary Outcomes

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.

Time frame: Evaluated at Baseline, Month 3, and Final Visit

Data from ClinicalTrials.gov

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