Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Study Overview
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Brief Summary
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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.

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

Conditions
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Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)

Intervention / Treatment
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* Biological: Fabrazyme (agalsidase beta)
* Biological: Fabrazyme (agalsidase beta)


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 16 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Fabrazyme 1mg/kg every 2 weeks<br>Fabrazyme 1.0 mg/kg every 2 weeks | Biological: Fabrazyme (agalsidase beta)<br>* 1.0 mg/kg every 2 weeks<br>* Other names: r-hαGAL;|
| Experimental: Fabrazyme 3mg/kg every 2 weeks<br>Fabrazyme 3.0 mg/kg every 2 weeks | Biological: Fabrazyme (agalsidase beta)<br>* 3.0 mg/kg every 2 weeks<br>* Other names: r-hαGAL;|

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Fabry Disease patients with Severe Renal Disease