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Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Primary Purpose

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

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Fabrazyme (agalsidase beta)
Fabrazyme (agalsidase beta)
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fabry Disease focused on measuring Fabry Disease patients with Severe Renal Disease

Eligibility Criteria

16 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Fabrazyme 1mg/kg every 2 weeks

Fabrazyme 3mg/kg every 2 weeks

Arm Description

Fabrazyme 1.0 mg/kg every 2 weeks

Fabrazyme 3.0 mg/kg every 2 weeks

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

Full Information

First Posted
October 23, 2008
Last Updated
March 19, 2015
Sponsor
Genzyme, a Sanofi Company
Collaborators
CRL/Medinet
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1. Study Identification

Unique Protocol Identification Number
NCT00837824
Brief Title
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
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 Type
Interventional

2. Study Status

Record Verification Date
March 2015
Overall Recruitment Status
Terminated
Why Stopped
The trial was terminated early due to inadequate study design.
Study Start Date
December 2002 (undefined)
Primary Completion Date
August 2003 (Actual)
Study Completion Date
August 2003 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Genzyme, a Sanofi Company
Collaborators
CRL/Medinet

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Keywords
Fabry Disease patients with Severe Renal Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Fabrazyme 1mg/kg every 2 weeks
Arm Type
Experimental
Arm Description
Fabrazyme 1.0 mg/kg every 2 weeks
Arm Title
Fabrazyme 3mg/kg every 2 weeks
Arm Type
Experimental
Arm Description
Fabrazyme 3.0 mg/kg every 2 weeks
Intervention Type
Biological
Intervention Name(s)
Fabrazyme (agalsidase beta)
Other Intervention Name(s)
r-hαGAL
Intervention Description
1.0 mg/kg every 2 weeks
Intervention Type
Biological
Intervention Name(s)
Fabrazyme (agalsidase beta)
Other Intervention Name(s)
r-hαGAL
Intervention Description
3.0 mg/kg every 2 weeks
Primary Outcome Measure Information:
Title
Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease
Description
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 Outcome Measure Information:
Title
Plasma Globotriaosylceramide (GL-3)
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme, a Sanofi Company
Official's Role
Study Director
Facility Information:
City
Farmington
State/Province
Connecticut
Country
United States
City
Coral Springs
State/Province
Florida
Country
United States
City
Kansas City
State/Province
Kansas
Country
United States
City
Boston
State/Province
Massachusetts
Country
United States
City
New York
State/Province
New York
Country
United States
City
Rochester
State/Province
New York
Country
United States
City
Pittsburgh
State/Province
Pennsylvania
Country
United States
City
Houston
State/Province
Texas
Country
United States
City
Milwaukee
State/Province
Wisconsin
Country
United States

12. IPD Sharing Statement

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Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

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