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A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.

Primary Purpose

Fabry Disease

Status
Withdrawn
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Fabrazyme (agalsidase beta)
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fabry Disease focused on measuring Agalsidase beta, alpha Galactosidase A, aGAL, rh aGAL, Fabry, GL3, Fabrazyme, dialysis, hemodialysis

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Patient must provide signed, written informed consent prior to any study-related procedures being performed. Patient is between 18 and 65 years of age, inclusive. Patient has documented Fabry disease. Patient has received bi-weekly 1 mg/kg infusions of Fabrazyme for at least 6 months prior to enrollment into the study. Patient has not experienced moderate or severe infusion-associated reactions (IARs) from Fabrazyme infusions, which were also associated with a rate reduction, within 3 months prior to enrollment into the study. Patient has been receiving chronic hemodialysis for treatment of end-stage renal insufficiency for at least 3 months prior to enrollment into the study. Patient has good vascular access for hemodialysis. Patient has not and will not have any other (investigational) drug(s) infused during their hemodialysis, and is expected to have a stable concomitant medication regimen at all PK assessments. Exclusion Criteria: Patient's hemoglobin is < 9 g/100 mL at Screening/Baseline. Patient has a clinically significant organic disease or an unstable condition that, in the opinion of the Investigator, would preclude participation in the study. Patient has a medical condition, serious intercurrent illness, or extenuating circumstance that would significantly decrease study compliance. Patient has participated in a study employing an investigational drug within 30 days prior to the start of their participation in this study. Patient is unwilling to comply with the requirements of the protocol.

Sites / Locations

  • Trident Nephrology Associates

Outcomes

Primary Outcome Measures

Verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis with a low-flux membrane.

Secondary Outcome Measures

Verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis with a high-flux membrane.

Full Information

First Posted
April 7, 2006
Last Updated
February 4, 2014
Sponsor
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT00312767
Brief Title
A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.
Official Title
A Multicenter, Open-Label, Cross-Over Trial to Evaluate the Pharmacokinetics of Fabrazyme During Simultaneous Fabrazyme Infusion and Chronic Hemodialysis in Patients With Fabry Disease.
Study Type
Interventional

2. Study Status

Record Verification Date
February 2014
Overall Recruitment Status
Withdrawn
Study Start Date
April 2006 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Genzyme, a Sanofi Company

4. Oversight

5. Study Description

Brief Summary
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid (globotriaosylceramide or GL-3) levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study is designed to verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis in patients currently receiving Fabrazyme at a dose of 1.0 mg/kg every 2 weeks.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease
Keywords
Agalsidase beta, alpha Galactosidase A, aGAL, rh aGAL, Fabry, GL3, Fabrazyme, dialysis, hemodialysis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Fabrazyme (agalsidase beta)
Primary Outcome Measure Information:
Title
Verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis with a low-flux membrane.
Secondary Outcome Measure Information:
Title
Verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis with a high-flux membrane.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patient must provide signed, written informed consent prior to any study-related procedures being performed. Patient is between 18 and 65 years of age, inclusive. Patient has documented Fabry disease. Patient has received bi-weekly 1 mg/kg infusions of Fabrazyme for at least 6 months prior to enrollment into the study. Patient has not experienced moderate or severe infusion-associated reactions (IARs) from Fabrazyme infusions, which were also associated with a rate reduction, within 3 months prior to enrollment into the study. Patient has been receiving chronic hemodialysis for treatment of end-stage renal insufficiency for at least 3 months prior to enrollment into the study. Patient has good vascular access for hemodialysis. Patient has not and will not have any other (investigational) drug(s) infused during their hemodialysis, and is expected to have a stable concomitant medication regimen at all PK assessments. Exclusion Criteria: Patient's hemoglobin is < 9 g/100 mL at Screening/Baseline. Patient has a clinically significant organic disease or an unstable condition that, in the opinion of the Investigator, would preclude participation in the study. Patient has a medical condition, serious intercurrent illness, or extenuating circumstance that would significantly decrease study compliance. Patient has participated in a study employing an investigational drug within 30 days prior to the start of their participation in this study. Patient is 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:
Facility Name
Trident Nephrology Associates
City
North Charleston
State/Province
South Carolina
ZIP/Postal Code
29405
Country
United States

12. IPD Sharing Statement

Learn more about this trial

A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.

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