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

February 4, 2014 updated by: Genzyme, a Sanofi Company

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.

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • South Carolina
      • North Charleston, South Carolina, United States, 29405
        • Trident Nephrology Associates

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 63 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Crossover Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genzyme, a Sanofi Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2006

Study Registration Dates

First Submitted

April 7, 2006

First Submitted That Met QC Criteria

April 7, 2006

First Posted (Estimate)

April 11, 2006

Study Record Updates

Last Update Posted (Estimate)

February 5, 2014

Last Update Submitted That Met QC Criteria

February 4, 2014

Last Verified

February 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AGAL03505

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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