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

NCT00312767 | Withdrawn Phase 4

• 1 other identifier: AGAL03505
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Fabry Disease

Keywords

Agalsidase beta; alpha Galactosidase A; aGAL; rh aGAL; Fabry; GL3; Fabrazyme; dialysis; hemodialysis

Outcome Measures

Primary Outcomes (1)

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

Secondary Outcomes (1)

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

Interventions

Fabrazyme (agalsidase beta) DRUG

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (1)

Trident Nephrology Associates

North Charleston, South Carolina, 29405, United States

Location

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase beta

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Medical Monitor

  Genzyme, a Sanofi Company

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: April 7, 2006
• First Posted: April 11, 2006
• Study Start: April 1, 2006
• Last Updated: February 5, 2014

Record last verified: 2014-02

Locations

US (1)