Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

This study has been completed.
Sponsor:
Information provided by:
National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT00097890
First received: November 30, 2004
Last updated: March 3, 2008
Last verified: December 2005
  Purpose

This study will determine the safety and effectiveness of the drug Replagal for treating people with Fabry disease, an inherited metabolic disorder. In this disease, an enzyme called alpha-galactosidase A, which normally breaks down a lipid (fatty substance) known as ceramidetrihexoside, is missing or does not function properly. As a result, the lipid accumulates in the body, causing problems with the kidneys, heart, nerves, and blood vessels. This study will examine whether replacing the missing alpha-galactosidase A with a genetically engineered form of the enzyme called Replagal can reverse the illness.

Patients with Fabry disease who are 18 years of age or older and have completed 10 weeks of Replagal therapy as participants in protocol TKT027 may be eligible for this 6-month study extension.

Participants undergo the following tests and procedures:

  • Intravenous (IV) infusions of Replagal every other week over 25 weeks for a total of 13 infusions, with close monitoring during and after the infusions.
  • Brief safety evaluations at the time of each infusion, including a check of vital signs (blood pressure, pulse, breathing rate, temperature), review of any side effects, and review of medications.
  • Comprehensive evaluations at baseline (before starting Replagal therapy), after 13 and 25 weeks of therapy, and 30 days after completing therapy. These include a medical history and physical examination, symptoms and pain questionnaire, blood and urine tests, check of vital signs, electrocardiogram (EKG), 2-hour Holter monitor, and sweat test (QSART).

Condition Intervention Phase
Fabry Disease
Drug: Replagal (Agalsidase Alfa)
Drug: Replagal
Phase 4

Study Type: Interventional
Study Design: Primary Purpose: Treatment
Official Title: An Open Label Six-Month Maintenance Clinical Trial of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease Who Have Completed TKT027

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Estimated Enrollment: 25
Study Start Date: November 2004
Estimated Study Completion Date: December 2005
Detailed Description:

Objectives: To evaluate the safety of the current standard Replagal treatment regimen of 0.2 mg/kg given intravenously every two weeks over an additional 6 months in patients who have completed 10 weeks of Replagal therapy in the TKT027 study.

Study Population: Hemizygous males with Fabry disease who are 18 years of age or older who have completed 10 weeks of Replagal therapy in TKT027.

Design: This is an open-label, multi-center study that will assess the safety of 0.2 mg/kg every two weeks of enzyme replacement therapy with Replagal.

  Eligibility

Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

INCLUSION CRITERIA:

Subject must have completed 10 weeks of infusions in Study TKT027 with intent to complete participation in the study.

Subject must have adequate general health (as determined by the investigators) to undergo the specified phlebotomy regimen and protocol related procedures.

Subject must consent to participate in the protocol and must have voluntarily signed an Institutional Review Board/Independent Ethics Committee (IRB/IEC) approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient.

EXCLUSION CRITERIA:

Subject is unable to understand the nature, scope, and possible consequences of the study.

Subject is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the investigator or the medical monitor.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00097890

Locations
United States, Maryland
National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
  More Information

Publications:
ClinicalTrials.gov Identifier: NCT00097890     History of Changes
Other Study ID Numbers: 050046, 05-N-0046
Study First Received: November 30, 2004
Last Updated: March 3, 2008
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Lysosomes
Storage
Glycolipid
Fabry Disease
Stroke

Additional relevant MeSH terms:
Fabry Disease
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
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on August 26, 2014