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Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

NCT00097890 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2008-03-04

No results posted yet for this study

Summary

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).

Conditions

Interventions

DRUG

Replagal (Agalsidase Alfa)

DRUG

Replagal

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    lead NIH

Study Design

Purpose
TREATMENT

Eligibility

Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-11-30
Completion
2005-12-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00097890 on ClinicalTrials.gov