Status:
COMPLETED
Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Fabry Disease
Eligibility:
MALE
18+ years
Brief Summary
This study will collect data needed to design a treatment trial for patients with Fabry disease using the experimental drug AT-1001. Fabry disease is an inherited metabolic disorder in which an enzyme...
Detailed Description
This protocol is designed to characterize the clinical and laboratory profile of patients with Fabry disease who have residual levels of Alpha-galactosidase (Alpha- Gal A) activity. Normally these are...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Age 18 and older males
- Patients will be included if they possess an Alpha-Gal A mutation that in previous investigations has been associated with enhancement of enzyme activity, as judged by the Principal Investigator.
- EXCLUSION CRITERIA:
- Patients whose general health prevents them from participating.
- Patients with significant disease unrelated to Fabry disease (e.g. diabetes, cancer).
- Patients who refuse to sign the informed consent form or who are unable to travel to the NIH Clinical Center.
- Patients who are currently participating in a clinical trial of small molecule or gene therapy for Fabry disease.
- Patients who are currently participating in a clinical trial for any condition other than Fabry disease.
- Patients who are judged by the Principal Investigator to be not qualified to participate.
Exclusion
Key Trial Info
Start Date :
March 28 2005
Trial Type :
OBSERVATIONAL
End Date :
March 27 2008
Estimated Enrollment :
35 Patients enrolled
Trial Details
Trial ID
NCT00106912
Start Date
March 28 2005
End Date
March 27 2008
Last Update
July 2 2017
Active Locations (1)
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1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892