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NCT00106912 · ClinicalTrials.gov registry record
Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity
A clinical trial, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- Completed
- Registry status
- 35
- Enrollment target
NCT00106912: Completed study, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
NCT00106912 is a clinical trial that has completed, run by National Institute of Neurological Disorders and Stroke (NINDS). The registered enrollment target is 35 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00106912 has completed, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- COMPLETED
- Registry status
- 35 participants
- Enrollment target
Study 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 called alpha-galactosidase A, which normally breaks down fatty substances called glycolipids, is missing or does not function properly. As a result, glycolipids accumulate in various tissues, causing liver, kidney, nerves, skin, muscle and blood vessel problems. No treatment is given in this survey study. Males 18 years of age and older with Fabry disease who have certain genetic mutations associated with enhancement of alpha-galactosidase A activity may be eligible for this study. Participants undergo the following tests and procedures over 5 days: Day 1 Medical history and physical examination, blood tests, electrocardiogram (EKG), routine urinalysis, measurements of height, weight, and vital signs (blood pressure, heart rate, breathing rate, and temperature). Day 2 Blood tests, 24-hour urine collection, vital signs and sweat test. The sweat test (also called QSART, or quantitative sudomotor axon reflex test) measures the amount of sweat in a particular area of skin. A small amount of medication called acetylcholine is put on an area of the skin and a small electric current is applied to stimulate the sweat glands. Day 3 Blood tests, 24-hour urine collection, vital signs, and skin biopsy. For the skin biopsy, a small area of skin is numbed and a punch device is used to remove a 3-mm (1/8-inch) layer of skin for microscopic examination. Day 4 Blood tests, 24-hour urine collection, vital signs, and QSART. Day 5 Blood tests and vital signs. In addition to the above, patients are scheduled at some point in the 5-day study for an eye examination, brain magnetic resonance angiogram (MRA), and a heart examination and echocardiogram. MRA uses a strong magnetic field and radio waves to provide images of the blood vessels in the head and neck. It can detect abnorma
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 35 participants |
| Start Date | 2005-03-28 |
| Est. Completion | 2008-03-27 |
What the finished NCT00106912 record still lists
NCT00106912 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 35 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT00106912 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00106912 about?
NCT00106912 is a clinical study titled "Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity". 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 called alpha-galactosidase A, which normally breaks down fatty substances called glycolipids, is mi...
What is the current status of trial NCT00106912?
This trial is currently completed. The enrollment target is 35 participants. The study started on 2005-03-28. Estimated completion is 2008-03-27.
Who is sponsoring clinical trial NCT00106912?
This trial is sponsored by National Institute of Neurological Disorders and Stroke (NINDS), which has 567 total clinical trials registered on ClinicalTrials.gov.
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