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NCT00515398 · ClinicalTrials.gov registry record
A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
A clinical trial, sponsored by Amicus Therapeutics.
- Completed
- Registry status
- 30
- Enrollment target
NCT00515398: Completed study, sponsored by Amicus Therapeutics.
NCT00515398 is a clinical trial that has completed, run by Amicus Therapeutics. The registered enrollment target is 30 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00515398 has completed, sponsored by Amicus Therapeutics.
- COMPLETED
- Registry status
- 30 participants
- Enrollment target
Study Summary
The purpose of this study is to see how molecules called pharmacological chaperones affect the cells of patients with Pompe disease. The study will last 1 or 2 visits which will include a blood collection, urine collection, and two skin biopsies. Information will also be collected from the medical records about disease history and diagnosis. Patients will not receive any study medication.
Interventions
- OTHER Observation
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2007-08 |
| Est. Completion | 2008-01 |
What the finished NCT00515398 record still lists
NCT00515398 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 30 participants, a relatively small participant target.
The record links to 0 conditions, and to 1 intervention - of which Observation is the first listed.
NCT00515398 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00515398 about?
NCT00515398 is a clinical study titled "A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease". The purpose of this study is to see how molecules called pharmacological chaperones affect the cells of patients with Pompe disease. The study will last 1 or 2 visits which will include a blood collection, urine collection, and two skin biopsies. Information will also be collected from the medical r...
What is the current status of trial NCT00515398?
This trial is currently completed. The enrollment target is 30 participants. The study started on 2007-08. Estimated completion is 2008-01.
What interventions are being tested in trial NCT00515398?
The interventions under investigation include: Observation (OTHER).
Who is sponsoring clinical trial NCT00515398?
This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.
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