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NCT00227266 · ClinicalTrials.gov registry record · Phase 2
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
A Phase 2 study, sponsored by University of Utah.
- Completed
- Registry status
- Phase 2
- Development phase
- 94
- Enrollment target
NCT00227266 is a Phase 2 study that has completed, run by University of Utah. The registered enrollment target is 94 participants.
The verdict
NCT00227266, a Phase 2 study, has completed, sponsored by University of Utah.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 94 participants
- Enrollment target
Study Summary
This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8 years of age. Cohort 2 is an open label protocol for SMA "standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes. Outcome measures will include blood chemistries, functional testing, pulmonary function testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign parameters. Six centers will enroll a total of 90 patients.
Interventions
- DRUG Placebo
- DRUG Valproic Acid and Levocarnitine
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 94 participants |
| Start Date | 2005-09 |
| Est. Completion | 2007-11 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00227266
The ClinicalTrials.gov registry entry for NCT00227266 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 94 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is University of Utah, which has 818 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT00227266 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00227266 about?
NCT00227266 is a clinical study titled "Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy". This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8...
What is the current status of trial NCT00227266?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 94 participants. The study started on 2005-09. Estimated completion is 2007-11.
What interventions are being tested in trial NCT00227266?
The interventions under investigation include: Placebo (DRUG), Valproic Acid and Levocarnitine (DRUG).
Who is sponsoring clinical trial NCT00227266?
This trial is sponsored by University of Utah, which has 818 total clinical trials registered on ClinicalTrials.gov.
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