Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Completed Phase 2 study, sponsored by University of Utah.
NCT00227266 is a Phase 2 study that has completed, run by University of Utah. The registered enrollment target is 94 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (29% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Participants will have labs drawn regularly to maintain appropriate dosing and monitor liver function
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 |
What the finished NCT00227266 record still lists
NCT00227266 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 94 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (29% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT00227266 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00227266's enrollment target sits among peer trials
94 764th of 2000 higher than 1,235 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT03747146 · 94 participants · NA
Enhanced Recovery After Surgery (ERAS) Total Knee Replacement (TKR) With a Transitional Pain Service
- NCT04061421 · 94 participants · Phase 1
Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes Overlap Syndromes (ABNL-MARRO)
- NCT04205968 · 94 participants · Phase 2
Ramucirumab and Paclitaxel or FOLFIRI in Advanced Small Bowel Cancers
- NCT04464759 · 94 participants · Phase 1
A Study of Nivolumab and Hydroxychloroquine or Nivolumab/Ipilimumab and Hydroxychloroquine in Advanced Melanoma
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia