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NCT00439218 · ClinicalTrials.gov registry record · Phase 1
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I
A Phase 1 study, sponsored by Westat.
- Terminated
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
NCT00439218: Clinical Trial Phase 1 study, sponsored by Westat.
NCT00439218 is a Phase 1 study that was terminated before completion, run by Westat. The registered enrollment target is 5 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00439218, a Phase 1 study, was terminated before completion, sponsored by Westat.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
Study Summary
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy type I; and to determine if the drug has an effect on SMN mRNA and protein levels.
Primary Outcome
Number of DLTs to determine the maximum tolerated dosage. A DLT is defined as any Grade(GR)3 or higher adverse event, GR 1 or higher cardiac arrhythmia; GR 2 or higher vomiting; GR 2 or higher liver dysfunction/failure (clinical); GR 2 elevation of amylase or lipase accompanied by clinical symptoms of pancreatitis. The following GR 2 events are classified as DLTs if evaluated to be clinically significant by the principal investigator or medical safety monitor: decrease of hemoglobin, WBCs, plate
Interventions
- DRUG sodium phenylbutyrate
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2008-01 |
| Est. Completion | 2009-05 |
| Phase | Phase 1 |
Why NCT00439218 stopped before completion
NCT00439218 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 5 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower).
The record links to 0 conditions, and to 1 intervention - of which sodium phenylbutyrate is the first listed.
NCT00439218 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00439218 about?
NCT00439218 is a clinical study titled "Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I". The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy type I; and to determine if the drug has an effect on SMN mRNA and protein levels.
What is the current status of trial NCT00439218?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2008-01. Estimated completion is 2009-05.
What interventions are being tested in trial NCT00439218?
The interventions under investigation include: sodium phenylbutyrate (DRUG).
Who is sponsoring clinical trial NCT00439218?
This trial is sponsored by Westat, which has 13 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00439218's enrollment target sits among peer trials
5 1989th of 2000 higher than 6 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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
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