Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I
Public ClinicalTrials.gov record NCT00439218. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Phase I/IIa Clinical Trial of Sodium Phenylbutyrate in Pediatric Subjects With Type I Spinal Muscular Atrophy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT00439218
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 5 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 Months to 48 Months
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 2008 (Type not available)
- Primary completion
- May 2009 (Actual)
- Completion
- May 2009 (Actual)
- Last update posted
- Nov 9, 2010
2008 – 2009
United States locations
- U.S. sites
- 5
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Stanford University Medical Center, 300 Pasteur Drive, Room A343 | Stanford | California | 94305-5235 | — |
| Children's Hospital, Boston, 300 Longwood Avenue, Fegan 11 | Boston | Massachusetts | 02115 | — |
| Columbia University, 180 Fort Washington Avenue, 5th Floor | New York | New York | 10032 | — |
| The Children's Hospital of Philadelphia, Clinical Trials Office, A-230, 34th St. and Civic Center Boulevard | Philadelphia | Pennsylvania | 19104-4399 | — |
| University of Texas Southwestern Medical Center at Dallas, Division of Pediatric Neurology, Children's Medical Center of Dallas, Ambulatory Care Pavilion, 2350 Stemmons Freeway, Suite #5074 | Dallas | Texas | 75207 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00439218, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Nov 9, 2010 · Synced Oct 22, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT00439218 live on ClinicalTrials.gov.