A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
Completed · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Muscular Atrophy, Spinal
In brief
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Type I Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Type I SMA; and to detect the clinical efficacy of HU treatment in children with Type I SMA.
Key facts
- Study ID
- NCT00568698
- Run by
- Stanford University
- People needed
- 29
- Starts
- 2004-01-01
- Expected to finish
- 2012-02-01
- Last updated by the study team
- 2019-10-02
Who can join
Age: any, up to 2. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Stanford University School of Medicine — Stanford, California, United States
Full record on ClinicalTrials.gov
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