A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III 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 Types II and III Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Types II and III SMA; and to detect the clinical efficacy of HU treatment in children with Types II and III SMA.

Key facts

Study ID
NCT00568802
Run by
Stanford University
People needed
27
Starts
2004-01-01
Expected to finish
2010-03-01
Last updated by the study team
2019-10-02

Who can join

Age: 1 and older, up to 10. Sex: any. Healthy volunteers: not accepted.

Where it is running

Full record on ClinicalTrials.gov

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