A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients

NCT00568698 Phase 1/Phase 2 COMPLETED Results posted

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.

Details

Lead sponsor	Stanford University
Phase	Phase 1/Phase 2
Status	COMPLETED
Enrolment	29
Start date	2004-01
Completion	2012-02

Conditions

• Muscular Atrophy, Spinal

Interventions

• Hydroxyurea
• Placebo to match hydroxyurea

Primary outcomes

• Safety: Frequency of Adverse Events/Lab Abnormalities — Up to 8 years, 1 month
• Efficacy: Length of Survival (LOS) and Age of Ventilator Dependence (AVD) — Up to 8 years, 1 month

Countries

United States