At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 29 enrolled
Drug / intervention
Hydroxyurea +1 moredrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
In Brief
A Phase 2 clinical trial evaluating Hydroxyurea and Placebo to match hydroxyurea for Muscular Atrophy, Spinal. Completed, enrolled 29 participants across 1 site.
Detailed Summary
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.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMuscular Atrophy, Spinal
CountriesUnited States
Collaborators--
Timeline
Phase 2CompletedFinished
200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
Enrollment StartJan 2004
First PostedDec 2007
Primary CompletionFeb 2012
TodayJul 2026
First PostedDec 6, 2007
Enrollment StartJan 1, 2004
Primary CompletionFeb 1, 2012
TodayJul 2, 2026
Enrollment to primary: 8.1 yearsPosted 18.6 years ago
Interventions
Hydroxyureadrug
Placebo to match hydroxyureadrug