Phase 2
N=27
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
Muscular Atrophy, Spinal
Bottom Line
View on ClinicalTrials.gov: NCT00568802 ↗Enrolled (actual)
27
Serious AEs
—
Results posted
Oct 2019
Primary outcome: Primary: Efficacy: Functional Motor Testing, Including Gross Motor Function Measure (GMFM) and Timed Motor Tests
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Hydroxyurea (Drug); Placebo to match hydroxyurea (Drug)
- Age
- Pediatric · 1+ yrs
- Sex
- All
- Sponsor
- Stanford University
- Primary completion
- Mar 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Efficacy: Functional Motor Testing, Including Gross Motor Function Measure (GMFM) and Timed Motor Tests |
— | — |
| PRIMARY Safety: Frequency of Adverse Events/Lab Abnormalities |
— | — |
| SECONDARY Pulmonary Function Testing |
— | — |
| SECONDARY Motor Unit Number Estimation (MUNE) |
— | — |
| SECONDARY Biomarker Assays: SMN Protein and SMN mRNA |
— | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria:1. Laboratory confirmation of a homozygous deletion or mutation of the SMN1 gene 2. (Type II) Can sit independently but cannot walk without support by the age of 16 months and never achieve independent walking thereafter; OR (Type III) Can walk independently within the first 2 years of life, but showing rapid progression of weakness resulting in the loss of independent ambulation by 6 years of age 3. Patient is older than 16 months and younger than 8 years old at the time of enrollment
Exclusion Criteria:1. Known hematological disorders, other systemic disorders, or severe birth asphyxia 2. Participation in SMA clinical trials for other experimental drugs 3. Requiring continuous respiratory support before the initiation of HU treatment
Data sourced from ClinicalTrials.gov (NCT00568802). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.