Phase 2
Completed N=29
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
Muscular Atrophy, Spinal
Source: ClinicalTrials.gov NCT00568698 ↗
Enrolled (actual)
29
Serious AEs
—
Results posted
Oct 2019
Primary outcomePrimary: Safety: Frequency of Adverse Events/Lab Abnormalities
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety: Frequency of Adverse Events/Lab Abnormalities |
— | — |
| PRIMARY Efficacy: Length of Survival (LOS) and Age of Ventilator Dependence (AVD) |
— | — |
| SECONDARY Motor Unit Number Estimation (MUNE) |
— | — |
| SECONDARY Biomarker Assays: SMN Protein and SMN mRNA |
— | — |
Eligibility Criteria
Inclusion Criteria:1. Laboratory confirmation of a homozygous deletion or mutation of the SMN1 gene 2. Clinical Diagnosis of Type I SMA (never achieved independent sitting) 3. Onset of disease before the age of 6 months 4. Enrollment in study within 6 months of diagnosis Exclusion Criteria:1. Known hematological disorders, such as chronic anemia (defined as platelet count less than 100,000/mm^3) in two contiguous measures in two weeks 2. Severe systemic disorders such as congenital heart disease, other major birth defects involving internal organs, or severe birth asphyxia 3. Participation in SMA clinical trials for other experimental drugs 4. Requiring continuous respiratory support before the initiation of HU treatment
Data sourced from ClinicalTrials.gov (NCT00568698). 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. Informational only — not medical advice.