Education and Training
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
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.
Stanford is currently not accepting patients for this trial.
Intervention(s):
- drug: Hydroxyurea
- drug: Placebo to match hydroxyurea
Eligibility
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
Ages Eligible for Study
N/A - 2 Years
Genders Eligible for Study
All
Not currently accepting new patients for this trial
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Virginia Wedell
6504987658
Not Recruiting