Education and Training
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
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.
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. (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
Ages Eligible for Study
1 Year - 10 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