A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients

Study Identifier:
SU-11012007-783
ClinicalTrials.gov Identifier:
NCT00568698
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Stanford University
Study Complete

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: Hydroxyurea
  • Drug: Placebo to match hydroxyurea
Date
Jan 2004 - Feb 2012
Patient Requirements
Sex: Female & Male
Age: N/A - 2 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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
  • 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

Protocol 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.

Trial Locations

Location
Status
Location
Stanford University School of Medicine
Stanford, California, United States, 94305
Status
N/A

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