A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients

Study Identifier:
SU-11012007-781
ClinicalTrials.gov Identifier:
NCT00568802
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 - Mar 2010
Patient Requirements
Sex: Female & Male
Age: 1 Year - 10 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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
  • 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

Protocol 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.

Trial Locations

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

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