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

NCT00568698 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 29

Last updated 2019-10-02

Study results available
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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.

Conditions

  • Muscular Atrophy, Spinal

Interventions

DRUG

Hydroxyurea

DRUG

Placebo to match hydroxyurea

Sponsors & Collaborators

Principal Investigators

  • Dr Ching H. Wang · Stanford University

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
SINGLE_GROUP

Eligibility

Max Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-01-31
Primary Completion
2012-02-29
Completion
2012-02-29

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00568698 on ClinicalTrials.gov