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Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

NCT00439218 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2010-11-09

Study results available
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Summary

The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy type I; and to determine if the drug has an effect on SMN mRNA and protein levels.

Conditions

  • Spinal Muscular Atrophy Type I

Interventions

DRUG

sodium phenylbutyrate

500 mg/kg/day, depending upon tolerability subsequent dosages may increase to 675, 900, or 1200 mg/kg/day to identify maximum tolerated dose (MTD) and then an additional 6 participants will enroll at the MTD.

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • Westat

    lead OTHER

Principal Investigators

  • René Gonin, PhD · Mathematical Statistician, Westat

  • Peter R Gilbert, ScM · The National Institute of Neurological Disorders and Stroke

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Months
Max Age
48 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-01-31
Primary Completion
2009-05-31
Completion
2009-05-31

Countries

  • United States

Study Locations

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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 NCT00439218 on ClinicalTrials.gov