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Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III

NCT00439569 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2010-09-08

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 types II or III; and to determine if the drug has an effect on SMN mRNA and protein levels.

Conditions

  • Spinal Muscular Atrophy Type II
  • Spinal Muscular Atrophy Type III

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 · National Institute of Neurological Disorders and Stroke, Program Director

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
11 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-01-31
Primary Completion
2008-08-31
Completion
2008-08-31

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 NCT00439569 on ClinicalTrials.gov