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Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy

NCT00374075 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 42

Last updated 2023-05-10

No results posted yet for this study

Summary

This is an open label phase I/II clinical trial to assess safety, tolerability and potential effect on SMN mRNA and protein in vivo of a compound in which preliminary evidence supports a potential effect on SMN levels in vitro.

Conditions

Interventions

DRUG

Valproic Acid

Sponsors & Collaborators

  • Families of Spinal Muscular Atrophy

    collaborator OTHER

  • Leadiant Biosciences, Inc.

    collaborator INDUSTRY

  • Abbott

    collaborator INDUSTRY

  • University of Utah

    lead OTHER

Principal Investigators

  • Kathryn J Swoboda, M.D · University of Utah/Primary Children's Medical Center

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2003-09-30
Completion
2006-02-28

Countries

  • United States

Study Locations

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Entities

Diseases
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 NCT00374075 on ClinicalTrials.gov