CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I

Study Identifier:
25409
ClinicalTrials.gov Identifier:
NCT00661453
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
University of Utah
Study Complete

Study Details

Medical Condition
  • Spinal Muscular Atrophy Type I
Study Drug
  • Drug: Valproic Acid and Levocarnitine
Date
Apr 2008 - May 2012
Patient Requirements
Sex: Female & Male
Age: 2 Weeks - 12 Months
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Laboratory documentation of SMN mutation/deletion consistent with a genetic diagnosis of SMA
  • Clinical diagnosis of SMA type I
  • Age 2 weeks to 12 months
  • Written informed consent of parents/guardian
Exclusion Criteria
  • Any clinical or laboratory evidence of hepatic or pancreatic insufficiency.
  • Laboratory results drawn within 14 days prior to start of study drug demonstrating:
  • Liver transaminases (AST, ALT), lipase, amylase: \> 1.5 x ULN White Blood Cell Count: \< 3 Neutropenia: \<1 Platelet: \<100K Hematocrit: \<30, persisting over a 30-day period
  • Serious illness requiring systemic treatment and/or hospitalization within two weeks prior to study entry.
  • Use of medications or supplements within 30 days of study enrollment that interfere with VPA or carnitine metabolism; that increase the potential risks of VPA or carnitine; or that are hypothesized to have a beneficial effect in SMA animal models or human neuromuscular disorders, including riluzole, valproic acid, hydroxyurea, oral use of albuterol, sodium phenylbutyrate, butyrate derivatives, creatinine, growth hormone, anabolic steroids, probenecid, oral or parenteral use of corticosteroids at entry, or agents anticipated to increase or decrease muscle strength or agents with presumed histone deacetylase (HDAC) inhibition.
  • Infants who have participated in a treatment trial for SMA within 30 days of study entry or who will become enrollees in any other treatment trial during the course of this study.
  • Unwillingness to travel for study assessments.
  • Coexisting medical conditions that contradict use of VPA/carnitine or travel to and from study site.

Protocol Summary

This is a multi-center trial to test safety and evaluate early treatment intervention with valproic acid and carnitine in moderating SMA symptoms of Type I infants.

Trial Locations

Location
Status
Location
Johns Hopkins University
Baltimore, Maryland, United States, 21287
Status
N/A
Location
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
Status
N/A
Location
Duke University Medical Center
Durham, North Carolina, United States, 27710
Status
N/A
Location
Ohio State University Medical Center, Dept. of Neurology
Columbus, Ohio, United States, 43210
Status
N/A
Location
University of Utah/Primary Children's Medical Center
Salt Lake City, Utah, United States, 84132
Status
N/A
Location
University of Wisconsin Children's Hospital
Madison, Wisconsin, United States, 53792-9988
Status
N/A
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