Status:
COMPLETED
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
Lead Sponsor:
University of Utah
Collaborating Sponsors:
Families of Spinal Muscular Atrophy
Leadiant Biosciences, Inc.
Conditions:
Spinal Muscular Atrophy Type I
Eligibility:
All Genders
2-12 years
Phase:
PHASE1
PHASE2
Brief 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.
Detailed Description
Spinal muscular atrophy (SMA) is a genetic disorder that results in severe muscle weakness. It is one of the most common conditions causing muscle weakness in children. Patients with SMA most often de...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
April 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 1 2012
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT00661453
Start Date
April 1 2008
End Date
June 1 2012
Last Update
June 15 2015
Active Locations (8)
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1
Johns Hopkins University
Baltimore, Maryland, United States, 21287
2
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
3
Duke University Medical Center
Durham, North Carolina, United States, 27710
4
Ohio State University Medical Center, Dept. of Neurology
Columbus, Ohio, United States, 43210