Trial of L-Carnitine and Valproic Acid in Children with Spinal Muscular Atrophy
Author Information
Author(s): Kissel John T., Scott Charles B., Reyna Sandra P., Crawford Thomas O., Simard Louise R., Krosschell Kristin J., Acsadi Gyula, Elsheik Bakri, Schroth Mary K., D'Anjou Guy, LaSalle Bernard, Prior Thomas W., Sorenson Susan, Maczulski Jo Anne, Bromberg Mark B., Chan Gary M., Swoboda Kathryn J.
Primary Institution: The Ohio State University
Hypothesis
Does the combination of L-Carnitine and Valproic Acid improve motor function in ambulatory children with Spinal Muscular Atrophy?
Conclusion
The study found that Valproic Acid and L-Carnitine do not improve strength or function in children with Spinal Muscular Atrophy.
Supporting Evidence
- Twenty-eight subjects completed the study.
- Adverse events occurred in 85% of subjects but were usually mild and transient.
- Weight gain of 20% above body weight occurred in 17% of subjects.
- Some pulmonary function measures showed improvement at one year.
- CMAP significantly improved suggesting a modest biologic effect.
Takeaway
The study tested a medicine to see if it could help kids with a muscle disease, but it didn't make them stronger or help them move better.
Methodology
The study involved 33 children with genetically proven type 3 SMA who were treated with Valproic Acid and L-Carnitine for 12 months, with assessments at baseline, 3, 6, and 12 months.
Potential Biases
Potential bias due to the open-label design and lack of a control group.
Limitations
The study was open-label and single-armed, which may limit the generalizability of the findings.
Participant Demographics
Participants were aged 3-17 years, predominantly male (66.7%), and mostly non-Hispanic white (90.9%).
Digital Object Identifier (DOI)
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