A clinical trial entitled “Phase I/II Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI-VAL Type I)” is currently recruiting type 1 SMA patients up to 9 months old. According to study description on ClinicalTrials.gov, investigators aim “to test safety and evaluate early treatment intervention with valproic acid and carnitine in moderating SMA symptoms of Type I infants.” The detailed description goes on to say:
Valproic acid (VPA) is a medicine that has been used for many years to treat patients with epilepsy. Recent research suggests that VPA may be able to upregulate expression of a backup copy of the SMN gene in SMA patient cell lines. In addition, some preliminary data suggests it may prolong survival in animal models of SMA. Because VPA can deplete carnitine in children with SMA Type I, carnitine is added to help prevent possible toxicity.
In this multi-center trial, we will evaluate the effects of VPA/carnitine on infants with SMA type I. A variety of outcome measures, including assessment of safety, will be performed at each study visit to follow the course of the disease. The protocol includes two baseline visits over a period of two weeks, two clinical assessments on medication at 3 and 6 months, and then 6 months additional followup via telephone. Total duration of the study will be approximately 12 months.
This is an international, multi-center trial with study sites at:
- Johns Hopkins University in Baltimore Maryland
- Children’s Hospital of Michigan in Detroit
- Ohio State University Medical Center in Columbus
- University of Utah/Primary Children’s Medical Center in Salt Lake City
- University of Wisconsin Children’s Hospital in Madison
- Hospital Sainte-Justine in Montreal, Quebec, Canada
- Klinikum der Universität zu Köln in Cologne, Germany
For more details about the clinical trial, including eligibility criteria and contact information, click here. For more information about clinical trials in general, click here.
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