Spinal Muscular Atrophy Clinical Trial
Official title:
Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study
The primary objective of this proposal is to determine whether oral VPA is effective in treating SMA in adult patients.
Participation in this study entails six visits and seven to eight blood draws over 13
months. Each visit entails a stay of two days and one night at the General Clinical Research
Center (GCRC).
Subjects who live within driving distance will be allowed to participate in the study
without an overnight stay through two consecutive outpatient visits. All subjects will be
evaluated at two screening visits 2-4 weeks apart to determine eligibility for
participation. Eligible subjects will be randomized to receive VPA or placebo for the first
six months. At the six-month visit, patients will be evaluated and crossed over to the other
regimen.
;
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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