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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
33
Drug: Valproic Acid and Levocarnitine; capsules
For six months, pts are randomized into placebo or treatment. After 6 months, all pts are on treatment
Ohio State University Medical Center, Dept. of Neurology
Columbus, Ohio, United States
The primary outcome for the study is change in muscle strength from baseline to six months in muscle strength as assessed by MVICT using a fixed testing system.
Time frame: 13 months
Change in SMAFRS
Time frame: 13 months
Change in strength assessed by hand-held dynamometer
Time frame: 13 months
Change in MUNE and CMAP
Time frame: 13 months
SMN2 copy number
Time frame: 13 months
Change in PFTs, including forced vital capacity (FVC) and negative inspiratory force (NIF)
Time frame: 13 months
Change in lean body mass through DEXA scanning
Time frame: 13 months
Change in distance walked in 6 minutes
Time frame: 13 months
Change in time to climb four standard stairs
Time frame: 13 months
Change in health-related QOL assessed through the modified sickness impact profile (SIP)
Time frame: 13 months
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