At a glance
ClinicalIndex Comparison Record- ✓Confirmed genetic diagnosis of 5q SMA
- ✓Cohort 1: SMA 2 or non-ambulatory SMA 3 with ability to sit independently for at least 3 seconds without support
- ✓Cohort 1: Age 2 to 8 years at enrollment
- ✓Cohort 2: SMA type 2 or 3 with ability to stand independently ≥2 seconds without braces or walk independently
- ✕Cohort 1: Requiring BiPAP support >12 hours per day
- ✕Spinal rod or fixation for scoliosis, or anticipated need within 6 months
- ✕Inability to reliably meet study visit requirements or cooperate with functional testing
- ✕Coexisting medical conditions contraindicating travel, testing, or study medications
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial)
In Brief
A Phase 2 clinical trial evaluating Valproic Acid and Levocarnitine and Placebo for Spinal Muscular Atrophy. Completed, enrolled 94 participants across 6 sites in 2 countries.
Detailed Summary
This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8 years of age. Cohort 2 is an open label protocol for SMA "standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes. Outcome measures will include blood chemistries, functional testing, pulmonary function testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign parameters. Six centers will enroll a total of 90 patients.
Study Details
Timeline
Interventions
VPA,sprinkle cap; Levocarnitine, syrup; dosage is by weight