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CARNI-VAL Type I: Valproic acid and carnitine in infants with spinal muscular atrophy type I
2008-003915-11Phase 2Completed
- Sponsor
- University of Utah
- Start
- Jun 2009
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of valproic acid and levocarnitine in spinal muscular atrophy type 1 and spinal muscular atrophy (SMA), sponsored by University of Utah. Primary endpoints: Safety after 6 months of treatment and Time to death or dependence on >16 hours/day ventilatory support. Started Jun 2009; status: completed.
02
Drugs
valproic acidlevocarnitine
03
Conditions
spinal muscular atrophy type 1spinal muscular atrophy (SMA)
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