NCT00481013

Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy

Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 33 (actual)
• Sponsor: University of Utah · Academic / Other
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this proposal is to determine whether oral VPA is effective in treating SMA in adult patients.

Detailed description

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.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	Valproic Acid (VPA)	Drug: Valproic Acid and Levocarnitine; capsules
DRUG	Placebo	For six months, pts are randomized into placebo or treatment. After 6 months, all pts are on treatment

Timeline

• Start date: 2007-07-01
• Primary completion: 2009-12-01
• Completion: 2010-11-01
• First posted: 2007-06-01
• Last updated: 2016-12-06

Locations

1 site across 1 country: United States