NCT00374075

Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy

In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 42 (planned)
• Sponsor: University of Utah · Academic / Other
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

This is an open label phase I/II clinical trial to assess safety, tolerability and potential effect on SMN mRNA and protein in vivo of a compound in which preliminary evidence supports a potential effect on SMN levels in vitro.

Detailed description

This is an open label phase I/II trial of valproic acid in 40 SMA subjects \> 2 years of age with severe, intermediate, and mild phenotypes. Primary outcome measures includes laboratory and physical examination assessments to monitor effects on liver, hematologic, metabolic and nutritional status. Secondary outcomes includes measures of gross motor function; electrophysiologic measures of denervation; DEXA estimates of body composition, bone mineral density and content; measures of pulmonary function; and quantitative SMN mRNA and protein levels in blood cells. Subjects will need 2-3 baseline visits over a 3 -6 month period prior to enrollment. Follow-up visits will be scheduled at 3, 6 and 12 months on treatment.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	Valproic Acid

Timeline

• Start date: 2003-09-01
• Completion: 2006-02-01
• First posted: 2006-09-08
• Last updated: 2023-05-10

Locations

1 site across 1 country: United States