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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief 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 Summary: 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.
Sponsor: University of Utah

Current Primary Outcome: To assess safety and tolerability of VPA in SMA patients greater than 2 years of age

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • To look for a potential in vivo effect of VPA on SMN mRNA in patient blood cells at routinely used clinical doses
  • Measures of gross motor function
  • Electrophysiologic measures of denervation
  • DEXA estimates of body composition, bone mineral density and content
  • Measures of pulmonary function


Original Secondary Outcome: To look for a potential in vivo effect of VPA on SMN mRNA and protein levels in patient white blood cells, monocytes and platelets at routinely used clinical doses

Information By: University of Utah

Dates:
Date Received: September 6, 2006
Date Started: September 2003
Date Completion: February 2006
Last Updated: August 22, 2016
Last Verified: August 2016