Clinical Trial: Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy

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

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

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

Detailed Summary:

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.


Sponsor: University of Utah

Current Primary Outcome: The primary outcome for the study is change in muscle strength from baseline to six months in muscle strength as assessed by MVICT using a fixed testing system. [ Time Frame: 13 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in SMAFRS [ Time Frame: 13 months ]
  • Change in strength assessed by hand-held dynamometer [ Time Frame: 13 months ]
  • Change in MUNE and CMAP [ Time Frame: 13 months ]
  • SMN2 copy number [ Time Frame: 13 months ]
  • Change in PFTs, including forced vital capacity (FVC) and negative inspiratory force (NIF) [ Time Frame: 13 months ]
  • Change in lean body mass through DEXA scanning [ Time Frame: 13 months ]
  • Change in distance walked in 6 minutes [ Time Frame: 13 months ]
  • Change in time to climb four standard stairs [ Time Frame: 13 months ]
  • Change in health-related QOL assessed through the modified sickness impact profile (SIP) [ Time Frame: 13 months ]


Original Secondary Outcome: Same as current

Information By: University of Utah

Dates:
Date Received: May 30, 2007
Date Started: July 2007
Date Completion:
Last Updated: December 5, 2016
Last Verified: December 2016