Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00481013
Recruitment Status : Completed
First Posted : June 1, 2007
Last Update Posted : December 6, 2016
Families of Spinal Muscular Atrophy
Information provided by (Responsible Party):
Kathryn Swoboda, University of Utah

May 30, 2007
June 1, 2007
December 6, 2016
July 2007
December 2009   (Final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT00481013 on Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy
Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study
The primary objective of this proposal is to determine whether oral VPA is effective in treating SMA in adult patients.

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.

Phase 2
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Spinal Muscular Atrophy
  • Drug: Valproic Acid (VPA)
    Drug: Valproic Acid and Levocarnitine; capsules
    Other Names:
    • Depakote
    • Carnitor
  • Drug: Placebo
    For six months, pts are randomized into placebo or treatment. After 6 months, all pts are on treatment
  • Placebo Comparator: 1a
    For six months, half of patients are randomized into placebo . After 6 months, all patients are on treatment.
    Intervention: Drug: Placebo
  • Active Comparator: 1b
    Cohort 1b patients are randomized onto treatment. After 6 months, all patients are on drug.
    Intervention: Drug: Valproic Acid (VPA)
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
November 2010
December 2009   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Ambulatory adults with SMA 3 ages 18-60. The diagnosis of SMA must be documented by the homozygous deletion of both SMN1 genes on standard genetic tests for the disorder. Patients must be able to walk thirty feet without assistance (i.e. no canes, walkers).
  2. Interest in participating and the ability to meet the study requirements.
  3. Women of child bearing age are required to be on birth control or abstain while participating in the study.

Exclusion Criteria:

  1. Non-ambulatory type 3 adults and all type 2 adults.
  2. Patients with co-morbid conditions that preclude travel, testing or study medications.
  3. Patients who have participated in a treatment trial for SMA in the 3 months prior to this trial, or plan on enrolling in any other treatment trial during the duration of this trial.
  4. Patients who are, in the investigator's opinion, mentally or legally incapacitated from providing informed consent for the study, or are otherwise unable to meet study requirements or cooperate reliably with study procedures, especially strength testing.
  5. Patients with a need for non-invasive ventilatory support (e.g. BiPAP) for > 12 hours/day
  6. Transaminases, amylase or lipase > 3.0 x normal values, WBC < 3.0 or neutropenia < 1.0, platelet count < 100 K, or hematocrit < 30 persisting over a 30 day period
  7. Use of medications or supplements which interfere with VPA metabolism and increase the potential risks of the medications, or are hypothesized to have a beneficial effect in SMA animal models or human neuromuscular disorders within 3 months of study enrollment. These agents include riluzole, creatine, butyrate derivatives, growth hormone, anabolic steroids, daily albuterol use, anticonvulsants, or other HDAC inhibitors.
  8. Women who are pregnant or who intend to become pregnant while participating in the research study or who are breastfeeding.
Sexes Eligible for Study: All
18 Years to 60 Years   (Adult)
Contact information is only displayed when the study is recruiting subjects
United States
Not Provided
Not Provided
Kathryn Swoboda, University of Utah
University of Utah
  • Families of Spinal Muscular Atrophy
  • Abbott
Principal Investigator: John T Kissel Ohio State University
Study Director: Sandra P Reyna, M.D. Families of Spinal Muscular Atrophy
Principal Investigator: Kathryn J Swoboda, M.D. University of Utah
University of Utah
December 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP