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

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ClinicalTrials.gov Identifier: NCT00374075
Recruitment Status : Completed
First Posted : September 8, 2006
Last Update Posted : August 23, 2016
Information provided by (Responsible Party):

Study Description
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.

Condition or disease Intervention/treatment Phase
Spinal Muscular Atrophy Drug: Valproic Acid Phase 1

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.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 42 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : September 2003
Estimated Study Completion Date : February 2006

Arms and Interventions

Outcome Measures

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

Secondary Outcome Measures :
  1. To look for a potential in vivo effect of VPA on SMN mRNA in patient blood cells at routinely used clinical doses
  2. Measures of gross motor function
  3. Electrophysiologic measures of denervation
  4. DEXA estimates of body composition, bone mineral density and content
  5. Measures of pulmonary function

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients must have a diagnosis of SMA, confirmed by genetic testing
  • Only patients 2 years of age and older at enrollment will be eligible

Exclusion Criteria:

  • Patients taking any medications with known hepatotoxicity, congenital metabolic disorders or on multiple anticonvulsant medications
  • Patients taking medications which may interact with VPA
  • Patients on ventilatory support for more than 16 hours per day
  • Patients currently enrolled in other treatment trials
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00374075

United States, Utah
University of Utah/Primary Children's Medical Center
Salt Lake City, Utah, United States, 84132
Sponsors and Collaborators
University of Utah
Families of Spinal Muscular Atrophy
Leadiant Biosciences, Inc.
Principal Investigator: Kathryn J Swoboda, M.D University of Utah/Primary Children's Medical Center
More Information

Additional Information:
Lindstedt S, Lindstedt G. Distribution and Excretion of Carnitine in the Rat. Acta. Chem. Scand. 1961; 15:701-702
Scriver C, Beautet A, Sly W ,Valle D. The Metabolic Basis of Inherited Disease. New York: McGraw Hill,1989
Schaub J, Van Hoof F, Vis H.Inborn Errors of Metabolism. New York:Raven Press, 1991

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Kathryn Swoboda, M.D., University of Utah
ClinicalTrials.gov Identifier: NCT00374075     History of Changes
Other Study ID Numbers: 11893
First Posted: September 8, 2006    Key Record Dates
Last Update Posted: August 23, 2016
Last Verified: August 2016

Keywords provided by Kathryn Swoboda, University of Utah:
Spinal Muscular Atrophy (SMA)
Valproic Acid

Additional relevant MeSH terms:
Muscular Atrophy
Muscular Atrophy, Spinal
Spinal Cord Diseases
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases
Valproic Acid
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
GABA Agents
Neurotransmitter Agents
Physiological Effects of Drugs
Antimanic Agents
Tranquilizing Agents
Central Nervous System Depressants
Psychotropic Drugs