Home
Search
Study Topics
Glossary
|
|
|
|
|
Sponsors and Collaborators: |
University of Utah Families of Spinal Muscular Atrophy Abbott |
---|---|
Information provided by: | University of Utah |
ClinicalTrials.gov Identifier: | NCT00481013 |
The primary objective of this proposal is to determine whether oral VPA is effective in treating SMA in adult patients.
Condition | Intervention | Phase |
---|---|---|
Spinal Muscular Atrophy |
Drug: Valproic Acid (VPA) Drug: Placebo |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Crossover Assignment, Efficacy Study |
Official Title: | Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study |
Estimated Enrollment: | 36 |
Study Start Date: | July 2007 |
Estimated Study Completion Date: | August 2010 |
Estimated Primary Completion Date: | August 2010 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
---|---|
1a: Placebo Comparator
For six months, half of patients are randomized into placebo . After 6 months, all patients are on treatment.
|
Drug: Placebo
For six months, pts are randomized into placebo or treatment. After 6 months, all pts are on treatment
|
1b: Active Comparator
Cohort 1b patients are randomized onto treatment. After 6 months, all patients are on drug.
|
Drug: Valproic Acid (VPA)
Drug: Valproic Acid and Levocarnitine; capsules
|
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.
Ages Eligible for Study: | 18 Years to 60 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Sharon Chelnick, . | 614-293-4973 |
United States, Ohio | |
Ohio State University Medical Center, Dept. of Neurology | Recruiting |
Columbus, Ohio, United States, 43210 | |
Contact: Sharon Chelnick 614-293-4973 | |
Principal Investigator: John T Kissel |
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 |
Responsible Party: | Ohio State University Medical Center ( John T. Kissel ) |
Study ID Numbers: | 2006H0249 |
Study First Received: | May 30, 2007 |
Last Updated: | July 17, 2009 |
ClinicalTrials.gov Identifier: | NCT00481013 History of Changes |
Health Authority: | United States: Institutional Review Board |
Spinal Muscular Atrophy Adult |
Pathological Conditions, Anatomical Neurotransmitter Agents Tranquilizing Agents Spinal Cord Diseases Psychotropic Drugs Spinal Muscular Atrophy Central Nervous System Depressants Central Nervous System Diseases Antimanic Agents Neurodegenerative Diseases Valproic Acid |
Signs and Symptoms Neuromuscular Diseases Muscular Atrophy, Spinal Neurologic Manifestations Atrophy Degenerative Motor System Disease Motor Neuron Disease Anticonvulsants Carnitine Muscular Atrophy Progressive Spinal Muscular Atrophy |
Pathological Conditions, Anatomical Neurotransmitter Agents Spinal Cord Diseases Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs Psychotropic Drugs Neurodegenerative Diseases Valproic Acid Signs and Symptoms Neuromuscular Diseases Muscular Atrophy, Spinal Therapeutic Uses Motor Neuron Disease Muscular Atrophy |
Neuromuscular Manifestations Tranquilizing Agents Nervous System Diseases Central Nervous System Diseases Central Nervous System Depressants Enzyme Inhibitors Antimanic Agents Pharmacologic Actions GABA Agents Neurologic Manifestations Atrophy Central Nervous System Agents Anticonvulsants |