- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00481013
Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA)
Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
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.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
Ohio
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Columbus, Ohio, United States, 43210
- Ohio State University Medical Center, Dept. of Neurology
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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).
- Interest in participating and the ability to meet the study requirements.
- Women of child bearing age are required to be on birth control or abstain while participating in the study.
Exclusion Criteria:
- Non-ambulatory type 3 adults and all type 2 adults.
- Patients with co-morbid conditions that preclude travel, testing or study medications.
- 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.
- 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.
- Patients with a need for non-invasive ventilatory support (e.g. BiPAP) for > 12 hours/day
- 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
- 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.
- Women who are pregnant or who intend to become pregnant while participating in the research study or who are breastfeeding.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: 1a
For six months, half of patients are randomized into placebo .
After 6 months, all patients are on treatment.
|
For six months, pts are randomized into placebo or treatment.
After 6 months, all pts are on treatment
|
Active Comparator: 1b
Cohort 1b patients are randomized onto treatment.
After 6 months, all patients are on drug.
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Drug: Valproic Acid and Levocarnitine; capsules
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
|
13 months
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Change in SMAFRS
Time Frame: 13 months
|
13 months
|
Change in strength assessed by hand-held dynamometer
Time Frame: 13 months
|
13 months
|
Change in MUNE and CMAP
Time Frame: 13 months
|
13 months
|
SMN2 copy number
Time Frame: 13 months
|
13 months
|
Change in PFTs, including forced vital capacity (FVC) and negative inspiratory force (NIF)
Time Frame: 13 months
|
13 months
|
Change in lean body mass through DEXA scanning
Time Frame: 13 months
|
13 months
|
Change in distance walked in 6 minutes
Time Frame: 13 months
|
13 months
|
Change in time to climb four standard stairs
Time Frame: 13 months
|
13 months
|
Change in health-related QOL assessed through the modified sickness impact profile (SIP)
Time Frame: 13 months
|
13 months
|
Collaborators and Investigators
Sponsor
Investigators
- Study Director: Sandra P Reyna, M.D., Families of Spinal Muscular Atrophy
- Principal Investigator: John T Kissel, Ohio State University
- Principal Investigator: Kathryn J Swoboda, M.D., University of Utah
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Central Nervous System Diseases
- Nervous System Diseases
- Neurologic Manifestations
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neuromuscular Manifestations
- Pathological Conditions, Anatomical
- Spinal Cord Diseases
- Motor Neuron Disease
- Muscular Atrophy
- Atrophy
- Muscular Atrophy, Spinal
- Physiological Effects of Drugs
- Neurotransmitter Agents
- Molecular Mechanisms of Pharmacological Action
- Central Nervous System Depressants
- Enzyme Inhibitors
- Tranquilizing Agents
- Psychotropic Drugs
- GABA Agents
- Anticonvulsants
- Antimanic Agents
- Valproic Acid
Other Study ID Numbers
- 2006H0249
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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