CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
NCT ID: NCT00661453
Last Updated: 2015-06-15
Study Results
Results available
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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Recruitment Status
COMPLETED
Clinical Phase
PHASE1/PHASE2
Total Enrollment
40 participants
Study Classification
INTERVENTIONAL
Study Start Date
2008-04-30
Study Completion Date
2012-06-30
Brief Summary
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This is a multi-center trial to test safety and evaluate early treatment intervention with valproic acid and carnitine in moderating SMA symptoms of Type I infants.
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Detailed Description
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Spinal muscular atrophy (SMA) is a genetic disorder that results in severe muscle weakness. It is one of the most common conditions causing muscle weakness in children. Patients with SMA most often develop weakness as babies or young children. Most people with SMA gradually lose muscle strength and abilities over time. Babies with the severe infantile form of SMA, SMA type I, usually lose abilities and strength quickly over a few weeks or months.
Valproic acid (VPA) is a medicine that has been used for many years to treat patients with epilepsy. Recent research suggests that VPA may be able to upregulate expression of a backup copy of the SMN gene in SMA patient cell lines. In addition, some preliminary data suggests it may prolong survival in animal models of SMA. Because VPA can deplete carnitine in children with SMA Type I, carnitine is added to help prevent possible toxicity.
In this multi-center trial, we will evaluate the effects of VPA/carnitine on infants with SMA type I. A variety of outcome measures, including assessment of safety, will be performed at each study visit to follow the course of the disease. The protocol includes two baseline visits over a period of two weeks, two clinical assessments on medication at 3 and 6 months, and then 6 months additional followup via telephone. Total duration of the study will be approximately 12 months.
Valproic acid (VPA) is a medicine that has been used for many years to treat patients with epilepsy. Recent research suggests that VPA may be able to upregulate expression of a backup copy of the SMN gene in SMA patient cell lines. In addition, some preliminary data suggests it may prolong survival in animal models of SMA. Because VPA can deplete carnitine in children with SMA Type I, carnitine is added to help prevent possible toxicity.
In this multi-center trial, we will evaluate the effects of VPA/carnitine on infants with SMA type I. A variety of outcome measures, including assessment of safety, will be performed at each study visit to follow the course of the disease. The protocol includes two baseline visits over a period of two weeks, two clinical assessments on medication at 3 and 6 months, and then 6 months additional followup via telephone. Total duration of the study will be approximately 12 months.
Conditions
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Spinal Muscular Atrophy Type I
Study Design
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Allocation Method
NA
Intervention Model
SINGLE_GROUP
Primary Study Purpose
TREATMENT
Blinding Strategy
NONE
Study Groups
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1
All patients will receive VPA and carnitine.
Group Type
EXPERIMENTAL
Valproic Acid and Levocarnitine
Intervention Type
DRUG
Drug: Valproic Acid and Levocarnitine; syrup; dosage is by weight
Interventions
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Valproic Acid and Levocarnitine
Drug: Valproic Acid and Levocarnitine; syrup; dosage is by weight
Intervention Type
DRUG
Eligibility Criteria
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Inclusion Criteria
* Laboratory documentation of SMN mutation/deletion consistent with a genetic diagnosis of SMA
* Clinical diagnosis of SMA type I
* Age 2 weeks to 12 months
* Written informed consent of parents/guardian
* Clinical diagnosis of SMA type I
* Age 2 weeks to 12 months
* Written informed consent of parents/guardian
Exclusion Criteria
* Any clinical or laboratory evidence of hepatic or pancreatic insufficiency.
* Laboratory results drawn within 14 days prior to start of study drug demonstrating:
Liver transaminases (AST, ALT), lipase, amylase: \> 1.5 x ULN White Blood Cell Count: \< 3 Neutropenia: \<1 Platelet: \<100K Hematocrit: \<30, persisting over a 30-day period
* Serious illness requiring systemic treatment and/or hospitalization within two weeks prior to study entry.
* Use of medications or supplements within 30 days of study enrollment that interfere with VPA or carnitine metabolism; that increase the potential risks of VPA or carnitine; or that are hypothesized to have a beneficial effect in SMA animal models or human neuromuscular disorders, including riluzole, valproic acid, hydroxyurea, oral use of albuterol, sodium phenylbutyrate, butyrate derivatives, creatinine, growth hormone, anabolic steroids, probenecid, oral or parenteral use of corticosteroids at entry, or agents anticipated to increase or decrease muscle strength or agents with presumed histone deacetylase (HDAC) inhibition.
* Infants who have participated in a treatment trial for SMA within 30 days of study entry or who will become enrollees in any other treatment trial during the course of this study.
* Unwillingness to travel for study assessments.
* Coexisting medical conditions that contradict use of VPA/carnitine or travel to and from study site.
* Laboratory results drawn within 14 days prior to start of study drug demonstrating:
Liver transaminases (AST, ALT), lipase, amylase: \> 1.5 x ULN White Blood Cell Count: \< 3 Neutropenia: \<1 Platelet: \<100K Hematocrit: \<30, persisting over a 30-day period
* Serious illness requiring systemic treatment and/or hospitalization within two weeks prior to study entry.
* Use of medications or supplements within 30 days of study enrollment that interfere with VPA or carnitine metabolism; that increase the potential risks of VPA or carnitine; or that are hypothesized to have a beneficial effect in SMA animal models or human neuromuscular disorders, including riluzole, valproic acid, hydroxyurea, oral use of albuterol, sodium phenylbutyrate, butyrate derivatives, creatinine, growth hormone, anabolic steroids, probenecid, oral or parenteral use of corticosteroids at entry, or agents anticipated to increase or decrease muscle strength or agents with presumed histone deacetylase (HDAC) inhibition.
* Infants who have participated in a treatment trial for SMA within 30 days of study entry or who will become enrollees in any other treatment trial during the course of this study.
* Unwillingness to travel for study assessments.
* Coexisting medical conditions that contradict use of VPA/carnitine or travel to and from study site.
Minimum Eligible Age
2 Weeks
Maximum Eligible Age
12 Months
Eligible Sex
ALL
Accepts Healthy Volunteers
No
Sponsors
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Families of Spinal Muscular Atrophy
OTHER
Sponsor Role
collaborator
Leadiant Biosciences, Inc.
INDUSTRY
Sponsor Role
collaborator
University of Utah
OTHER
Sponsor Role
lead
Responsible Party
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Kathryn Swoboda
Associate Professor, Neurology and Pediatrics Director, Pediatric Motor Disorders Research Program
Responsibility Role
PRINCIPAL_INVESTIGATOR
Principal Investigators
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Kathryn Swoboda, M.D.
Role: PRINCIPAL_INVESTIGATOR
University of Utah
Sandra P Reyna, M.D.
Role: STUDY_DIRECTOR
Families of Spinal Muscular Atrophy
Locations
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Johns Hopkins University
Baltimore, Maryland, United States
Site Status
Children's Hospital of Michigan
Detroit, Michigan, United States
Site Status
Duke University Medical Center
Durham, North Carolina, United States
Site Status
Ohio State University Medical Center, Dept. of Neurology
Columbus, Ohio, United States
Site Status
University of Utah/Primary Children's Medical Center
Salt Lake City, Utah, United States
Site Status
University of Wisconsin Children's Hospital
Madison, Wisconsin, United States
Site Status
Hospital Sainte-Justine
Montreal, Quebec, Canada
Site Status
Klinikum der Universität zu Köln
Cologne, , Germany
Site Status
Countries
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United States
Canada
Germany
References
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Other Identifiers
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IND 79276
Identifier Type: -
Identifier Source: secondary_id
25409
Identifier Type: -
Identifier Source: org_study_id
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