Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2/PHASE3
67 participants
INTERVENTIONAL
2011-01-31
2015-07-31
Brief Summary
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It has been suggested that levodopa, a medication that is usually used to treat Parkinson disease in adults, may help children with AS in their overall development and reduce the tremor that some of them have.
If levodopa is found to be beneficial for children with AS, this could lead to a new treatment for AS.
Funding Source - FDA-OOPD
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Detailed Description
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Recently, it was shown that excess phosphorylation of CaMKII may be responsible for some of the neurological deficits seen in Angelman syndrome. Therefore, it is hypothesized that levodopa may lead to an improvement in the neurodevelopment and abnormal movements (e.g. tremors) in children with Angelman syndrome.
Although many children have used levodopa for a variety of medical conditions over the last 30 years, it has not been approved by the Food and Drug Administration (FDA) for use in children, and it has not been formally studied in children with Angelman syndrome.
Therefore, the purpose of this study is to find out whether levodopa will lead to an improvement in the development and in the tremor in children with AS.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Levodopa
Levodopa is prescribed as a combination of levodopa/carbidopa (4:1) to reduce the peripheral side effects. The dosage used was 15 mg/kg/day in 3 divided doses.
Levodopa
Levodopa/Carbidopa (4:1)
Dosages are based on levodopa.
Subjects randomized to the levodopa arm will receive a levodopa dose of 5 mg/kg/day in the first 2 weeks of the study, a levodopa dose of 10 mg/kg/day in the second 2 weeks of the study, and a levodopa dose of 15 mg/kg/day (up to a maximum of 800 mg per day) for the remaining duration of the study.
Levodopa/Carbidopa is a combined formulation that will be dispensed as capsules. It should be taken 3 times a day.
Placebo
The placebo contains excipients similar to those in the active drug, but it does not contain levodopa or carbidopa, so it is not expected to have any effect.
Placebo Oral Capsule
The placebo contains excipients similar to those in the active drug, but it does not contain levodopa or carbidopa.
Interventions
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Levodopa
Levodopa/Carbidopa (4:1)
Dosages are based on levodopa.
Subjects randomized to the levodopa arm will receive a levodopa dose of 5 mg/kg/day in the first 2 weeks of the study, a levodopa dose of 10 mg/kg/day in the second 2 weeks of the study, and a levodopa dose of 15 mg/kg/day (up to a maximum of 800 mg per day) for the remaining duration of the study.
Levodopa/Carbidopa is a combined formulation that will be dispensed as capsules. It should be taken 3 times a day.
Placebo Oral Capsule
The placebo contains excipients similar to those in the active drug, but it does not contain levodopa or carbidopa.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Molecular confirmation of the diagnosis of AS, which may include abnormal methylation studies or UBE3A mutation analyses - only subjects with a molecular diagnosis will be allowed to enroll
3. Not on LD, CD, or any dopamine agonists in the 2 weeks prior to participation
Exclusion Criteria
2. Poorly controlled seizures - An average of more than 2 clinical seizures per month in the 12 months prior to enrollment.
3. Use of medications that may interact with LD/CD including atypical antipsychotics (aripiprazole, asenapine, iloperidone, olanzapine, paliperidone, risperidone, ziprasidone), monoamine oxidase inhibitors (isocarboxazid, phenelzine, selegiline, tranylcypromine), or phenytoin within the last 14 days, or other investigational interventions within the past 3 months
4. Presence of cardiovascular disease or instability, respiratory disease, liver disease, peptic ulcer disease, renal impairment, or hematological disorders
5. Pregnancy
4 Years
12 Years
ALL
No
Sponsors
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Rady Children's Hospital, San Diego
OTHER
University of California, San Francisco
OTHER
Baylor College of Medicine
OTHER
Vanderbilt University Medical Center
OTHER
Greenwood Genetic Center
OTHER
Children's Hospital Medical Center, Cincinnati
OTHER
Angelman Syndrome Foundation, Inc.
OTHER
Wen-Hann Tan
OTHER
Responsible Party
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Wen-Hann Tan
Attending Physician in Genetics
Principal Investigators
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Wen-Hann Tan, BMBS
Role: PRINCIPAL_INVESTIGATOR
Boston Children's Hospital
Lynne M. Bird, MD
Role: PRINCIPAL_INVESTIGATOR
Rady Children's Hospital, San Diego
Steven A. Skinner, MD
Role: PRINCIPAL_INVESTIGATOR
Greenwood Genetic Center
Carlos A. Bacino, MD
Role: PRINCIPAL_INVESTIGATOR
Baylor College of Medicine
Anne Slavotinek, MD
Role: PRINCIPAL_INVESTIGATOR
University of California, San Francisco
Cary Fu, MD
Role: PRINCIPAL_INVESTIGATOR
Vanderbilt University Medical Center
Logan Wink, M.D
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital Medical Center, Cincinnati
Locations
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Rady Children's Hospital, San Diego
San Diego, California, United States
University of California, San Francisco
San Francisco, California, United States
Children's Hospital Boston
Boston, Massachusetts, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
Greenwood Genetic Center
Greenwood, South Carolina, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Baylor College of Medicine
Houston, Texas, United States
Countries
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Other Identifiers
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3523
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
09-12-0610
Identifier Type: -
Identifier Source: org_study_id
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