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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01281475 |
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Date of registration:
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20/01/2011 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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A Trial of Levodopa in Angelman Syndrome
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Scientific title:
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A Phase 2 Randomized Placebo-Controlled Trial of Levodopa in Angelman Syndrome |
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Date of first enrolment:
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January 2011 |
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Target sample size:
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67 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01281475 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 2/Phase 3
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Countries of recruitment
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United States
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Contacts
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Name:
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Steven A. Skinner, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Greenwood Genetic Center |
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Name:
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Carlos A. Bacino, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Baylor College of Medicine |
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Name:
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Cary Fu, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Vanderbilt University Medical Center |
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Name:
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Lynne M. Bird, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Rady Children's Hospital, San Diego |
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Name:
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Anne Slavotinek, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of California, San Francisco |
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Name:
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Wen-Hann Tan, BMBS |
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Address:
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Telephone:
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Email:
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Affiliation:
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Boston Children's Hospital |
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Name:
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Logan Wink, M.D |
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Address:
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Telephone:
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Email:
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Affiliation:
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Children's Hospital Medical Center, Cincinnati |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Age between 4 years and 12 years (i.e., before the 13th birthday)
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:
1. Co-morbid disorders that may be associated with developmental or cognitive delays
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
Age minimum:
4 Years
Age maximum:
12 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Angelman Syndrome
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Intervention(s)
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Drug: Levodopa
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Drug: Placebo Oral Capsule
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Primary Outcome(s)
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Bayley Cognitive Age Equivalent at 1 Year
[Time Frame: 12 months]
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Secondary Outcome(s)
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Presence of Tremors
[Time Frame: 1 year]
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Secondary ID(s)
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09-12-0610
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3523
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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