A Trial of Levodopa in Angelman Syndrome
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ClinicalTrials.gov Identifier: NCT01281475 |
Recruitment Status :
Completed
First Posted : January 24, 2011
Results First Posted : January 13, 2017
Last Update Posted : July 15, 2020
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Tracking Information | ||||||||||||||||||||||
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First Submitted Date ICMJE | January 20, 2011 | |||||||||||||||||||||
First Posted Date ICMJE | January 24, 2011 | |||||||||||||||||||||
Results First Submitted Date ICMJE | August 6, 2016 | |||||||||||||||||||||
Results First Posted Date ICMJE | January 13, 2017 | |||||||||||||||||||||
Last Update Posted Date | July 15, 2020 | |||||||||||||||||||||
Study Start Date ICMJE | January 2011 | |||||||||||||||||||||
Actual Primary Completion Date | July 2015 (Final data collection date for primary outcome measure) | |||||||||||||||||||||
Current Primary Outcome Measures ICMJE |
Bayley Cognitive Age Equivalent at 1 Year [ Time Frame: 12 months ] | |||||||||||||||||||||
Original Primary Outcome Measures ICMJE |
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Change History | ||||||||||||||||||||||
Current Secondary Outcome Measures ICMJE |
Presence of Tremors [ Time Frame: 1 year ] | |||||||||||||||||||||
Original Secondary Outcome Measures ICMJE |
EEG [ Time Frame: 12 months, 24 months ] Changes in electroencephalogram (EEG) scores at 12 months and 24 months.
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Current Other Pre-specified Outcome Measures | Not Provided | |||||||||||||||||||||
Original Other Pre-specified Outcome Measures | Not Provided | |||||||||||||||||||||
Descriptive Information | ||||||||||||||||||||||
Brief Title ICMJE | A Trial of Levodopa in Angelman Syndrome | |||||||||||||||||||||
Official Title ICMJE | A Phase 2 Randomized Placebo-Controlled Trial of Levodopa in Angelman Syndrome | |||||||||||||||||||||
Brief Summary | This study is designed to determine whether levodopa will lead to an improvement in the development and tremor in children with Angelman syndrome (AS). 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 | Levodopa is a prodrug that "delivers" dopamine to the brain. It is usually given with carbidopa, a peripheral decarboxylase inhibitor, to increase the bioavailability of levodopa. Animal studies have suggested that levodopa can reverse the excess phosphorylation of some enzymes involved in synaptic and neuronal function, including calcium/calmodulin-dependent kinase type 2 (CaMKII). 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. |
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Study Type ICMJE | Interventional | |||||||||||||||||||||
Study Phase ICMJE | Phase 2 Phase 3 |
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Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
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Condition ICMJE | Angelman Syndrome | |||||||||||||||||||||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | |||||||||||||||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||||||||||||||||||||
Recruitment Status ICMJE | Completed | |||||||||||||||||||||
Actual Enrollment ICMJE |
67 | |||||||||||||||||||||
Original Estimated Enrollment ICMJE |
100 | |||||||||||||||||||||
Actual Study Completion Date ICMJE | July 2015 | |||||||||||||||||||||
Actual Primary Completion Date | July 2015 (Final data collection date for primary outcome measure) | |||||||||||||||||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 4 Years to 12 Years (Child) | |||||||||||||||||||||
Accepts Healthy Volunteers ICMJE | No | |||||||||||||||||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||||||||||||||||||||
Listed Location Countries ICMJE | United States | |||||||||||||||||||||
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Administrative Information | ||||||||||||||||||||||
NCT Number ICMJE | NCT01281475 | |||||||||||||||||||||
Other Study ID Numbers ICMJE | 09-12-0610 3523 ( Other Grant/Funding Number: OOPD ) |
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Has Data Monitoring Committee | Yes | |||||||||||||||||||||
U.S. FDA-regulated Product | Not Provided | |||||||||||||||||||||
IPD Sharing Statement ICMJE |
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Current Responsible Party | Wen-Hann Tan, Boston Children's Hospital | |||||||||||||||||||||
Original Responsible Party | Wen-Hann Tan, BMBS / Attending Physician in Genetics, Children's Hospital Boston | |||||||||||||||||||||
Current Study Sponsor ICMJE | Wen-Hann Tan | |||||||||||||||||||||
Original Study Sponsor ICMJE | Boston Children's Hospital | |||||||||||||||||||||
Collaborators ICMJE |
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Investigators ICMJE |
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PRS Account | Boston Children's Hospital | |||||||||||||||||||||
Verification Date | July 2020 | |||||||||||||||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |