At a glance
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A Phase 2 Randomized Placebo-Controlled Trial of Levodopa in Angelman Syndrome
In Brief
A Phase 3 clinical trial evaluating Levodopa and Placebo Oral Capsule for Angelman Syndrome. Completed, enrolled 67 participants across 7 sites.
Detailed 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
Study Details
Timeline
Interventions
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.
The placebo contains excipients similar to those in the active drug, but it does not contain levodopa or carbidopa.