drugset / Trial / NCT01281475
A Trial of Levodopa in Angelman Syndrome
Phase 2/3
Completed
67 enrolled
Wen-Hann Tan
Angelman Syndrome Foundation, Inc. · collabBaylor College of Medicine · collabChildren's Hospital Medical Center, Cincinnati · collabGreenwood Genetic Center · collabRady Children's Hospital, San Diego · collabUniversity of California, San Francisco · collabVanderbilt University Medical Center · collab
RandomizedParallel-groupQuadruple-blindTreatment
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
Timeline
- Start
- 2011-01
- Primary completion
- 2015-07
- Completion
- 2015-07
Outcome
Missed primary endpoint
registry analysis (superiority test); Levodopa vs Placebo; p = 0.75; Generalized estimating equations NCT01281475 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Levodopa | Unknown | 15 mg/kg | Oral |