Study on Tolerability of Levodopa/Carbidopa in Children With Angelman Syndrome

• Status: Completed
• Trial end date: 2010-06-01
• Target enrollment: 0
• Participant gender: All

Summary

This study is designed to determine the highest dose of levodopa/carbidopa that can be tolerated without any serious side effects by children with Angelman syndrome.

It has been hypothesized that levodopa may lead to an improvement in the neurodevelopment and abnormal movements (e.g. tremors) in children with Angelman syndrome.

Data from this study will be used to design a phase II trial to determine the efficacy of levodopa in treating children with Angelman syndrome.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Boston Children's Hospital; Boston Children’s Hospital
• Treatments: Carbidopa; Carbidopa, levodopa drug combination; Levodopa

Criteria

Inclusion Criteria:

• Angelman syndrome, confirmed by molecular testing

• Must be willing to come for research visit on 2 days, exactly 1 week apart

Exclusion Criteria:

• On levodopa, carbidopa, or any dopamine agonists in the 2 weeks prior to participation

• Other medical conditions that may be associated with developmental or cognitive delays

• More than 2 clinical seizures per month

• Used monoamine oxidase (MAO) inhibitors within the last 2 weeks

• Used phenytoin within the last 2 weeks

• Used phenothiazines, butyrophenones, and thioxanthenes within last 2 weeks

• Hypersensitive to levodopa or carbidopa

• Cardiovascular disease or instability

• Respiratory diseases, including asthma, emphysema, chronic cough, and shortness of breath

• Liver disease

• Stomach or intestinal ulcers

• Kidney disease

• Hematological problems, including anemia, leucopenia, and thrombocytopenia

• Used investigational drugs/interventions within the past three months