Phase 1 Completed N=16 Treatment

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

Angelman Syndrome

Source: ClinicalTrials.gov NCT00829439 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Nov 2016

Primary outcomePrimary: Maximum Dose of Levodopa/Carbidopa That Can be Tolerated (Without Any Dose Limiting Toxicity) by at Least 3 Subjects. — 15 mg/kg/day

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.

Outcome Measures

Outcome	Result	p-value
PRIMARY Maximum Dose of Levodopa/Carbidopa That Can be Tolerated (Without Any Dose Limiting Toxicity) by at Least 3 Subjects.	15	—

Eligibility 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00829439). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.