Overview

A Trial of Levodopa in Angelman Syndrome

Status:
Completed

Trial end date:
2015-07-01

Target enrollment:
0

Participant gender:
All

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

Phase:

Phase 2/Phase 3

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Wen-Hann Tan

Collaborators:

Angelman Syndrome Foundation, Inc.
Baylor College of Medicine
Children's Hospital Medical Center, Cincinnati
Greenwood Genetic Center
Rady Children's Hospital, San Diego
University of California, San Francisco
Vanderbilt University
Vanderbilt University Medical Center

Treatments:

Carbidopa
Levodopa

Criteria

Inclusion Criteria:

1. Age between 4 years and 12 years (i.e., before the 13th birthday)

2. Molecular confirmation of the diagnosis of AS, which may include abnormal methylation
studies or UBE3A mutation analyses - only subjects with a molecular diagnosis will be
allowed to enroll

3. Not on LD, CD, or any dopamine agonists in the 2 weeks prior to participation

Exclusion Criteria:

1. Co-morbid disorders that may be associated with developmental or cognitive delays

2. Poorly controlled seizures - An average of more than 2 clinical seizures per month in
the 12 months prior to enrollment.

3. Use of medications that may interact with LD/CD including atypical antipsychotics
(aripiprazole, asenapine, iloperidone, olanzapine, paliperidone, risperidone,
ziprasidone), monoamine oxidase inhibitors (isocarboxazid, phenelzine, selegiline,
tranylcypromine), or phenytoin within the last 14 days, or other investigational
interventions within the past 3 months

4. Presence of cardiovascular disease or instability, respiratory disease, liver disease,
peptic ulcer disease, renal impairment, or hematological disorders

5. Pregnancy