A Phase 2 Randomized Placebo-Controlled Trial of Levodopa in Angelman Syndrome

A multi-center clinical trial is now underway to research the use of the drug, levodopa, in individuals with Angelman syndrome. The goal of this study is to understand the efficacy of levodopa in children with Angelman syndrome. Many in the AS community were involved in Phase 1 of the study in which the appropriate dose of the drug to use for the next phase was determined.

The second phase of the study comprises a double-blind, placebo-controlled study for one (1) year followed by an open-label study with levodopa for an additional year. This means that if your child participates in Phase 2, s/he will receive either levodopa or placebo, but not both, for the first year. If s/he completes the first year of the study without dropping out of the study, you will be given the option of continuing with the second year of the study. During the second year of the study, your child will only receive levodopa. You and the researchers or providers with whom you will have direct contact will NOT know whether your child receives levodopa or the placebo until the whole study is completed. If you are interested in having your child participate in the Phase 2 trial, please read the inclusion/exclusion criteria below carefully and contact the study coordinator directly if your child is eligible.


Levodopa is a drug commonly used in adults with Parkinson's disease to control tremors and involuntary movements. Levodopa is not FDA-approved for use in children yet, but many children have used this drug for a variety of medical conditions over the last 30 years. Parents who put their children with AS on levodopa also self-reported improvements in cognition and abnormal movements. However, levodopa is not well-studied in children with AS, which is why levodopa is being studied in AS.

Inclusion criteria to participate in the levodopa study:

  1. Your child must be between 4-12 years of age (i.e. before his/her 13th birthday).
  2. Your child must have a confirmed molecular diagnosis of Angelman syndrome.
  3. You must be willing to bring your child to one of the study sites at least twice: Once for the initial study visit and again one (1) year later.

If you wish to continue with the study after the first year, then your child will need to be evaluated again at the end of the second year.

Exclusion criteria:

In addition to the inclusion criteria listed above, your child must also meet all of the following conditions:

  1. Not using levodopa, carbidopa, or any dopamine agonists in the two (2) weeks prior to participation
  2. No other medical condition that may be associated with developmental or cognitive delays
  3. Seizures, if any, are well controlled—no more than 2 clinical seizures per month
  4. Not using monoamine oxidase (MAO) inhibitors (e.g., isocarboxazid, phenelzine, selegiline, tranylcypromine) in the two (2) weeks prior to participation
  5. Not using atypical antipsychotics (e.g., aripiprazole, asenapine, iloperidone, olanzapine, paliperidone, risperidone, ziprasidone) in the two (2) weeks prior to participation
  6. Not hypersensitive to levodopa or carbidopa
  7. No cardiovascular disease or instability
  8. No respiratory diseases, including asthma, emphysema, chronic cough, and shortness of breath
  9. No liver disease
  10. No stomach or intestinal ulcers
  11. No kidney disease
  12. No hematological problems, including anemia, leucopenia, and thrombocytopenia
  13. Not using any investigational drugs/interventions within three (3) months prior to participation
  14. Not pregnant

The levodopa study is approved and partially funded by the FDA and partially funded by the Angelman Syndrome Foundation.

Greenwood Genetic Center is also an approved site for an Angelman Syndrome Natural History Study.

Study Participation and Information

Please direct all comments or questions regarding the levodopa study to the study site coordinator:

Fran Annese, LMSW
Clinical Research Coordinator
Greenwood Genetic Center
106 Gregor Mendel Circle
Greenwood, SC 29646

864-941-8100 or 1-888-GGC-GENE
This email address is being protected from spambots. You need JavaScript enabled to view it.