Vanderbilt Kennedy Center

Medication Study for Angelman syndrome: L-dopa Phase II Trial

Last Updated: Wednesday, May 15, 2013

Principal Investigator: Gregory N. Barnes, M.D., Ph.D.

Learn more about Research Family Partners

Description

L-dopa has been safely and effectively used by people with Parkinson's disease for many decades. Recent understandings of brain function in people with Angelman leads us to expect that L-dopa could be beneficial in people with Angelman syndrome.

Previous studies have already determined the safety of this dosage in 4 to 12 year old children. Now we are interested in whether and how it might help them.

This study works alongside our Natural History study to evaluate the medical and psychological functioning of children with Angelman. For this study there will also be:

  • There are 5 visits over 2 years.
  • Blood and urine samples will be taken.
  • EEG recordings will be provided.
  • Daily medication will be provided for two years. This medication may be the FDA approved L-dopa or a placebo capsule.
  • There will be monthly phone checks on the child's health and progress.
  • Study doctors will be available for questions and concerns.
  • Some travel assistance may be available.

Participant Criteria

4 to 12 year old children with Angelman syndrome

Compensation

Medical and psychological evaluations and reports

Visit Requirements

5 day long visits

Brochure

Download Brochure

Contact Information

Stefanie Barnett, CCRP
615-343-5750 (Mon-Wed) 615-875-4897 (Thur-Fri)
stefanie.barnett@vanderbilt.edu

Want to Participate in this Study?

Enter your name and contact information and this study's coordinators will contact you if there are openings available.

 
 
(Optional)