A Trial of Levodopa in Angelman Syndrome

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

Conditions

• Angelman Syndrome

Interventions

DRUG

Levodopa

Levodopa/Carbidopa (4:1) Dosages are based on levodopa. Subjects randomized to the levodopa arm will receive a levodopa dose of 5 mg/kg/day in the first 2 weeks of the study, a levodopa dose of 10 mg/kg/day in the second 2 weeks of the study, and a levodopa dose of 15 mg/kg/day (up to a maximum of 800 mg per day) for the remaining duration of the study. Levodopa/Carbidopa is a combined formulation that will be dispensed as capsules. It should be taken 3 times a day.

DRUG

Placebo Oral Capsule

The placebo contains excipients similar to those in the active drug, but it does not contain levodopa or carbidopa.

Sponsors & Collaborators

• Rady Children's Hospital, San Diego

  collaborator OTHER

• University of California, San Francisco

  collaborator OTHER

• Baylor College of Medicine

  collaborator OTHER

• Vanderbilt University Medical Center

  collaborator OTHER

• Greenwood Genetic Center

  collaborator OTHER

• Children's Hospital Medical Center, Cincinnati

  collaborator OTHER

• Angelman Syndrome Foundation, Inc.

  collaborator OTHER

• Wen-Hann Tan

  lead OTHER

Principal Investigators

• Wen-Hann Tan, BMBS · Boston Children's Hospital

• Lynne M. Bird, MD · Rady Children's Hospital, San Diego

• Steven A. Skinner, MD · Greenwood Genetic Center

• Carlos A. Bacino, MD · Baylor College of Medicine

• Anne Slavotinek, MD · University of California, San Francisco

• Cary Fu, MD · Vanderbilt University Medical Center

• Logan Wink, M.D · Children's Hospital Medical Center, Cincinnati

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

4 Years

Max Age

12 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2011-01-31

Primary Completion

2015-07-31

Completion

2015-07-31

Countries

• United States

Study Locations