Clinical Trial: A Trial of Levodopa in Angelman Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief 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

Detailed Summary:

Levodopa is a prodrug that "delivers" dopamine to the brain. It is usually given with carbidopa, a peripheral decarboxylase inhibitor, to increase the bioavailability of levodopa. Animal studies have suggested that levodopa can reverse the excess phosphorylation of some enzymes involved in synaptic and neuronal function, including calcium/calmodulin-dependent kinase type 2 (CaMKII).

Recently, it was shown that excess phosphorylation of CaMKII may be responsible for some of the neurological deficits seen in Angelman syndrome. Therefore, it is hypothesized that levodopa may lead to an improvement in the neurodevelopment and abnormal movements (e.g. tremors) in children with Angelman syndrome.

Although many children have used levodopa for a variety of medical conditions over the last 30 years, it has not been approved by the Food and Drug Administration (FDA) for use in children, and it has not been formally studied in children with Angelman syndrome.

Therefore, the purpose of this study is to find out whether levodopa will lead to an improvement in the development and in the tremor in children with AS.

Sponsor: Wen-Hann Tan

Current Primary Outcome: Bayley Cognitive Age Equivalent at 1 Year [ Time Frame: 12 months ]

Original Primary Outcome:

• Developmental Outcome Measures [ Time Frame: 12 months ]

  Changes in raw or standard scores between baseline and after 12 months of treatment with either placebo or LD/CD of:

  I. Bayley Scales of Infant and Toddler Development, 3rd edition (or the Mullen Scales of Early Learning in the more developmentally advanced subjects) II. Vineland Adaptive Behavior Scales, 2nd edition (standard scores only) III. Preschool Language Scale, 4th edition IV. Aberrant Behavior Checklist - Community version

• Tremor and Movement Disorder [ Time Frame: 12 months ]
  Changes in raw score of a modified Unified Parkinson's Disease Rating Scale (mUPDRS), a measure of tremor and movement disorder, from baseline after 12 months in the placebo or the LD/CD group.

Current Secondary Outcome: Presence of Tremors [ Time Frame: 1 year ]

Original Secondary Outcome: EEG [ Time Frame: 12 months, 24 months ]

Information By: Boston Children’s Hospital

Dates:
Date Received: January 20, 2011
Date Started: January 2011
Date Completion:
Last Updated: November 16, 2016
Last Verified: November 2016