Angelman Syndrome Clinical Trial
Official title:
A Phase 2 Randomized Placebo-Controlled Trial of Levodopa in Angelman Syndrome
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
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.
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