Dextromethorphan in Rett Syndrome

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Dr. SakkuBai Naidu, of the Kennedy Krieger Institute, is initiating a clinical drug trial using dextromethorphan (DM) in Rett Syndrome (RTT), at the Pediatric Clinical Research Unit (PCRU) of the Johns Hopkins Hospital. It has been shown that receptors for a certain brain chemical called glutamate, in particular the NMDA type, are increased in the brain of young RTT patients. This chemical and its receptors, when in excess, cause harmful over-stimulation of nerve cells in the brain, contributing in part to the seizures, behavioral problems, gastrointestinal (GI) and respiratory abnormalities, motor, and learning disabilities in RTT. We propose to initiate a specific treatment to counter/block the effects of this brain chemical and its excessive receptors using DM because of its identified ability to block NMDA receptors. This drug is available for human consumption. Infants and children with respiratory infections and cough, as well as non-ketotic hyperglycinemia, are treated with DM, which has been well tolerated. 

This clinical trial, which is a dose-dependent study, will determine the benefits of various doses of DM on EEG, seizures, cognition, behavior, respiratory irregularities, GI dysfunction, bone density, and motor impairment. The study will be limited to MeCP2 mutation-positive children, younger than 15 years of age, but will be initiated in those who are between 5-14.99 years of age, initially, to establish safety-before including children younger than 5 years of age. 

There is no financial compensation for participating in this research study. If you would like more information regarding risks, benefits, and other details, please contact the Kennedy Krieger Institute, Department of Neurogenetics, Genila Bibat, MD, Research Associate ( or BarbaraAnn Bradford, Clinical Coordinator ( phone #443-923-2778 or #1-800-873-3377.

  Approved 09/21/2006

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Last revised 08-Oct-2006 by Steven Leber