Dextromethorphan in Rett syndrome

Principal Investigator: SakkuBai Naidu

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 glutamine, 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 between two to 14.99 years of age.

Participate in Research:

If you or someone you know is interested in participating in this research study, please download this informational flyer.