Increased brain glutamate and its N-methyl-D-aspartate (NMDA) receptors found in the brain of
younger Rett syndrome (RTT) patients cause toxic damage to neurons (the brain's nerve cells),
and contributing to EEG spikes. Dextromethorphan (DM) acts by blocking NMDA/glutamate
receptors. This study is being done to determine if DM will prevent the harmful
over-stimulation of the neurons thereby reducing EEG spike activity. Treatment with DM
consists of one of 3 different doses (0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day),
and aims to find out which dose if any will help improve EEG abnormalities, behavior,
cognition, and reduce seizures, as well as improve breathing abnormalities, motor
capabilities, bone density, and GI dysfunction.
The study will include 90 females and males with RTT, 2 years-14.99 years of age, with a
mutation in the methyl CpG binding protein 2 (MECP2) gene, and spikes on EEG, with or without
clinical seizures.
Phase:
Phase 2
Details
Lead Sponsor:
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.