Placebo Controlled Trial of Dextromethorphan in Rett Syndrome
Status:
Completed
Trial end date:
2016-10-26
Target enrollment:
Participant gender:
Summary
Dr. Sakkubai Naidu, Principal Investigator, is initiating a double blinded placebo controlled
clinical drug trial using dextromethorphan (DM) in Rett Syndrome (RTT), at the Pediatric
Clinical Research Unit (PCRU) of the Johns Hopkins Hospital/Kennedy Krieger Institute.
Funding source , FDA-00PD
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 (<10 years of age). 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, and learning
disabilities in RTT.
The investigators propose to initiate a specific treatment using DM to counter/block the
effects of this brain chemical and its excessive receptors to improve the ill effects of
increased glutamate/NMDA receptors, because of DM's identified ability to block NMDA
receptors. DM 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.
Phase:
Phase 2
Details
Lead Sponsor:
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Collaborator:
The Johns Hopkins Institute for Clinical and Translational Research (ICTR)