Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

• Status: Completed
• Trial end date: 2016-10-26
• Target enrollment: 0
• Participant gender: All

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
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
• Collaborator: The Johns Hopkins Institute for Clinical and Translational Research (ICTR)
• Treatments: Dextromethorphan

Criteria

Inclusion Criteria:

• males and females who have classic or atypical RTT with a proven mutation in the MECP2 gene;

• subjects must be between one year - 10 years of age.

Exclusion Criteria:

• those without an established mutation in the MECP2 gene;

• those with mutations in the MECP2 gene but who have had brain resection or surgical intervention; for example, tumor, hydrocephalus, severe head trauma; or, an associated severe medical illnesses such as vasculopathies, malignancies, diabetes, thyroid dysfunction, etc;

• those on medications that could interact with DM, e.g. MAO inhibitors, SSRI, sibutramine etc. to avoid a serotonin syndrome; quinidine and drugs metabolized by the CYP450 isoform CYP2D6 (e.g. amiodarone, haloperidol, propafenone, thioridazine);

• those proven to be intermediate or slow metabolizers of DM;

• those with reported adverse reactions to DM;

• those whose pregnancy test is positive;

• those showing poor compliance with any aspect of the study;

• foster children.