Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

Completed · Phase 2 · Has a placebo group

Conditions studied: Rett Syndrome

In brief

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.

Key facts

Study ID

NCT01520363

Run by

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

People needed

57

Starts

2012-03-01

Expected to finish

2016-10-26

Last updated by the study team

2018-12-04

Who can join

Age: 1 and older, up to 10. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• The Johns Hopkins Institute for Clinical and Translational Research — Baltimore, Maryland, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.