Placebo-Controlled Trial of Dextromethorphan in Rett Syndrome

Principal Investigator: SakkuBai Naidu

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, that is
sponsored by the FDA and Johns Hopkins Institute for Clinical and Translational Research
(ICTR).
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.
We propose to initiate a specific treatment using DM to counter/block the effects of this brain
chemical and its excessive 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.

Dr. Sakkubai Naidu, Principal Investigator, is initiating a double blinded placebo controlledclinical drug trial using dextromethorphan (DM) in Rett Syndrome (RTT), at the PediatricClinical Research Unit (PCRU) of the Johns Hopkins Hospital/Kennedy Krieger Institute, that issponsored by the FDA and Johns Hopkins Institute for Clinical and Translational Research(ICTR).

It has been shown that receptors for a certain brain chemical called glutamate, in particular theNMDA type, are increased in the brain of young RTT patients (<10 years of age). This chemicaland 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.We propose to initiate a specific treatment using DM to counter/block the effects of this brainchemical and its excessive receptors because of DM’s identified ability to block NMDAreceptors. DM is available for human consumption. Infants and children with respiratoryinfections and cough, as well as non-ketotic hyperglycinemia, are treated with DM, which hasbeen well tolerated.

The study will last for 3 months and will be limited to MECP2 mutation-positive males and females age one year – 9.99 years of age. This clinical trial, which is a placebo-controlled study, will randomize patients to the drug or placebo to determine the benefits of DM vs placebo on cognition, behavior, or seizures if present.

Your child will stay twice in the Pediatric Clinical Research Unit (PCRU) at Johns Hopkins ICTR. The first hospital stay will be for 3 days, before s/he starts the DM or placebo. The follow-up hospital stay will be 3 months after s/he starts taking DM or placebo. There will also be two interim follow up evaluations at 2 weeks and 1 month after s/he starts taking the DM or placebo consisting of a neurological evaluation, EKG, and blood work, which can take place at your local doctor’s office or at Johns Hopkins, and will be paid for by this study. Our research nurse or research associate will contact you at least weekly during the first month and at least monthly thereafter until the end of the 3-month study.

Participate in Research:

There is no financial compensation for participating in this research study. If you would like more information regarding the study’s procedures, risks, benefits, or other details, please contact our study coordinator, Barbara Ann Bradford, in the Neurogenetics Unit at the Kennedy Krieger Institute at bradford@kennedykrieger.org or (443)923-2778 or 1(800)873-3377If you or someone you know may be interested in participating in this research study, please download this informative flyer.