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)/NBRU. This study is still actively looking for participants. Canadian individuals with Rett syndrome are welcome to apply.
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.
If you would like more information regarding the study’s Procedures, Risks, Benefits, or other details, please see the attached documents. You can contact the study coordinator, Barbara Ann Bradford, in the Neurogenetics Unit at the Kennedy Krieger Institute (firstname.lastname@example.org) or at phone # 443-923-2778 or # 1-800-873-3377. You can also contact Dr. Melissa Carter by phone at (416) 813-7654 x228986 to discuss the 2 interim assessments that could be completed at Sick Kids if your local provider is not able.