Placebo Controlled Trial of Dextromethorphan in Rett Syndrome
Condition: Rett Syndrome
MeSH terms: Rett Syndrome; Syndrome
Study type: Interventional
Phase: Phase 2

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.
    

Detailed description: 
      The study will last for 3 months and will be limited to MECP2 mutation-positive children, 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, for 3 days during each admission. The first hospital stay will be for 3 days, before
      she starts the DM or placebo. The follow-up 3-day hospital stay will be 3 months after she
      starts taking DM or placebo. There will also be two interim follow up evaluations at 2 weeks
      and 1 month after she 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.
    

Intervention name(s): dextromethorphan; placebo
Intervention type(s): Drug; Drug
Intervention description(s): The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study. The pharmacists will dispense the DM to the study participants.; The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist will dispense the placebo to the participants.
Other intervention name(s): Delsym
Intervention MeSH terms: Dextromethorphan
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Rett syndrome; RTT; MECP2; dextromethorphan; DM