Clinical Trial: Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

Brief 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 OOPD and the 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.

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 Summary:

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.

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

Current Primary Outcome: Neuropsychological test (Mullen Scales of Early Learning). [ Time Frame: Initial evaluation and at the end of the 3 month trial ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Behavior scale: Vineland Adaptive Behavior Scales (VABS) [ Time Frame: Initial evaluation and at the end of the 3 month trial will take 45 minutes per evaluation ]
  Vineland Adaptive Behavior Scales (VABS)will be performed by the Neuropsychologist.
• Behavior and temperament dysregulation measured by the Ghuman-Folstein Screen for Social Interaction (SSI);. [ Time Frame: Initial evaluation and at the end of the 3 month study. The test lasts 45 minutes ]
  Behavior and temperament dysregulation.

Original Secondary Outcome: Behavior scales: a) Vineland Adaptive Behavior Scales (VABS); b) Behavior and temperament dysregulation, measured by the Ghuman-Folstein Screen for Social Interaction (SSI); [ Time Frame: Initial evaluation and at the end of the 3 month trial ]

Information By: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Dates:
Date Received: January 25, 2012
Date Started: March 2012
Date Completion:
Last Updated: May 15, 2017
Last Verified: May 2017