Placebo Controlled Trial of Dextromethorphan in Rett Syndrome (PCTDMRTT)
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 OOPD 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.
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.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||Placebo Controlled Trial of Dextromethorphan in Rett Syndrome|
- Neuropsychological test (Mullen Scales of Early Learning). [ Time Frame: Initial evaluation and at the end of the 3 month trial ] [ Designated as safety issue: No ]The Neuropsychologist will assess cognitive status using the Mullen Scales of Early Learning (MULLEN), which will take approximately 2 - 2 1/2 hours during each of the two time points.
- 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 ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]Behavior and temperament dysregulation.
|Study Start Date:||March 2012|
|Estimated Study Completion Date:||March 2015|
|Estimated Primary Completion Date:||September 2014 (Final data collection date for primary outcome measure)|
Active Comparator: Study drug-dextromethorphan (DM)
MECP2 mutation positive subjects randomized to receive DM
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.
Other Name: Delsym
Placebo Comparator: Placebo group
MECP2 positive subjects randomized to the placebo compound
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 Name: Placebo
The study will last for 3 months and will be limited to MECP2 mutation-positive children, 2 years - 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.
|Contact: BarbaraAnn Bradfordfirstname.lastname@example.org|
|Contact: Marybeth Yablonski, RN, MSemail@example.com|
|United States, Maryland|
|The Johns Hopkins Institute for Clinical and Translational Research||Recruiting|
|Baltimore, Maryland, United States, 21287|
|Principal Investigator:||Sakkubai R Naidu, MD||The Kennedy Krieger Institute and Johns Hopkins SOM|