Placebo Controlled Trial of Dextromethorphan in Rett Syndrome (PCTDMRTT)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01520363|
Recruitment Status : Completed
First Posted : January 27, 2012
Last Update Posted : January 29, 2018
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.
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.
|Condition or disease||Intervention/treatment||Phase|
|Rett Syndrome||Drug: dextromethorphan Drug: placebo||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||57 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Placebo Controlled Trial of Dextromethorphan in Rett Syndrome|
|Actual Study Start Date :||March 2012|
|Actual Primary Completion Date :||October 26, 2016|
|Actual Study Completion Date :||October 26, 2016|
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.
- Neuropsychological test (Mullen Scales of Early Learning). [ Time Frame: Initial evaluation and at the end of the 3 month trial ]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 ]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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01520363
|United States, Maryland|
|The Johns Hopkins Institute for Clinical and Translational Research|
|Baltimore, Maryland, United States, 21287|
|Principal Investigator:||Sakkubai R Naidu, MD||The Kennedy Krieger Institute and Johns Hopkins SOM|