Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

Status Completed

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT01520363
Study type	Interventional
Source	Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Contact
Status	Completed
Phase	Phase 2
Start date	March 2012
Completion date	October 26, 2016

View Details

See also
Status	Clinical Trial	Phase
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Enrolling by invitation	`NCT06139172` - Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities	N/A
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Not yet recruiting	`NCT04041713` - A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome	Phase 2
Completed	`NCT02705677` - Biobanking of Rett Syndrome and Related Disorders
Terminated	`NCT02790034` - Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms	Phase 2/Phase 3
Enrolling by invitation	`NCT03655223` - Early Check: Expanded Screening in Newborns
Recruiting	`NCT05932589` - Neurophysiologic Biomarkers in Rett Syndrome
Recruiting	`NCT04463316` - GROWing Up With Rare GENEtic Syndromes
Completed	`NCT04776746` - Open-Label Extension Study of Trofinetide for Rett Syndrome	Phase 3
Completed	`NCT04181723` - Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)	Phase 3
Enrolling by invitation	`NCT03836300` - Parent and Infant Inter(X)Action Intervention (PIXI)	N/A
Completed	`NCT04514549` - ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME
Completed	`NCT02738281` - Natural History of Rett Syndrome & Related Disorders
Terminated	`NCT02562820` - An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome	Phase 1
Completed	`NCT05687214` - Osteopathic Manipulative Treatment for Constipation in People With Rett Syndrome	N/A
Recruiting	`NCT06199700` - Esketamine for the Treatment of Rett Syndrome	Early Phase 1
Completed	`NCT03941444` - ANAVEX2-73 Study in Patients With Rett Syndrome	Phase 3
Recruiting	`NCT06346106` - The Diagnostic Experience of Male Rett Syndrome

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Placebo Controlled Trial of Dextromethorphan in Rett Syndrome — PCTDMRTT

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms