Phase 2 N=52 Randomized Quadruple-blind Treatment

Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

Rett Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT01520363 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Oct 2018

Primary outcome: Primary: Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention — 5.45; 6.61; 6.64; 6.57 score on a scale — p=.211

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: dextromethorphan (Drug); placebo (Drug)
Age: Pediatric · 1+ yrs
Sex: All
Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Primary completion: Oct 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention	5.45; 6.61; 6.64; 6.57	.211
PRIMARY Change in Mullen; Fine Motor Sub-scale Scores, Pre- and Post-Intervention	5.38; 7.00; 5.05; 7.04	.541
PRIMARY Change in Mullen; Receptive Language Subscale Scores, Pre- and Post-Intervention	6.45; 6.95; 6.68; 6.55	.736
PRIMARY Change in Mullen, Expressive Language Sub-scale Scores, Pre- and Post-Intervention	5.33; 6.73; 6.76; 7.29	.05
SECONDARY Change in VABS: Motor Skills Domain Scores, Pre- and Post-Intervention	9.86; 11.36; 9.64; 11.12	0.488
SECONDARY Change in VABS:Daily Living Skills Domain Scores, Pre- and Post-Intervention	21.64; 21.00; 20.95; 19.84	0.182
SECONDARY Change in VABS: Socialization Domain Scores, Pre- and Post-Intervention	24.14; 22.96; 24.05; 22.48	0.90
SECONDARY Change in VABS:Communication Domain Scores, Pre- and Post-Intervention	18.05; 18.08; 17.91; 17.96	0.836
SECONDARY Change in Ghuman-Folstein Screen for Social Interaction (SSI) Score, Pre- and Post-Intervention.	73.1; 71.3; 73.1; 68.0	0.42
SECONDARY Change in Rett Syndrome Behavior Questionnaire Score, Pre- and Post-Intervention	33.8; 33.3; 33.8; 33.3	<0.73

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.

Eligibility Criteria

Inclusion Criteria

males and females who have classic or atypical RTT with a proven mutation in the MECP2 gene;
subjects must be between one year - 10 years of age.

Exclusion Criteria

those without an established mutation in the MECP2 gene;
those with mutations in the MECP2 gene but who have had brain resection or surgical intervention; for example, tumor, hydrocephalus, severe head trauma; or, an associated severe medical illnesses such as vasculopathies, malignancies, diabetes, thyroid dysfunction, etc;
those on medications that could interact with DM, e.g. MAO inhibitors, SSRI, sibutramine etc. to avoid a serotonin syndrome; quinidine and drugs metabolized by the CYP450 isoform CYP2D6 (e.g. amiodarone, haloperidol, propafenone, thioridazine);
those proven to be intermediate or slow metabolizers of DM;
those with reported adverse reactions to DM;
those whose pregnancy test is positive;
those showing poor compliance with any aspect of the study;
foster children.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01520363). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.