Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01520363
• Date of registration: 25/01/2012
• Prospective Registration: Yes
• Primary sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
• Public title: Placebo Controlled Trial of Dextromethorphan in Rett Syndrome PCTDMRTT
• Scientific title: Placebo Controlled Trial of Dextromethorphan in Rett Syndrome
• Date of first enrolment: March 2012
• Target sample size: 57
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01520363
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Sakkubai R Naidu, MD
• Affiliation: The Kennedy Krieger Institute and Johns Hopkins SOM

Key inclusion & exclusion 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.



Age minimum: 1 Year
Age maximum: 10 Years
Gender: All

Health Condition(s) or Problem(s) studied

Rett Syndrome

Intervention(s)

Drug: dextromethorphan

Drug: placebo

Primary Outcome(s)

Change in Mullen; Receptive Language Subscale Scores, Pre- and Post-Intervention [Time Frame: Baseline and 3 months]

Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention [Time Frame: Initial evaluation and at the end of the 3 month trial]

Change in Mullen; Fine Motor Sub-scale Scores, Pre- and Post-Intervention [Time Frame: Baseline and 3 months]

Change in Mullen, Expressive Language Sub-scale Scores, Pre- and Post-Intervention [Time Frame: Baseline and 3 months]

Secondary Outcome(s)

Change in Rett Syndrome Behavior Questionnaire Score, Pre- and Post-Intervention [Time Frame: Initial evaluation and at the end of the 3 month study]

Change in VABS:Communication Domain Scores, Pre- and Post-Intervention [Time Frame: Baseline and at the end of the 3 month trial]

Change in VABS: Motor Skills Domain Scores, Pre- and Post-Intervention [Time Frame: Baseline evaluation and at the end of the 3 month study]

Change in VABS:Daily Living Skills Domain Scores, Pre- and Post-Intervention [Time Frame: Baseline and at the end of the 3 month trial]

Change in Ghuman-Folstein Screen for Social Interaction (SSI) Score, Pre- and Post-Intervention. [Time Frame: Initial evaluation and at the end of the 3 month study. The test lasts 45 minutes]

Change in VABS: Socialization Domain Scores, Pre- and Post-Intervention [Time Frame: Baseline and at the end of the 3 month trial]

Secondary ID(s)

FD-004247-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

The Johns Hopkins Institute for Clinical and Translational Research (ICTR)

Results

• Results available: Yes
• Date Posted: 23/10/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01520363