DEXTROMETORPHAN IN RETT SYNDROME

• Register: EUCTR
• Last refreshed on: 2 October 2017
• Main ID: EUCTR2008-005571-10-NO
• Date of registration: 09/01/2009
• Prospective Registration: Yes
• Primary sponsor: Kennedy Krieger Inst. Johns Hopkins Medicine
• Public title: DEXTROMETORPHAN IN RETT SYNDROME
• Scientific title: DEXTROMETORPHAN IN RETT SYNDROME
• Target sample size: 90
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-005571-10
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Norway

Key inclusion & exclusion criteria

Inclusion criteria:
1) Those who have classic or atypical RTT with a proven mutation in the MeCP2 gene

2) Those with documented EEG evidence of spike activity (epileptic EEG) who may or may not have clinical seizures

3) Subjects must be between 2years -14.99 years of age. Once safety is established in the 2-4.99-year-old subjects, we will then consider reducing the inclusion age to below 2 years of age.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1) Those with clinical detectable Rett syndrome or similar clinical picture but without an established mutation in the MeCP2 gene

2) Those with clinical detectable Rett syndrome or similar clinical picture but who do not have EEG evidence of spike activity

3) 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

4) 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, propfenone, thioridazine)

5) Those proven to be intermediate or slow metabolizers of DM

6) Those with reported adverse reactions to DM

7) Those whose pregnancy test is positive

8) Those showing poor compliance with any aspect of the study

9) foster children.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: no

Health Condition(s) or Problem(s) studied

Rett syndrome (RTT) is a neurological disorder with devastating consequences on the brain. It is characterized by stagnation of development followed by regression, both occurring between age 6 months and 3 years. The clinical picture is dominated by cognitive impairment, loss of communication skills, purposeful hand movements, hand stereotypies, progressive deceleration of head growth, and abnormal locomotion. RTT syndrome presently has no effective therapy other than pallitative care

Intervention(s)

Product Name: Dextromethorphan
Pharmaceutical Form: Capsule, soft

Primary Outcome(s)

Main Objective: The main objective is to test the efficiacy of Dextromethorphan (DM) in RTT to block the disproportionate numbers of glutamate/NMDA receptors in the brain, to alleviate symptoms.

Primary end point(s):

Secondary Objective: Secondary objectives of the trial will additionally be a mode of therapy for other epileptic conditons that are associated with glutamatergic excitotoxicity.

Secondary ID(s)

03-08-21-12