Trial of Dextromethorphan in Rett Syndrome

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Brief Title

Trial of Dextromethorphan in Rett Syndrome

Official Title

Trial of Dextromethorphan in Rett Syndrome

Brief Summary

      Increased brain glutamate and its N-methyl-D-aspartate (NMDA) receptors found in the brain of
      younger Rett syndrome (RTT) patients cause toxic damage to neurons (the brain's nerve cells),
      and contributing to EEG spikes. Dextromethorphan (DM) acts by blocking NMDA/glutamate
      receptors. This study is being done to determine if DM will prevent the harmful
      over-stimulation of the neurons thereby reducing EEG spike activity. Treatment with DM
      consists of one of 3 different doses (0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day),
      and aims to find out which dose if any will help improve EEG abnormalities, behavior,
      cognition, and reduce seizures, as well as improve breathing abnormalities, motor
      capabilities, bone density, and GI dysfunction.

      The study will include 90 females and males with RTT, 2 years-14.99 years of age, with a
      mutation in the methyl CpG binding protein 2 (MECP2) gene, and spikes on EEG, with or without
      clinical seizures.
    

Detailed Description

      Patients meeting eligibility criteria(mutation +ve and having EEG spikes), will be admitted
      to the Pediatric Clinical Research Unit at Johns Hopkins Hospital and will have
      pharmacokinetics of DM determined to establish that they are rapid metabolizers of the drug.
      The baseline studies on initial admission include neurological, neuropsychology,EEG,
      gastroenterology, Occupational and Physical therapy evaluations. If the subject is a rapid
      metabolizer they will be randomized to one of the three drug doses. They are contacted by
      telephone, weekly in the first month, and monthly thereafter. They will be examined by a
      neurologist at 2 weeks,1 month, and 3 months during the drug trial. At each of these visits
      they will also be monitored for changes in complete blood count (CBC), electrolytes, and EKG.
      At the end of the 6 month drug trial the patients will be readmitted to Johns Hopkins
      Hospital when all baseline studies are repeated. Cost of travel, hospitalization and interim
      tests are free to participants.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Difference in EEG Spike Counts at Six Months Compared to Baseline for Each Treatment Arm.

Secondary Outcome

 Improvement in Receptive Language as Measured by the Mullen Scale.

Condition

Rett Syndrome

Intervention

Dextromethorphan

Study Arms / Comparison Groups

 DM1( 0.25 mg/kg /day)
Description:  Dextromethorphan 0.25 mg/kg per day

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

38

Start Date

August 2004

Completion Date

June 2010

Primary Completion Date

April 2010

Eligibility 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 who may or may not have clinical
             seizures;

          3. subjects must be between 2years -14.99 years of age.

        Exclusion Criteria:

          1. those without an established mutation in the MeCP2 gene;

          2. those 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. monoamine oxidase (MAO)
             inhibitors, selective serotonin reuptake inhibitor (SSRI), sibutramine etc. to avoid a
             serotonin syndrome; quinidine and drugs metabolized by the Cytochrome P450 (CYP450)
             isoform cytochrome P450 2D6 (CYP2D6) (e.g. amiodarone, haloperidol, propafenone,
             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; and,

          8. those showing poor compliance with any aspect of the study;

          9. foster children
      

Gender

All

Ages

2 Years - 15 Years

Accepts Healthy Volunteers

No

Contacts

SakkuBai Naidu, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00593957

Organization ID

FD2408


Responsible Party

Principal Investigator

Study Sponsor

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.


Study Sponsor

SakkuBai Naidu, MD, Principal Investigator, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.


Verification Date

July 2013