Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome

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Brief Title

Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome

Official Title

Pathogenesis of Rett Syndrome: Natural History and Treatment

Brief Summary

      Rett syndrome (RTT) is a disorder in which the nervous system does not develop properly. RTT
      generally affects girls, but there are some boys who have been diagnosed with RTT. Symptoms
      of RTT include small brain size, poor language skills, repetitive hand movements, and
      seizures. This study will evaluate the effectiveness of two drugs in treating the symptoms of
      RTT.
    

Detailed Description

      RTT is a neurodevelopmental disorder characterized by apparently normal early development
      followed by loss of purposeful hand use, distinctive hand stereotypies, slowed brain growth,
      loss of language, respiratory irregularities, GI disturbances, gait abnormalities, seizures,
      and mental retardation. These symptoms appear between ages 6 and 18 months (stage 2 of the
      disease) following apparently normal development (stage 1). Subsequently, there is gradual
      stabilization of severe mental retardation and motor compromise (stage 3). The majority (70%
      to 80%) of patients demonstrate mutations in the methyl-CpG-binding-protein-2 (MeCP2) gene, a
      transcription repressor located on chromosome Xq28. The disorder predominantly affects
      females, but a few males with mutations in MeCP2 have been identified, even though many of
      them do not have the classic symptoms recognized in females.

      Recent studies demonstrate increased brain N-methyl-D-aspartate (NMDA) receptors in stages 2
      and 3 of the disease. This age-specific increase in glutamate levels and their receptors
      contribute to brain damage. This first study will examine the effectiveness of
      dextromethorphan, an NMDA receptor antagonist, to ameliorate symptoms. Participants will be
      randomized to receive one of three doses of dextromethorphan. All participants will be
      admitted to the hospital for three days at the beginning of the study. During the
      hospitalization, participants will undergo physical exam, Dexascan, MRI, EEG, behavioral
      assessment, laboratory testing, and neuropsychological evaluations. Six months after baseline
      assessment, participants will be rehospitalized for 3 days for similar assessments.

      Reduction in choline acetyltransferase activity in RTT patients may also contribute to
      disturbed cortical development and psychomotor retardation in RTT. Therefore, the second part
      of the study will evaluate the effect of donepezil hydrochloride, an inhibitor of
      acetylcholine-esterase, on acetylcholine levels. This portion of the study will not begin
      until pharmacokinetic data for donepezil in children is available.
    

Study Phase

Phase 3

Study Type

Interventional




Condition

Rett Syndrome

Intervention

dextromethorphan


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

90

Start Date

September 2004

Completion Date

June 2008


Eligibility Criteria

        Inclusion Criteria

          -  Diagnosis of Rett syndrome

          -  Mutation in MeCP2 gene

          -  Typical EEG abnormalities (disorganized background, frontal central spikes, rhythmic
             theta)

        Exclusion Criteria

          -  Features of Rett syndrome with absence of MeCP2 mutation

          -  Non-specific EEG changes
      

Gender

All

Ages

1 Year - 15 Years

Accepts Healthy Volunteers

No

Contacts

SakkuBai R. Naidu, MD, 443-923-2778, 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00069550

Organization ID

HD024448

Secondary IDs

5P01HD024448


Study Sponsor

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)


Study Sponsor

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


Verification Date

December 2004