Placebo-Controlled Pilot Trial of Mecamylamine for Treatment of Autism Spectrum Disorders

Overview

The purpose of this study is to examine the safety and efficacy of mecamylamine for the core symptoms of autism.

Full Title of Study: “Neuronal Nicotinic Receptor Modulation in the Treatment of Autism: A Pilot Trial of Mecamylamine”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Factorial Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: November 2012

Detailed Description

This is a placebo-controlled pilot clinical trial of a nicotinic agent based on evidence that a disturbance occurs in nicotinic cholinergic transmission in the brain of autistic children. This pharmacotherapeutic trial will be conducted as a direct result of findings of a nicotinic receptor abnormality in autopsied brain tissue from individuals who suffered from autism. A pharmacological agent, mecamylamine, with specificity for the nicotinic receptors implicated in autism, will be tested in children with autism spectrum disorder. Twenty children who meet inclusion/exclusion criteria will be randomized in a 2:3 ratio to 14 weeks of either matched placebo (n=8) or mecamylamine (n=12). Children randomized to placebo who do not respond will be given the opportunity to enroll in a ten week open label extension. The trial has been completed and published in J. of Child & Adolescent Psychopharmacology.

Interventions

  • Drug: mecamylamine
    • The dosage is titrated at 6 weeks of .5mg, 2 weeks of 2.5 mg, and 6 weeks of 5.0 mg. One pill is taken once daily.
  • Drug: placebo
    • One pill is taken once daily.

Arms, Groups and Cohorts

  • Experimental: Active Mecamylamine
    • There will be 12 children in this arm. These children will receive the active medication (mecamylamine).
  • Placebo Comparator: Placebo
    • There will be 8 children in this arm. These children will receive placebo instead of the active medication.

Clinical Trial Outcome Measures

Primary Measures

  • The primary measure will be a global composite derived by calculating the z scores on each measure other than CGI from published norms (or from the baseline descriptives, then averaging the z scores of the various outcome measures for each individual.
    • Time Frame: Baseline, weeks 2, 4, 6, 8, 10,12, and 14.

Secondary Measures

  • OSU Autism Rating Scale-DSM-IV
    • Time Frame: Baseline, week 6, 8, and 14.
  • Repetitive Behavior Questionnaire
    • Time Frame: Baseline, week 2, 4, 6, 8, 10, 12, and 14
  • Aberrant Behavior Checklist
    • Time Frame: Baseline, week 2, 4, 6, 8, 10, 12, and 14.
  • Ohio Autism Clinical Impressions Scale
    • Time Frame: Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.
  • Social Responsiveness Scale
    • Time Frame: Baseline, weeks 2, 4, 6, 8, 10, 12, and 14.
  • Expressive Vocabulary test-Second Edition
    • Time Frame: Baseline and week 14
  • Adverse Experience checklist and AE log
    • Time Frame: Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.
  • Cognitive Battery
    • Time Frame: baseline, weeks 6, 8, and 14

Participating in This Clinical Trial

Inclusion Criteria

  • Autistic Disorder or Pervasive Developmental Disorder-Not Otherwise Specified – Age 4-12 – General good health – IQ of >=36 or mental age of >=18 months – Parent/caregiver willingness to accompany child to clinic and monitor for side effects Exclusion Criteria:

  • Unstable Seizure Disorder – Psychoactive medication in the process of adjustment – Antipsychotic medication in previous 3 months before baseline – Systemic corticoids (inhalers allowed) – Planned beginning of major behavioral intervention in 3 months of study or the 2 months prior to the study.

Gender Eligibility: All

Minimum Age: 4 Years

Maximum Age: 12 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Ohio State University
  • Collaborator
    • Autism Speaks
  • Provider of Information About this Clinical Study
    • Principal Investigator: L. Eugene Arnold, Interim Director of Nisonger Center – Ohio State University
  • Overall Official(s)
    • L E Arnold, M.D., Principal Investigator, Ohio State University

Citations Reporting on Results

Arnold LE, Aman MG, Hollway J, Hurt E, Bates B, Li X, Farmer C, Anand R, Thompson S, Ramadan Y, Williams C. Placebo-controlled pilot trial of mecamylamine for treatment of autism spectrum disorders. J Child Adolesc Psychopharmacol. 2012 Jun;22(3):198-205. doi: 10.1089/cap.2011.0056. Epub 2012 Apr 26.

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