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

January 11, 2017 updated by: L. Eugene Arnold, Ohio State University

Neuronal Nicotinic Receptor Modulation in the Treatment of Autism: A Pilot Trial of Mecamylamine

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

20

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Nisonger Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years to 12 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Factorial Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Active Mecamylamine
There will be 12 children in this arm. These children will receive the active medication (mecamylamine).
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.
Other Names:
  • Inversine
Placebo Comparator: Placebo
There will be 8 children in this arm. These children will receive placebo instead of the active medication.
Drug: placebo
One pill is taken once daily.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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.
Baseline, weeks 2, 4, 6, 8, 10,12, and 14.

Secondary Outcome Measures

Outcome Measure
Time Frame
OSU Autism Rating Scale-DSM-IV
Time Frame: Baseline, week 6, 8, and 14.
Baseline, week 6, 8, and 14.
Repetitive Behavior Questionnaire
Time Frame: Baseline, week 2, 4, 6, 8, 10, 12, and 14
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.
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.
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.
Baseline, weeks 2, 4, 6, 8, 10, 12, and 14.
Expressive Vocabulary test-Second Edition
Time Frame: Baseline and week 14
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.
Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.
Cognitive Battery
Time Frame: baseline, weeks 6, 8, and 14
baseline, weeks 6, 8, and 14

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ohio State University

Collaborators

Autism Speaks

Investigators

  • Principal Investigator: L E Arnold, M.D., Ohio State University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2007

Primary Completion (Actual)

November 1, 2012

Study Completion (Actual)

November 1, 2012

Study Registration Dates

First Submitted

October 15, 2008

First Submitted That Met QC Criteria

October 15, 2008

First Posted (Estimate)

October 16, 2008

Study Record Updates

Last Update Posted (Estimate)

January 13, 2017

Last Update Submitted That Met QC Criteria

January 11, 2017

Last Verified

January 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2007H0096

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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