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

Phase 1

Completed

• Conditions: Pervasive Developmental Disorder; Autistic Disorder
• Interventions: Drug: placebo; Drug: mecamylamine

• Registration Number: NCT00773812
• Lead Sponsor: Ohio State University

Brief Summary

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

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 Timeline

• Study Start: 2007-07
• Study First Submitted: 2008-10-15
• Study First Submitted QC: 2008-10-15
• Study First Posted: 2008-10-16
• Primary Completion: 2012-11
• Study Completion: 2012-11
• Status Verified: 2017-01
• Last Update Submitted: 2017-01-11
• Last Update Posted: 2017-01-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: FACTORIAL

Arm && Interventions

Group	Intervention	Description
Placebo	placebo	There will be 8 children in this arm. These children will receive placebo instead of the active medication.
Active Mecamylamine	mecamylamine	There will be 12 children in this arm. These children will receive the active medication (mecamylamine).

Primary Outcome Measures

Name	Time	Method
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.	Baseline, weeks 2, 4, 6, 8, 10,12, and 14.

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Nisonger Center; 🇺🇸 Columbus, Ohio, United States