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

This study has been completed.
Sponsor:
Collaborator:
Autism Speaks
Information provided by (Responsible Party):
L. Eugene Arnold, Ohio State University
ClinicalTrials.gov Identifier:
NCT00773812
First received: October 15, 2008
Last updated: April 7, 2014
Last verified: April 2014
  Purpose

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


Condition Intervention Phase
Autistic Disorder
Pervasive Developmental Disorder
Drug: mecamylamine
Drug: placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Factorial Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Neuronal Nicotinic Receptor Modulation in the Treatment of Autism: A Pilot Trial of Mecamylamine

Resource links provided by NLM:


Further study details as provided by Ohio State University:

Primary Outcome 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. ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • OSU Autism Rating Scale-DSM-IV [ Time Frame: Baseline, week 6, 8, and 14. ] [ Designated as safety issue: No ]
  • Repetitive Behavior Questionnaire [ Time Frame: Baseline, week 2, 4, 6, 8, 10, 12, and 14 ] [ Designated as safety issue: No ]
  • Aberrant Behavior Checklist [ Time Frame: Baseline, week 2, 4, 6, 8, 10, 12, and 14. ] [ Designated as safety issue: No ]
  • Ohio Autism Clinical Impressions Scale [ Time Frame: Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14. ] [ Designated as safety issue: No ]
  • Social Responsiveness Scale [ Time Frame: Baseline, weeks 2, 4, 6, 8, 10, 12, and 14. ] [ Designated as safety issue: No ]
  • Expressive Vocabulary test-Second Edition [ Time Frame: Baseline and week 14 ] [ Designated as safety issue: No ]
  • Adverse Experience checklist and AE log [ Time Frame: Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14. ] [ Designated as safety issue: Yes ]
  • Cognitive Battery [ Time Frame: baseline, weeks 6, 8, and 14 ] [ Designated as safety issue: No ]

Enrollment: 20
Study Start Date: July 2007
Study Completion Date: November 2012
Primary Completion Date: November 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: 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.

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. There was no advantage of active drug over placebo.

  Eligibility

Ages Eligible for Study:   4 Years to 12 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00773812

Locations
United States, Ohio
Nisonger Center
Columbus, Ohio, United States, 43210
Sponsors and Collaborators
Ohio State University
Autism Speaks
Investigators
Principal Investigator: L E Arnold, M.D. Ohio State University
  More Information

Additional Information:
Publications:
Responsible Party: L. Eugene Arnold, Interim Director of Nisonger Center, Ohio State University
ClinicalTrials.gov Identifier: NCT00773812     History of Changes
Other Study ID Numbers: 2007H0096
Study First Received: October 15, 2008
Last Updated: April 7, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Ohio State University:
Autistic Disorder
Autism
Mecamylamine
Pervasive Developmental Disorder-Not Otherwise Specified

Additional relevant MeSH terms:
Autistic Disorder
Developmental Disabilities
Child Development Disorders, Pervasive
Mental Disorders Diagnosed in Childhood
Mental Disorders
Mecamylamine
Antihypertensive Agents
Cardiovascular Agents
Therapeutic Uses
Pharmacologic Actions
Ganglionic Blockers
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Nicotinic Antagonists
Cholinergic Antagonists
Cholinergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on August 20, 2014