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

October 15, 2008
April 7, 2014
July 2007
November 2012   (final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT00773812 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
Placebo-Controlled Pilot Trial of Mecamylamine for Treatment of Autism Spectrum Disorders
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.

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.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Factorial Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Autistic Disorder
  • Pervasive Developmental Disorder
  • 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
  • Drug: placebo
    One pill is taken once daily.
  • Experimental: Active Mecamylamine
    There will be 12 children in this arm. These children will receive the active medication (mecamylamine).
    Intervention: Drug: mecamylamine
  • Placebo Comparator: Placebo
    There will be 8 children in this arm. These children will receive placebo instead of the active medication.
    Intervention: Drug: placebo
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
20
November 2012
November 2012   (final data collection date for primary outcome measure)

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.
Both
4 Years to 12 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00773812
2007H0096
No
L. Eugene Arnold, Ohio State University
Ohio State University
Autism Speaks
Principal Investigator: L E Arnold, M.D. Ohio State University
Ohio State University
April 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP