Placebo-Controlled Pilot Trial of Mecamylamine for Treatment of Autism Spectrum Disorders
Pervasive Developmental Disorder
|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|
- 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 ]
- 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 ]
|Study Start Date:||July 2007|
|Study Completion Date:||November 2012|
|Primary Completion Date:||November 2012 (Final data collection date for primary outcome measure)|
Experimental: Active Mecamylamine
There will be 12 children in this arm. These children will receive the active medication (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.
One pill is taken once daily.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00773812
|United States, Ohio|
|Columbus, Ohio, United States, 43210|
|Principal Investigator:||L E Arnold, M.D.||Ohio State University|