Placebo-Controlled Pilot Trial of Mecamylamine for Treatment of Autism Spectrum Disorders
The purpose of this study is to examine the safety and efficacy of mecamylamine for the core symptoms of autism.
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.