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.

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.