Title
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
Phase
Phase 1Lead Sponsor
Ohio State UniversityStudy Type
InterventionalStatus
Completed No Results PostedIndication/Condition
Autistic Disorder Pervasive Developmental DisorderIntervention/Treatment
mecamylamine ...Study Participants
20The 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.
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.
One pill is taken once daily.
There will be 12 children in this arm. These children will receive the active medication (mecamylamine).
There will be 8 children in this arm. These children will receive placebo instead of the active medication.
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.
