Placebo-Controlled Pilot Trial of Mecamylamine for Treatment of Autism Spectrum Disorders

The Ohio State University

Status and phase: Completed; Phase 1

Conditions: Pervasive Developmental Disorder; Autistic Disorder

Treatments: Drug: mecamylamine; Drug: placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT00773812

2007H0096

Details and patient eligibility

About

The purpose of this study is to examine the safety and efficacy of mecamylamine for the core symptoms of autism.

Full description

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.

Enrollment: 20 patients

Sex: All

Ages: 4 to 12 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Factorial Assignment

Masking: Quadruple Blind

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20 participants in 2 patient groups, including a placebo group

Active Mecamylamine

Experimental group

Description:

There will be 12 children in this arm. These children will receive the active medication (mecamylamine).

Treatment:

Drug: mecamylamine

Placebo

Placebo Comparator group

Description:

There will be 8 children in this arm. These children will receive placebo instead of the active medication.

Treatment:

Drug: placebo