Clinical trial NCT00773812: Placebo-Controlled Pilot Trial of Mecamylamine for Treatment of Autism Spectrum Disorders
Official title: Neuronal Nicotinic Receptor Modulation in the Treatment of Autism: A Pilot Trial of Mecamylamine
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Autistic Disorder
Pervasive Developmental Disorder
Keywords: Autistic Disorder
Autism
Mecamylamine
Pervasive Developmental Disorder-Not Otherwise Specified
Lead sponsor: Ohio State University
Sponsor class: OTHER
Collaborators: name: Autism Speaks
First posted: 2008-10-16
Enrollment: 20
Sex: ALL
Minimum age (years): 4
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: FACTORIAL
Primary purpose: TREATMENT

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

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: 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.
timeFrame: Baseline, weeks 2, 4, 6, 8, 10,12, and 14.
Secondary outcomes:
measure: OSU Autism Rating Scale-DSM-IV
timeFrame: Baseline, week 6, 8, and 14.
measure: Repetitive Behavior Questionnaire
timeFrame: Baseline, week 2, 4, 6, 8, 10, 12, and 14
measure: Aberrant Behavior Checklist
timeFrame: Baseline, week 2, 4, 6, 8, 10, 12, and 14.
measure: Ohio Autism Clinical Impressions Scale
timeFrame: Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.
measure: Social Responsiveness Scale
timeFrame: Baseline, weeks 2, 4, 6, 8, 10, 12, and 14.
measure: Expressive Vocabulary test-Second Edition
timeFrame: Baseline and week 14
measure: Adverse Experience checklist and AE log
timeFrame: Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.
measure: Cognitive Battery
timeFrame: baseline, weeks 6, 8, and 14
Other outcomes:
Locations:
city: Columbus
country: United States
facility: Nisonger Center
state: Ohio
Study officials:
affiliation: Ohio State University
name: L E Arnold, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Arnold LE, Aman MG, Hollway J, Hurt E, Bates B, Li X, Farmer C, Anand R, Thompson S, Ramadan Y, Williams C. Placebo-controlled pilot trial of mecamylamine for treatment of autism spectrum disorders. J Child Adolesc Psychopharmacol. 2012 Jun;22(3):198-205. doi: 10.1089/cap.2011.0056. Epub 2012 Apr 26.