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

Autistic Disorder Clinical Trial

Official title:

Neuronal Nicotinic Receptor Modulation in the Treatment of Autism: A Pilot Trial of Mecamylamine

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00773812
• Other study ID #: 2007H0096
• Status: Completed
• Phase: Phase 1
• First received: October 15, 2008
• Last updated: January 11, 2017
• Start date: July 2007
• Est. completion date: November 2012

Study information

• Verified date: January 2017
• Source: Ohio State University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: November 2012
• Est. primary completion date: November 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 12 Years

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Factorial Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Autism Spectrum Disorder
• Autistic Disorder
• Child Development Disorders, Pervasive
• Developmental Disabilities
• Disease
• Pervasive Developmental Disorder

Intervention

Drug:
• 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.
• placebo
One pill is taken once daily.

Locations

Country	Name	City	State
United States	Nisonger Center	Columbus	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
Ohio State University	Autism Speaks

Country where clinical trial is conducted

United States, 

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.		Baseline, weeks 2, 4, 6, 8, 10,12, and 14.	No
Secondary	OSU Autism Rating Scale-DSM-IV		Baseline, week 6, 8, and 14.	No
Secondary	Repetitive Behavior Questionnaire		Baseline, week 2, 4, 6, 8, 10, 12, and 14	No
Secondary	Aberrant Behavior Checklist		Baseline, week 2, 4, 6, 8, 10, 12, and 14.	No
Secondary	Ohio Autism Clinical Impressions Scale		Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.	No
Secondary	Social Responsiveness Scale		Baseline, weeks 2, 4, 6, 8, 10, 12, and 14.	No
Secondary	Expressive Vocabulary test-Second Edition		Baseline and week 14	No
Secondary	Adverse Experience checklist and AE log		Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.	Yes
Secondary	Cognitive Battery		baseline, weeks 6, 8, and 14	No

External Links

•   Ohio State University Research Unit for Pediatric Pyschopharmocology Website