Autistic Disorder, Pervasive Developmental Disorder
Conditions
Keywords
Autistic Disorder, Autism, Mecamylamine, Pervasive Developmental Disorder-Not Otherwise Specified
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.
Interventions
DRUGmecamylamine
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.
DRUGplacebo
One pill is taken once daily.
Sponsors
Autism Speaks
Ohio State University
Study design
Allocation
RANDOMIZED
Intervention model
FACTORIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
4 Years to 12 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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. |
Secondary
| Measure | Time frame |
|---|---|
| Social Responsiveness Scale | Baseline, weeks 2, 4, 6, 8, 10, 12, and 14. |
| Expressive Vocabulary test-Second Edition | Baseline and week 14 |
| Adverse Experience checklist and AE log | Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14. |
| Ohio Autism Clinical Impressions Scale | Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14. |
| OSU Autism Rating Scale-DSM-IV | Baseline, week 6, 8, and 14. |
| Repetitive Behavior Questionnaire | Baseline, week 2, 4, 6, 8, 10, 12, and 14 |
| Aberrant Behavior Checklist | Baseline, week 2, 4, 6, 8, 10, 12, and 14. |
| Cognitive Battery | baseline, weeks 6, 8, and 14 |
Countries
United States
Outcome results
None listed