Phase I Nicotinic Agonist Treatment Trial for Autism

Study Overview
=================
Brief Summary
-----------------
This study plans to learn more about whether a nicotine-like investigational new drug, DMXB-A [3-(2,4-dimethoxybenzylidene) anabaseine] may have positive effects on mental focus and may help us to find new types of treatment for autism spectrum disorder. Subjects will come in for a screening visit and three more drug visits. There will be at least one week between each drug visit to allow the drug to be eliminated from your system completely. During each drug visit subjects will receive either a placebo (a capsule that looks like medicine but is not real), 75 mg of DMXB-A, or 150 mg of DMXB-A. During the drug day vital signs and well being will be monitored by the study physician. Subjects will complete some paper and pencil tasks to test memory, attention, speed, and problem solving, some rating scales and questionnaires, and the study team will record brain wave patterns with an electroencephalogram (EEG) .

Official Title
-----------------
Phase I Nicotinic Agonist Treatment Trial for Autism

Conditions
-----------------
Autism

Intervention / Treatment
-----------------
* Drug: DMXB-A 150 mg
* Other: placebo
* Drug: DMXB-A 75 mg


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: age 18-50 meet DSM-5 criteria for Autism Spectrum Disorder, Levels 1 or 2, as defined by Autism Diagnostic Observation Schedule non-smoking persons in good health Exclusion Criteria: persons with estimated verbal and nonverbal IQ < 70. abuse of other substances. Persons not sufficiently fluent in English to permit testing those with history of severe head injury Fragile X Syndrome Rett Syndrome

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 50 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: DMXB-A 75 mg<br>DMXB-A 75 mg dose followed by a second dose of 37.5 mg at 2 hours to maintain the blood level | Drug: DMXB-A 150 mg<br> <br> Other: placebo<br> <br> |
| Experimental: DMXB-A 150 mg<br>DMXB-A 150 mg followed by a second dose of 75 mg at 2 hours to maintain the blood level | Other: placebo<br> <br> Drug: DMXB-A 75 mg<br> <br> |
| Placebo Comparator: Sugar Pill<br>placebo comparator dose followed by a second placebo dose at 2 hours to maintain blind | Drug: DMXB-A 150 mg<br> <br> Drug: DMXB-A 75 mg<br> <br> |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Difference between drug and placebo on theTotal Scale Score on the repeatable Battery for the Assessment of neuropsychological Status at 2 hours after administration |  | 2 hours after drug administration | 
| Difference between drug and placebo on the CPT at 30 minutes after administration |  | 30 minutes after drug administration | 
| Difference between the P50 auditory evoked potential test/conditioning ratio 1 hour after administration |  | 1 hour after drug administration | 
| Difference between placebo and drug on the Social Responsiveness scale 2 hours after administration |  | 2 hours after drug administration | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Nicotinic agonist, Cognition, P50, Alpha 7, Autism