Intranasal Vasopressin Treatment in Children With Autism

Inclusion Criteria:

- Medically healthy outpatients between 6 and 17 years of age;

- Diagnostic and Statistical Manual 5th edition (DSM-5) criteria for Autism Spectrum
Disorder (ASD) on the basis of clinical evaluation, confirmed with the Autism
Diagnostic Interview Revised (ADI-R) and Autism Diagnostic Observation Schedule,
Second Edition (ADOS-2);

- males and females;

- intelligence quotient (IQ) of 50 and above;

- rating of 4 or higher on the Social Communication domain of the Clinical Global
Impressions Severity (CGI-S);

- Social Responsiveness Scale-2 Total Score of 70 and above;

- care provider who can reliably bring participant to clinic visits, provide trustworthy
ratings, and interacts with participant on a regular basis;

- stable concomitant psychotropic medications or medications potentially affecting
vasopressin for at least 4 weeks (with the exception of fluoxetine, 6 weeks);

- no planned changes in psychosocial and biomedical interventions during the trial;

- willingness to provide blood samples and ability to participate in key study
procedures (i.e., diagnostic assessments and laboratory safety measurements).

Exclusion Criteria:

- DSM-5 diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder;

- regular nasal obstruction or nosebleeds;

- unstable medical conditions such as migraine, asthma attacks, or seizures, and
significant physical illness (e.g. serious liver disease, renal dysfunction, or
cardiac pathology);

- clinically significant abnormal electrocardiogram reading;

- history of hypersensitivity to vasopressin, its analogs, or compounding preservatives
(e.g., chlorobutanol);

- evidence of a genetic mutation known to cause ASD or intellectual disability (e.g.,
Fragile X Syndrome); or metabolic, or infectious etiology for ASD on the basis of
medical history, neurologic history, and available tests for inborn errors of
metabolism and chromosomal analysis;

- significant hearing or vision impairments;

- habitually drinks large volumes of water;

- pregnant or sexually active females not using a reliable method of contraception;

- current use of any medications known to interact with vasopressin including: 1)
carbamazepine (i.e., Tegretol); chlorpropamide; clofibrate; urea; fludrocortisone;
tricyclic antidepressants (all of which may potentiate the antidiuretic effect of
vasopressin when used concurrently); 2) demeclocycline; norepinephrine; lithium;
heparin; alcohol (all of which may decrease the antidiuretic effect of vasopressin
when used concurrently); 3) ganglionic blocking agents including benzohexonium,
chlorisondamine, pentamine (all of which may produce a marked increase in sensitivity
to the pressor effects of vasopressin);

- previous participation in a vasopressin clinical trial or current use of vasopressin;

- current use of desmopressin (DDAVP) or oxytocin.