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 SpectrumDisorder (ASD) on the basis of clinical evaluation, confirmed with the AutismDiagnostic Interview Revised (ADI-R) and Autism Diagnostic Observation Schedule,Second Edition (ADOS-2) or Childhood Autism Rating Scale, Second Edition (CARS-2);

• males and females;

• intelligence quotient (IQ) of 40 and above;

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

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

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

• stable concomitant psychotropic medications or medications potentially affectingvasopressin 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 studyprocedures (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, andsignificant physical illness (e.g. serious liver disease, renal dysfunction, orcardiac pathology);

• clinically significant abnormal electrocardiogram reading;

• history of hypersensitivity to vasopressin, its analogs, or compoundingpreservatives (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 ofmedical history, neurologic history, and available tests for inborn errors ofmetabolism 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 ofvasopressin when used concurrently); 2) demeclocycline; norepinephrine; lithium;heparin; alcohol (all of which may decrease the antidiuretic effect of vasopressinwhen used concurrently); 3) ganglionic blocking agents including benzohexonium,chlorisondamine, pentamine (all of which may produce a marked increase insensitivity to the pressor effects of vasopressin);

• previous participation in a vasopressin clinical trial or current use ofvasopressin;

• current use of desmopressin (DDAVP) or oxytocin.