Intranasal Vasopressin Treatment in Children With Autism

NCT ID: NCT03204786

Last Updated: 2025-09-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 157 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-02-20
• Study Completion Date: 2024-03-18

Brief Summary

The purpose of this clinical trial is to investigate the effectiveness of vasopressin nasal spray for treating symptoms associated with autism. Vasopressin is a hormone that is produced naturally within the body and has been implicated in regulating social behaviors. It has been proposed that administration of the hormone may also help improve social functioning in individuals with autism.

Conditions

Autism; Autism Spectrum Disorder; ASD

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Vasopressin-Vasopressin

8 weeks of vasopressin nasal spray (16 international units twice daily)

Group Type: EXPERIMENTAL

Vasopressin (USP) Injectable Solution [Vasostrict]

Intervention Type: DRUG

Nasal Spray

Placebo-Vasopressin

4 weeks of placebo nasal spray followed by 4 weeks of vasopressin nasal spray (16 international units twice daily)

Group Type: EXPERIMENTAL

Vasopressin (USP) Injectable Solution [Vasostrict]

Intervention Type: DRUG

Nasal Spray

Placebo

Intervention Type: DRUG

Placebo Nasal Spray

Placebo-Placebo

8 weeks of placebo nasal spray; followed by a 4 week open-label extension of vasopressin nasal spray (16 international units twice daily)

Group Type: PLACEBO_COMPARATOR

Vasopressin (USP) Injectable Solution [Vasostrict]

Intervention Type: DRUG

Nasal Spray

Placebo

Intervention Type: DRUG

Placebo Nasal Spray

Interventions

Vasopressin (USP) Injectable Solution [Vasostrict]

Nasal Spray

Intervention Type: DRUG

Placebo

Placebo Nasal Spray

Intervention Type: DRUG

Eligibility Criteria

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) 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 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.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

Sponsor Role: collaborator

Stanford University

OTHER

Sponsor Role: lead

Responsible Party

Antonio Hardan

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Antonio Y. Hardan, M.D.

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Karen J. Parker, Ph.D.

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Stanford University

Stanford, California, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

1R01HD091972

Identifier Type: NIH

Identifier Source: secondary_id

View Link

IRB-39972

Identifier Type: -

Identifier Source: org_study_id