Intranasal Vasopressin Treatment in Children With Autism

NCT03204786 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: IRB-399721R01HD091972
• Study Type: interventional
• Target: 157
• Locations: 1 country
• Sites: 1

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

Keywords

Autism Spectrum Disorder (ASD); Autism

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Parent Rated Social Responsiveness Scale, Second Edition (SRS-2) Total Scores During Treatment.

  Social Responsiveness Scale, 2nd Edition (SRS) scores measure social abilities with lower scores meaning better social abilities. The SRS-2 is reported as a total score (T-Score Range: 37 to above 90), and the Diagnostic and Statistical Manual of Mental Disorders (DSM)-5-compatible Social Communication and Interaction (SCI) score (T-Score Range: 36 to above 90) and Restricted Interests and Repetitive Behavior (RRB) score (T-Score range: 41 to above 90). A T-score of 50 indicates the population mean with a standard deviation of 10. Higher scores correspond to greater symptom levels (≤ 59: within normal limits; 60-65: mild range; 66-75: moderate range; ≥ 76: severe range). Change is reported as 4-week minus the baseline score, and 8-week minus the 4-week score. For this outcome, the baseline score is the average of the screening visit and baseline visit (average approximately 2 to 3 weeks after the screening visit).

  baseline, 4-week, 8-week

Secondary Outcomes (29)

• Change From Baseline in Clinical Global Impression (CGI) Scores During Treatment.

  baseline; 4-week; 8-week

• Change From Baseline on Reading the Mind in the Eyes Test (RMET) During Treatment.

  baseline; 4-week; 8-week

• Change From Baseline on the Facial Emotion Recognition Test During Treatment.

  baseline; 4-week; 8-week

• Change From Baseline in Parent Rated Repetitive Behavior Scale Revised (RBS-R) Scores During Treatment.

  baseline; 4-week; 8-week

• Change From Baseline in Parent Rated Spence Children's Anxiety Scale (SCAS) During Treatment.

  baseline; 4-week; 8-week

Other Outcomes (18)

• Change From Baseline in Vineland Adaptive Behavior Scales, Third Edition (VABS-3) - Social Skills and Relationships Domain During Treatment.

  4-week; 8-week

• Change From Baseline in Parent Rated Pediatric Quality of Life (PedsQL) Inventory Scores During Treatment.

  4-week; 8-week

• Change From Baseline on Eye Gaze Assessment (Eye Tracking) During Treatment.

  4-week; 8-week

Study Arms (3)

Vasopressin-Vasopressin

EXPERIMENTAL

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

Drug: Vasopressin (USP) Injectable Solution [Vasostrict]

Placebo-Vasopressin

EXPERIMENTAL

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

Drug: Vasopressin (USP) Injectable Solution [Vasostrict]; Drug: Placebo

Placebo-Placebo

PLACEBO COMPARATOR

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

Drug: Vasopressin (USP) Injectable Solution [Vasostrict]; Drug: Placebo

Interventions

Vasopressin (USP) Injectable Solution [Vasostrict] DRUG

Nasal Spray

Placebo-Placebo; Placebo-Vasopressin; Vasopressin-Vasopressin

Placebo DRUG

Placebo Nasal Spray

Placebo-Placebo; Placebo-Vasopressin

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Stanford University: lead
• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): collaborator

Study Sites (1)

Stanford University

Stanford, California, 94305-5719, United States

Location

MeSH Terms

Conditions

Autistic Disorder; Autism Spectrum Disorder

Interventions

Vasopressins; Arginine Vasopressin

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Posterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Neuropeptides; Peptides; Amino Acids, Peptides, and Proteins; Oligopeptides; Nerve Tissue Proteins; Proteins

Results Point of Contact

• Title: Antonio Hardan, MD
• Organization: Stanford University

autismdd@stanford.edu

(650)736-1235

Study Officials

• Antonio Y. Hardan, M.D.

  Stanford University

  PRINCIPAL INVESTIGATOR

• Karen J. Parker, Ph.D.

  Stanford University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: June 28, 2017
• First Posted: July 2, 2017
• Study Start: February 20, 2018
• Primary Completion: March 18, 2024
• Study Completion: March 18, 2024
• Last Updated: September 19, 2025
• Results First Posted: September 19, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will share

Locations

US (1)