AzTrials
AzTrials

AutismPhase 2/3NCT03204786

Intranasal Vasopressin Treatment in Children With Autism

A Phase 2/3 study testing Vasopressin (USP) Injectable Solution [Vasostrict] in people with Autism and Autism Spectrum Disorder, run by Stanford University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 6 to 17 years
Conditions the study is for
Autism, Autism Spectrum Disorder, ASD
What is being tested?
Vasopressin (USP) Injectable Solution [Vasostrict]
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
157 participants
Where
1 siteUnited States
Study dates
20 Feb 2018to 18 Mar 2024
Phase
Phase 2/3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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;
Show all 10Show fewer
  • 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).

Who cannot join

  • 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;
Show all 12Show fewer
  • 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.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Stanford University

    Stanford, California, 94305-5719

Other trials in United States

About this study

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.

Being tested
Vasopressin (USP) Injectable Solution [Vasostrict], Placebo
Keywords
Autism Spectrum Disorder (ASD), Autism
Medical subject headings
Autistic Disorder, Autism Spectrum Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Vasopressin (USP) Injectable Solution [Vasostrict]Drug

    Nasal Spray

  • PlaceboDrug

    Placebo Nasal Spray

Study groups in NCT03204786
Study groupRoleWhat they receive
Vasopressin-VasopressinExperimental8 weeks of vasopressin nasal spray (16 international units twice daily)
Receives: Drug: Vasopressin (USP) Injectable Solution [Vasostrict]
Placebo-VasopressinExperimental4 weeks of placebo nasal spray followed by 4 weeks of vasopressin nasal spray (16 international units twice daily)
Receives: Drug: Vasopressin (USP) Injectable Solution [Vasostrict]; Drug: Placebo
Placebo-PlaceboPlacebo comparator8 weeks of placebo nasal spray; followed by a 4 week open-label extension of vasopressin nasal spray (16 international units twice daily)
Receives: Drug: Vasopressin (USP) Injectable Solution [Vasostrict]; Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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).

    Measured: baseline, 4-week, 8-week

Secondary outcomes (28)
  1. Change From Baseline in Clinical Global Impression (CGI) Scores During Treatment.

    Clinician assessment of CGI severity (CGI-S) and CGI improvement (CGI-I) scores.

    • Higher scores on the CGI-S mean greater social and communication deficits (range: 1 to 7).
    • Lower scores on the CGI-I correspond to greater improvement in the areas assessed in the CGI-S, and higher scores correspond to worsening (range: 1 to 7). There is no baseline score for the CGI-I, it represents the clinician's subjective assessment of change.

    Measured: baseline; 4-week; 8-week

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

    Score range: 0 to 28; higher scores mean better ability to read emotions and lower scores mean worse ability to read emotions.

    Measured: baseline; 4-week; 8-week

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

    Score range: 0 to 42; higher scores mean better facial emotion recognition abilities. Lower scores mean worse facial emotion recognition abilities.

    Measured: baseline; 4-week; 8-week

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

    Score range: 0 to 129; higher scores on the RBS-R mean higher levels of repetitive and restricted behaviors.

    Measured: baseline; 4-week; 8-week

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

    Scale measuring severity of anxiety symptoms. Score range: 0 to 114; higher scores mean higher levels of anxiety, lower scores mean lower levels of anxiety.

    Measured: baseline; 4-week; 8-week

  6. Change From Baseline on Electrocardiogram (EKG) P Duration During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  7. Change From Baseline on Electrocardiogram (EKG) PR Interval During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  8. Change From Baseline on Electrocardiogram (EKG) QRS Interval During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  9. Change From Baseline on Electrocardiogram (EKG) QT Interval During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  10. Change From Baseline on Blood Clinical Labs (Sodium) During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  11. Change From Baseline on Blood Clinical Labs (Potassium) During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  12. Change From Baseline on Blood Clinical Labs (Chloride) During Treatment.

    Change from baseline on blood clinical labs (Chloride) during treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  13. Change From Baseline on Blood Clinical Labs (CO2) During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  14. Change From Baseline on Blood Clinical Labs (Anion Gap) During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  15. Change From Baseline on Blood Clinical Labs (Glucose) During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  16. Change From Baseline on Blood Clinical Labs (Creatinine) During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  17. Change From Baseline on Blood Clinical Labs (Urea Nitrogen) During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  18. Change From Baseline on Blood Clinical Labs (Calcium) During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  19. Change From Baseline on Blood Clinical Labs (Osmolality) During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  20. Change From Baseline on Urine Clinical Labs (Osmolality) During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  21. Change From Baseline on Vital Signs (Systolic Blood Pressure) During Treatment.

    Measured: Up to 12 weeks

  22. Change From Baseline on Vital Signs (Diastolic Blood Pressure) During Treatment.

    Measured: Up to 12 weeks

  23. Change From Baseline on Vital Signs (Pulse) During Treatment.

    Measured: Up to 12 weeks

  24. Change From Baseline on Height During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  25. Change From Baseline on Weight During Treatment.

    Measured: baseline to 4-week, 8-week, and 12-week

  26. Change From Baseline on the Dosage Record Treatment Emergent Symptom Scale (DOTES) During Treatment.

    The DOTES evaluates a subset of symptoms related to various medical conditions. The clinician assesses intensity (0=Not assessed, 1=Not present, 2=Mild, 3=Moderate, 4=Severe), relatedness (0=None, 1=Remote, 2=Possible, 3=Probable, 4=Defined), and action taken (0=None, 1=Increased , 2=Contractive Rx, 3=Change Dose, 4=Change Dose Plus Contractive Rx, 5=Suspend Rx, 6=Discontinue Rx). Side effects are included that have increased in severity, become more likely to be related, or require action. Change from baseline is reported as the number of participants with change in these side effects during treatment.

    Measured: Up to 12 weeks

  27. Number of Participants Reporting Aggression Using the Overt Aggression Scale (OAS) During Treatment.

    The scale evaluates the child participant's aggression. The number of children who exhibited aggression as reported by the parent is reported.

    Measured: baseline, 4-weeks, 8-weeks

  28. Adverse Event Severity

    Adverse events collated according to intensity for each pre-allocated treatment group.

    Measured: Up to 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Jul 2017
  2. Study start20 Feb 2018
  3. Primary completion18 Mar 2024
  4. Study completion18 Mar 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
157 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03204786
Sponsor's protocol ID
IRB-39972
Other IDs
1R01HD091972
Status last verified
May 2026
First submitted
28 Jun 2017
Last updated
4 Jun 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: National Database for Autism Research (NDAR) Submission per NIH requirements

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Vasopressin (USP) Injectable Solution [Vasostrict] in people with Autism and Autism Spectrum Disorder, run by Stanford University at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 19 Sep 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Parent Rated Social Responsiveness Scale, Second Edition (SRS-2) Total Scores During Treatment. (time frame: baseline, 4-week, 8-week). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jun 2026.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in