Vasopressin Nasal Spray for Autism

Overseen By Esha Dhawan

Age: < 18

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Stanford University

Must not be taking: Tricyclics, Lithium, Alcohol, others

Disqualifiers: Schizophrenia, Seizures, Genetic mutations, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether a vasopressin nasal spray can improve social skills in individuals with autism. Vasopressin, a hormone, may regulate social behaviors. Participants will receive either the vasopressin spray or a placebo (a substance with no active ingredients) to compare effects. This trial may suit children and teens diagnosed with autism who face challenges with social communication. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to important findings.

Will I have to stop taking my current medications?

The trial requires that any psychotropic medications or medications affecting vasopressin be stable for at least 4 weeks (6 weeks for fluoxetine) before participating. You cannot participate if you are currently using medications that interact with vasopressin, such as certain antidepressants and other specified drugs.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that vasopressin nasal spray is generally safe for use. One study found it has a good safety record and does not cause major side effects, suggesting it might be safe for treating symptoms related to autism. Specifically, past patients demonstrated that altering how the body uses vasopressin can be promising, without serious side effects. While any treatment carries some risk, current evidence suggests that vasopressin is a relatively safe option for those considering participation in clinical trials. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about vasopressin nasal spray for autism because it offers a new approach that differs from standard treatments like behavioral therapies and medications such as antipsychotics. Vasopressin, a hormone known for its role in social behavior, is administered via nasal spray, which is a novel delivery method compared to oral medications. This treatment targets social functioning, a core challenge in autism, potentially improving social interactions and communication in a way that other treatments don't specifically address. By focusing on vasopressin, researchers hope to tap into its unique mechanism to unlock new possibilities for managing autism symptoms.

What evidence suggests that vasopressin nasal spray might be an effective treatment for autism?

Studies have shown that vasopressin, a hormone, might help improve social behaviors in people with autism. Research indicates that children with autism often have lower levels of vasopressin, which could be linked to their social challenges. Early findings suggest that using a nasal spray to deliver vasopressin may enhance social communication. In this trial, participants will join different treatment arms: one group will receive vasopressin nasal spray for 8 weeks, another will receive placebo nasal spray for 4 weeks followed by vasopressin for 4 weeks, and a third group will receive placebo nasal spray for 8 weeks followed by a 4-week open-label extension of vasopressin. Some studies have observed improvements when vasopressin affects certain brain areas involved in social interactions. However, only specific groups of individuals with autism might benefit the most from this treatment. ^{1 2 5 6 7}

Who Is on the Research Team?

Antonio Y. Hardan, M.D.

Principal Investigator

Stanford University

Karen J. Parker, Ph.D.

Principal Investigator

Stanford University

Are You a Good Fit for This Trial?

This trial is for medically healthy children aged 6-17 with Autism Spectrum Disorder, as confirmed by specific diagnostic tools. Participants must have a certain level of social impairment and an IQ over 40. They should be on stable medications, if any, and not planning to change treatments during the study. Exclusions include previous vasopressin trials, certain mental health diagnoses, unstable medical conditions, pregnancy without contraception use, and regular nasal issues.

Inclusion Criteria

I am either male or female.

Meet DSM-5 criteria for Autism Spectrum Disorder (ASD) confirmed with ADI-R, ADOS-2 or CARS-2

Social Responsiveness Scale-2 Total Score of 70 and above

Exclusion Criteria

I have major hearing or vision problems.

I am not pregnant and use reliable birth control.

I am currently taking medication that interacts with vasopressin.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either vasopressin or placebo nasal spray for 8 weeks

8 weeks

Open-label extension

Participants in the placebo group receive vasopressin nasal spray for 4 weeks

4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• Vasopressin

Trial Overview The trial tests whether a hormone called vasopressin can improve social functioning in autistic children when given as a nasal spray compared to a placebo (a substance with no active drug). Vasopressin naturally occurs in the body and influences social behavior. Children will randomly receive either the hormone or placebo to assess its effectiveness.

How Is the Trial Designed?

Group I: Vasopressin-Vasopressin Experimental Treatment 1 Intervention

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

Group II: Placebo-Vasopressin Experimental Treatment 2 Interventions

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

Group III: Placebo-Placebo Placebo Group 2 Interventions

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

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials

2,527

Recruited

17,430,000+

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

Collaborator

Trials

2,103

Recruited

2,760,000+

Published Research Related to This Trial

A systematic review of five randomized controlled trials involving 223 participants found that long-term use of intranasal oxytocin in treating autism spectrum disorder (ASD) is generally well tolerated, with common side effects like nasal discomfort and tiredness not significantly linked to the treatment.

While some severe adverse events were reported, such as aggression and seizures, the overall safety profile suggests that intranasal oxytocin is safe for use in the ASD population, warranting further research to assess its efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Systematic review and meta-analysis of reported adverse events of long-term intranasal oxytocin treatment for autism spectrum disorder. Cai, Q., Feng, L., Yap, KZ.[2018]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10603886/

Vasopressin as Possible Treatment Option in Autism ... We concluded that although vasopressin might be an effective candidate for ASD treatment, we might assume that only a subgroup (e.g., with stress-axis ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT01962870

The Role of Vasopressin in the Social Deficits of Autism ... effectiveness of vasopressin, a neuropeptide, in treating children with autism spectrum disorder. ... Baseline vasopressin levels are included in the outcome data ...

3. epag.springeropen.com epag.springeropen.com/articles/10.1186/s43054-025-00383-6

Can vasopressin levels predict autism spectrum disorder ... The data from this study indicated that plasma vasopressin (VP) concentrations were lower in the autistic children group compared to the ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/27711048/

A Single Dose, Randomized, Controlled Proof-Of ... These results provide preliminary evidence from experimental and behavioral biomarkers, that blockade of the V1a receptor may improve social communication in ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT03204786

Intranasal Vasopressin Treatment in Children With Autism The purpose of this clinical trial is to investigate the effectiveness of vasopressin nasal spray for treating symptoms associated with autism.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3657571/

Oxytocin and vasopressin in children and adolescents with ... Previous studies have shown that boys with autism spectrum disorders (ASD) have lower levels of OT than boys without ASD, and treatment studies have found that ...

7. science.org science.org/doi/10.1126/scitranslmed.aat7838

A phase 2 clinical trial of a vasopressin V1a receptor ... Both drugs were well tolerated and had an acceptable safety profile, suggesting that modulating the vasopressin pathway may be a useful therapeutic strategy for ...