What side effects are associated with this type of intervention?

Intranasal oxytocin, studied for its potential to improve social behaviors in individuals with ASD, has been associated with side effects such as temporary gynecomastia. Vasopressin nasal spray, another treatment under investigation, has not had detailed side effects reported in the provided context. Broadly, common pharmacologic treatments for ASD, such as antipsychotics (e.g., risperidone and aripiprazole), can cause weight gain, drowsiness, and metabolic changes. Behavioral interventions generally have fewer side effects but may require significant time and effort from caregivers.

What prior FDA approvals exist?

The FDA has approved two medications for the treatment of irritability associated with Autism Spectrum Disorder: risperidone and aripiprazole. These antipsychotic drugs help manage symptoms such as aggression, self-injury, and severe tantrums. While these medications do not specifically target social behaviors, they can indirectly improve social functioning by reducing disruptive behaviors. Other treatments, such as selective serotonin reuptake inhibitors (SSRIs) and stimulants, have been used off-label to address various symptoms of ASD, but their efficacy in regulating social behaviors is less clear. The ongoing trial of vasopressin nasal spray aims to directly improve social functioning, which represents a novel approach in ASD pharmacotherapy.

What other types of research exist?

Promising alternatives to vasopressin nasal spray for ASD patients include oxytocin nasal spray, which has been studied for its potential to improve social behaviors, and bumetanide, a diuretic that has shown promise in reducing core symptoms of autism. Additionally, behavioral interventions such as Applied Behavior Analysis (ABA) and emerging digital therapeutics are also being explored as effective treatments.

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Vasopressin Nasal Spray for Autism

Phase 2 & 3

Waitlist Available

Led By Karen J. Parker, Ph.D.

Research Sponsored by Stanford University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Medically healthy outpatients between 6 and 17 years of age

IQ of 40 and above

Must not have

Previous participation in a vasopressin clinical trial or current use of vasopressin

DSM-5 diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial is testing a nasal spray containing vasopressin, a hormone that naturally occurs in the body. The spray is aimed at helping people with autism improve their social interactions. Vasopressin is known to play a role in regulating social behaviors, and the trial hopes to see if it can make social functioning better for those with autism. Vasopressin has been implicated in social bonding processes and social behaviors in both animals and humans, and it is being explored for its potential to improve social functioning in autism.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects from vasopressin may include water retention leading to an imbalance of salts in the blood (hyponatremia), headache, abdominal cramps, nausea or vomiting. There might also be local reactions at the site of administration like irritation or nosebleeds.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 6 and 17 years old and generally healthy.

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My IQ is 40 or higher.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been in a vasopressin study or am currently using vasopressin.

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I have been diagnosed with schizophrenia, schizoaffective disorder, or another psychotic disorder.

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I have a genetic mutation linked to autism or intellectual disability.

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I have major hearing or vision problems.

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I am not pregnant and use reliable birth control.

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I am currently taking medication that interacts with vasopressin.

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I do not have uncontrolled medical conditions like severe migraines, asthma, or seizures.

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I often have a blocked nose or nosebleeds.

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I am currently taking desmopressin or oxytocin.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Vasopressin-VasopressinExperimental Treatment1 Intervention

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

Group II: Placebo-VasopressinExperimental Treatment2 Interventions

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

Group III: Placebo-PlaceboPlacebo Group2 Interventions

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vasopressin (USP) Injectable Solution [Vasostrict]

2018

Completed Phase 3

~110

Placebo

1995

Completed Phase 3

~2670

0 / 11Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Autism Spectrum Disorder (ASD) that focus on regulating social behaviors include the use of hormones like oxytocin and vasopressin. Oxytocin is known to enhance social bonding, trust, and emotional recognition, while vasopressin is implicated in social communication and behaviors. These treatments aim to improve social functioning, which is a core challenge in ASD. Additionally, behavioral and educational interventions, such as Applied Behavior Analysis (ABA) and the Early Start Denver Model (ESDM), focus on improving social skills through structured, individualized programs. These treatments are crucial as they address the social deficits that significantly impact the quality of life for individuals with ASD.

Clinical and neural effects of six-week administration of oxytocin on core symptoms of autism.Intranasal oxytocin versus placebo in the treatment of adults with autism spectrum disorders: a randomized controlled trial.