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

108 Participants Needed

Trial Summary

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.

What evidence supports the effectiveness of the drug vasopressin nasal spray for autism?

A pilot trial showed that intranasal vasopressin improved social abilities in children with autism, as measured by the Social Responsiveness Scale, and also reduced anxiety and some repetitive behaviors. These findings suggest that vasopressin could be a promising treatment for social impairments in autism. ^{1 2 3 4 5}

Is vasopressin nasal spray safe for use in humans?

In a study with children with autism, vasopressin nasal spray was well tolerated with minimal side effects, and no participants dropped out due to adverse effects. There were no significant changes in vital signs or other health measures after 4 weeks of treatment. ^{1 5 6 7 8}

How is the drug vasopressin nasal spray unique for treating autism?

Vasopressin nasal spray is unique because it is administered intranasally (through the nose), which can enhance and prolong its effects compared to other administration routes. This method is less invasive and may offer a novel approach for autism treatment, as there are no standard treatments specifically targeting this condition with vasopressin. ^{9 10 11 12 13}

What is the purpose of this trial?

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.

Research Team

Antonio Y. Hardan, M.D.

Principal Investigator

Stanford University

Karen J. Parker, Ph.D.

Principal Investigator

Stanford University

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

A randomized placebo-controlled pilot trial shows that intranasal vasopressin improves social deficits in children with autism. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

A phase 2 clinical trial of a vasopressin V1a receptor antagonist shows improved adaptive behaviors in men with autism spectrum disorder. [2020]

3. England pubmed.ncbi.nlm.nih.gov

Efficacy and safety of balovaptan for socialisation and communication difficulties in autistic adults in North America and Europe: a phase 3, randomised, placebo-controlled trial. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Cerebrospinal fluid vasopressin and symptom severity in children with autism. [2021]

5. United States pubmed.ncbi.nlm.nih.gov

Tinkering with the vasopressin pathway in autism. [2020]

6. United States pubmed.ncbi.nlm.nih.gov

Efficacy and safety of V1a receptor antagonists in autism spectrum disorder: A meta-analysis. [2023]

7. Australia pubmed.ncbi.nlm.nih.gov

Systematic review and meta-analysis of reported adverse events of long-term intranasal oxytocin treatment for autism spectrum disorder. [2018]

8. England pubmed.ncbi.nlm.nih.gov

New V1a receptor antagonist. Part 1. Synthesis and SAR development of urea derivatives. [2021]

9. United States pubmed.ncbi.nlm.nih.gov

Neurogenic diabetes insipidus: management with dDAVP (1-desamino-8-D arginine vasopressin). [2019]

10. Australia pubmed.ncbi.nlm.nih.gov

Diabetes insipidus treated by DDAVP. [2021]

11. United States pubmed.ncbi.nlm.nih.gov

DIABETES INSIPIDUS. TREATMENT WITH 8-LYSINE VASOPRESSIN IN A NASAL SPRAY. [2018]

12. Ireland pubmed.ncbi.nlm.nih.gov

Effect of 1-deamino 8-D-arginine vasopressin spray on nasal cytology and mucociliary clearance in patients with nocturnal enuresis. [2013]

13. Russia (Federation) pubmed.ncbi.nlm.nih.gov

[Dependence of neuropeptide physiological effects on a route of administration]. [2016]