Intranasal Vasopressin Treatment in Children With Autism

Study Overview
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Brief Summary
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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.

Official Title
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Intranasal Vasopressin Treatment in Children With Autism

Conditions
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Autism, Autism Spectrum Disorder, ASD

Intervention / Treatment
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* Drug: Vasopressin (USP) Injectable Solution [Vasostrict]
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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). Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Vasopressin-Vasopressin<br>8 weeks of vasopressin nasal spray (16 international units twice daily) | Drug: Vasopressin (USP) Injectable Solution [Vasostrict]<br>* Nasal Spray<br>|
| Experimental: Placebo-Vasopressin<br>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]<br>* Nasal Spray<br>Drug: Placebo<br>* Placebo Nasal Spray<br>|
| Placebo Comparator: Placebo-Placebo<br>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]<br>* Nasal Spray<br>Drug: Placebo<br>* Placebo Nasal Spray<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in parent rated Social Responsiveness Scale, Second Edition Total Scores (SRS-2) total scores during treatment. |  | 4-week; 8-week | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in Clinical Global Impression (CGI) scores during treatment. |  | 4-week; 8-week | 
| Change from baseline on Reading the Mind in the Eyes Test (RMET) during treatment. |  | 4-week; 8-week | 
| Change from baseline on the Facial Emotion Recognition Test during treatment. |  | 4-week; 8-week | 
| Change from baseline in parent rated Repetitive Behavior Scale Revised (RBS-R) scores during treatment. |  | 4-week; 8-week | 
| Change from baseline in parent rated Spence Children's Anxiety Scale (SCAS) during treatment. |  | 4-week; 8-week | 
| Change from baseline on electrocardiogram (EKG) (P Duration, PR Interval, QRS Interval, QT Interval) during treatment. |  | 4-week; 8-week | 
| Change from baseline on clinical labs (Sodium, Potassium, Chloride) during treatment. |  | 4-week; 8-week | 
| Change from baseline on blood pressure (systolic and diastolic) during treatment. |  | 4-week; 8-week | 
| Change from baseline on the Dosage Record Treatment Emergent Symptom Scale (DOTES) during treatment. |  | 2-week, 4-week; 6-week, 8-week | 
| Change from baseline in Overt Aggression Scale (OAS) during treatment. |  | 2-week, 4-week; 6-week, 8-week | 
| Baseline vasopressin concentration predicting primary and secondary behavioral outcome measures. |  | 4-week; 8-week | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Autism Spectrum Disorder (ASD), Autism