MedPath

Intranasal Vasopressin Treatment in Children With Autism

Phase 2
Completed
Conditions
Autism
Autism Spectrum Disorder
ASD
Interventions
Registration Number
NCT03204786
Lead Sponsor
Stanford University
Brief 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. It has been proposed that administration of the hormone may also help improve social functioning in individuals with autism.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
108
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Placebo-VasopressinVasopressin (USP) Injectable Solution [Vasostrict]4 weeks of placebo nasal spray followed by 4 weeks of vasopressin nasal spray (16 international units twice daily)
Placebo-VasopressinPlacebo4 weeks of placebo nasal spray followed by 4 weeks of vasopressin nasal spray (16 international units twice daily)
Placebo-PlaceboPlacebo8 weeks of placebo nasal spray; followed by a 4 week open-label extension of vasopressin nasal spray (16 international units twice daily)
Vasopressin-VasopressinVasopressin (USP) Injectable Solution [Vasostrict]8 weeks of vasopressin nasal spray (16 international units twice daily)
Placebo-PlaceboVasopressin (USP) Injectable Solution [Vasostrict]8 weeks of placebo nasal spray; followed by a 4 week open-label extension of vasopressin nasal spray (16 international units twice daily)
Primary Outcome Measures
NameTimeMethod
Change from baseline in parent rated Social Responsiveness Scale, Second Edition (SRS-2) Total Scores during treatment.4-week; 8-week
Secondary Outcome Measures
NameTimeMethod
Change from baseline on Reading the Mind in the Eyes Test (RMET) during treatment.4-week; 8-week
Change from baseline on electrocardiogram (EKG) PR Interval during treatment.4-week; 8-week
Change from baseline on blood clinical labs (Potassium) during treatment.4-week; 8-week
Change from baseline on blood clinical labs (Osmolality) during treatment.4-week; 8-week
Change from baseline on blood clinical labs (Chloride) during treatment.4-week; 8-week
Change from baseline on blood clinical labs (Calcium) during treatment.4-week; 8-week
Change from baseline on electrocardiogram (EKG) QT Interval during treatment.4-week; 8-week
Change from baseline on blood clinical labs (CO2) during treatment.4-week; 8-week
Change from baseline on blood clinical labs (Urea Nitrogen) during treatment.4-week; 8-week
Change from baseline on vital signs (pulse) during treatment.4-week; 8-week
Change from baseline in Clinical Global Impression (CGI) scores 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 on electrocardiogram (EKG) QRS Interval during treatment.4-week; 8-week
Change from baseline on blood clinical labs (Sodium) during treatment.4-week; 8-week
Change from baseline on blood clinical labs (Glucose) during treatment.4-week; 8-week
Change from baseline on electrocardiogram (EKG) P Duration during treatment.4-week; 8-week
Change from baseline on height 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 blood clinical labs (Anion Gap) during treatment.4-week; 8-week
Change from baseline on blood clinical labs (Creatinine) during treatment.4-week; 8-week
Change from baseline on urine clinical labs (Osmolality) during treatment.4-week; 8-week
Change from baseline on vital signs (systolic blood pressure) 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 on vital signs (diastolic blood pressure) during treatment.4-week; 8-week
Change from baseline on weight during treatment.4-week; 8-week
Baseline vasopressin concentration predicting primary and secondary behavioral outcome measures.4-week; 8-week
Change from baseline in Overt Aggression Scale (OAS) during treatment.2-week, 4-week; 6-week, 8-week

Trial Locations

Locations (1)

Stanford University

🇺🇸

Stanford, California, United States

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