INtranasal OXyTocin for the Treatment of Autism Spectrum Disorders (INOXT)

INtranasal OXyTocin for the Treatment of Autism Spectrum Disorders (ASD)

There is substantial evidence from animal model and healthy control data, that oxytocin is involved in the modulation of social cognition. In addition, recent genetics and plasma level studies suggest a possible role for oxytocin in the pathophysiology of Autism Spectrum Disorders (ASD). As a large number of children with ASD are transitioning into adulthood and will likely require treatment, the lack of data to make meaningful treatment recommendations to facilitate adult living is an urgent issue. This study will examine the effect of intranasal oxytocin (IN-OXT) on social function in adults with ASD. It is hypothesized that IN-OXT will be superior to placebo in improving social function by the end of study treatment.

Study Overview

• Status: Completed
• Conditions: Autism Spectrum Disorder
• Intervention / Treatment: Drug: Intranasal Oxytocin; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 70
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Hamilton, Ontario, Canada, L8S 4K1
      • St. Joseph's Healthcare Hamilton
    • Toronto, Ontario, Canada, M4G 1R8
      • Holland Bloorview Kids Rehabilitation Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 45 years (Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria

1. Male or female outpatients 18-45 years of age, inclusive
2. Meet Diagnostic and Statistical Manual of Mental Disorders. Diagnostic and Statistical Manual (DSM-V) criteria will be established by a clinician with expertise with individuals with ASD. Best estimate Diagnosis will be reached using DSM-V criteria, the Autism Diagnostic Observation Schedule (ADOS) and the Autism Diagnostic Interview (ADI-R).
3. Have a Clinical Global Impression-Severity (CGI-S) score ≥ 4 (moderately ill) at Screening.
4. Verbal scale Intelligence Quotient (IQ) ≥ 70
5. If already receiving stable concomitant medications affecting behavior, have stable regimens with no changes during the preceding 1 month prior to Screening (with the exception of fluoxetine, where a period of 6 weeks is needed), and will not electively initiate new or modify ongoing medications for the duration of the study
6. If already receiving stable non-pharmacological educational, behavioral, and/or dietary interventions, have continuous participation during the preceding 3 months prior to Screening, and not electively initiate new or modify ongoing interventions for the duration of the study
7. Have normal physical examination and laboratory test results at screening. If abnormal, the finding(s) must be deemed clinically insignificant by the Treating Clinician.
8. Ability to speak and understand English sufficiently to allow for the completion of all study assessments
9. Ability to obtain written informed consent from the subject (if developmentally appropriate), or ability to obtain written informed consent from their surrogate decision maker (SDM), if the subject is unable to provide consent.

Exclusion Criteria

1. Patients born prior to 28 weeks gestational age
2. Patients with a primary psychiatric diagnosis other than ASD
3. Patients with a medical history of neurological disease, including, but not limited to, epilepsy/seizure disorder, movement disorder, tuberous sclerosis, fragile X, and any other known genetic syndromes, or known abnormal brain MRI/structural lesion. Exceptions: 1) simple febrile seizures, 2) epilepsy/ seizure free for at least 2 years prior to Screening
4. Pregnant female patients, sexually active female patients on hormonal birth control and sexually active females who do not use at least two types of non-hormonal birth control
5. Patients with evidence or history of malignancy or any significant hematological, endocrine, cardiovascular (including any rhythm disorder), respiratory, renal, hepatic, or gastrointestinal disease
6. Patients with one or more of the following: HIV, Hepatitis B virus, Hepatitis C virus, hemophilia (bleeding problems, recent nose and brain injuries), abnormal blood pressure (hypotension or hypertension), drug abuse, immunity disorder or severe depression.
7. Patients unable to tolerate venipuncture procedures for blood sampling
8. Patients who are currently taking oxytocin or have taken intranasal oxytocin in the past with no response
9. Patients with a sensitivity to oxytocin or any components of its formulation

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; 24 IU taken twice daily (BID), in the morning and at noon/early afternoon
Active Comparator: Intranasal Oxytocin	Drug: Intranasal Oxytocin; 24 IU taken twice daily (BID), in the morning and at noon/early afternoon; Other Names: Syntocinon

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy of intranasal oxytocin vs. placebo on social function in adults with ASD	This will be measured by the Clinical Global Impressions - Improvement Scale - Social (CGI-I-Social).	12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy of intranasal oxytocin vs. placebo on a continuous measure of social cognition in adults with ASD	This will be measured by the Revised Eyes Test	12 weeks
Efficacy of intranasal oxytocin vs. placebo on a continuous measure of social function in adults with ASD	This will be measured by the Vineland Adaptive Behavior Scale (VABS-II)	12 weeks
Efficacy of intranasal oxytocin vs. placebo on a continuous measure of social responsiveness in adults with ASD	This will be measured by the Social Responsiveness Scale (SRS-2)	12 weeks
Efficacy of intranasal oxytocin vs. placebo on a continuous measure of social responsiveness in adults with ASD	This will be measured by the Aberrant Behavior Checklist (ABC)	12 weeks
Safety and tolerability of intranasal oxytocin in adults with ASD	This will be measured by the Safety Monitoring Uniform Report Form (SMURF)	12 weeks
Safety and tolerability of intranasal oxytocin in adults with ASD	This will be measured by the Clinical Global Impressions - Improvement Scale - Global (CGI-I-Global)	12 weeks
Efficacy of intranasal oxytocin vs. placebo on quality of life	This will be measured by the World Health Organization Quality of Life Survey (WHOQOL-BREF)	12 weeks
Efficacy of intranasal oxytocin vs. placebo on anxiety	This will be measured by the Symptom Checklist 90-Revised (SCL-90-R)	12 weeks

Collaborators and Investigators

• Sponsor: Holland Bloorview Kids Rehabilitation Hospital
• Collaborators: McMaster University; Unity Health Toronto
• Investigators: Principal Investigator: Evdokia Anagnostou, M.D., Holland Bloorview Kids Rehabilitation Hospital; Principal Investigator: Marc Woodbury-Smith, M.D., McMaster University

Study record dates

Study Major Dates

• Study Start: June 1, 2014
• Primary Completion (Actual): August 1, 2017
• Study Completion (Actual): October 1, 2017

Study Registration Dates

• First Submitted: February 7, 2013
• First Submitted That Met QC Criteria: February 7, 2013
• First Posted (Estimated): February 11, 2013

Study Record Updates

• Last Update Posted (Actual): July 16, 2025
• Last Update Submitted That Met QC Criteria: July 14, 2025
• Last Verified: January 1, 2018

More Information

Terms related to this study

• Keywords: Adults; Oxytocin; Autism Spectrum Disorders
• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Autism Spectrum Disorder; Autistic Disorder; Child Development Disorders, Pervasive; Physiological Effects of Drugs; Reproductive Control Agents; Oxytocics; Oxytocin
• Other Study ID Numbers: INOXT-10-2013

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO