INtranasal OXyTocin for the Treatment of Autism Spectrum Disorders

NCT01788072 | Completed Phase 2 DMC

• 1 other identifier: INOXT-10-2013
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Autism Spectrum Disorder

Keywords

Autism Spectrum Disorders; Oxytocin; Adults

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (8)

• Efficacy of intranasal oxytocin vs. placebo on a continuous measure of social cognition in adults with ASD

  12 weeks

• Efficacy of intranasal oxytocin vs. placebo on a continuous measure of social function in adults with ASD

  12 weeks

• Efficacy of intranasal oxytocin vs. placebo on a continuous measure of social responsiveness in adults with ASD

  12 weeks

• Efficacy of intranasal oxytocin vs. placebo on a continuous measure of social responsiveness in adults with ASD

  12 weeks

• Safety and tolerability of intranasal oxytocin in adults with ASD

  12 weeks

Study Arms (2)

Intranasal Oxytocin

ACTIVE COMPARATOR

Drug: Intranasal Oxytocin

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Intranasal Oxytocin DRUG

24 IU taken twice daily (BID), in the morning and at noon/early afternoon

Also known as: Syntocinon

Intranasal Oxytocin

Placebo DRUG

24 IU taken twice daily (BID), in the morning and at noon/early afternoon

Placebo

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Male or female outpatients 18-45 years of age, inclusive
• 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).
• Have a Clinical Global Impression-Severity (CGI-S) score ≥ 4 (moderately ill) at Screening.
• Verbal scale Intelligence Quotient (IQ) ≥ 70
• 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
• 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
• Have normal physical examination and laboratory test results at screening. If abnormal, the finding(s) must be deemed clinically insignificant by the Treating Clinician.
• Ability to speak and understand English sufficiently to allow for the completion of all study assessments
• 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.

You may not qualify if:

• Patients born prior to 28 weeks gestational age
• Patients with a primary psychiatric diagnosis other than ASD
• 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
• 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
• Patients with evidence or history of malignancy or any significant hematological, endocrine, cardiovascular (including any rhythm disorder), respiratory, renal, hepatic, or gastrointestinal disease
• 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.
• Patients unable to tolerate venipuncture procedures for blood sampling
• Patients who are currently taking oxytocin or have taken intranasal oxytocin in the past with no response
• Patients with a sensitivity to oxytocin or any components of its formulation

Sponsors & Collaborators

• Holland Bloorview Kids Rehabilitation Hospital: lead
• McMaster University: collaborator
• Unity Health Toronto: collaborator

Study Sites (2)

St. Joseph's Healthcare Hamilton

Hamilton, Ontario, L8S 4K1, Canada

Location

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, M4G 1R8, Canada

Location

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Oxytocin

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Posterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Evdokia Anagnostou, M.D.

  Holland Bloorview Kids Rehabilitation Hospital

  PRINCIPAL INVESTIGATOR

• Marc Woodbury-Smith, M.D.

  McMaster University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 7, 2013
• First Posted: February 11, 2013
• Study Start: June 1, 2014
• Primary Completion: August 1, 2017
• Study Completion: October 1, 2017
• Last Updated: July 16, 2025

Record last verified: 2018-01

Data Sharing

• IPD Sharing: Will not share

Locations

CA (2)