Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism Spectrum Disorders (ASD)

NCT01908205 | Completed Phase 2 Results DMC

• 1 other identifier: OXY-R07-2013
• Study Type: interventional
• Target: 60
• Locations: 2 countries
• Sites: 2

Brief Summary

We are studying an investigational drug called intranasal oxytocin (Syntocinon®). Syntocinon® has been approved by the U.S. Food and Drug Administration for use in helping women breastfeed, but it has not been approved for use in children with ASD. However, there is previous research conducted that has indicated that after administration of oxytocin, adults with ASD demonstrated improvements in social cognition, and reduced repetitive behaviours and anxiety. There is also early research to suggest that children may also benefit in these areas. The purpose of this study is to test if oxytocin works to help children and adolescents with ASD.

Conditions

Autism Spectrum Disorder

Keywords

Autism; Social; Functioning; Cognition; Repetitive Behaviors; Anxiety; Quality of Life

Outcome Measures

Primary Outcomes (1)

• Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Function

  This will be measured by a change in score on the Aberrant Behavior Checklist (ABC) - Social Withdrawal Subscale (0-48, where lower scores indicate improvement)

  12 and 24 weeks

Secondary Outcomes (10)

• Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Cognition

  12 Weeks

• Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Cognition

  12 Weeks

• Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Function

  12 Weeks

• Number of Participant Considered Social Responders

  12 Weeks

• Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Repetitive Behaviors

  12 Weeks

Study Arms (2)

Intranasal Oxytocin (Syntocinon)

EXPERIMENTAL

The proposed dosing schedule is 0.4 IU/kg, taken twice daily, for a maximum of 24 IUs per dose

Drug: Intranasal Oxytocin

Placebo

PLACEBO COMPARATOR

The proposed dosing schedule is 0.4 IU/kg, taken twice daily, for a maximum of 24 IUs per dose

Drug: Placebo

Interventions

Intranasal Oxytocin DRUG

Also known as: Syntocinon

Intranasal Oxytocin (Syntocinon)

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 10 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or female outpatients, 10-17 years of age inclusive.
• Meet Diagnostic and Statistical Manual of Mental Disorders, 4th Edition. Diagnostic and Statistical Manual (DSM-IV) criteria will be established by a clinician with expertise with individuals with ASD. Best estimate Diagnosis will be reached using DSM-IV criteria, the Autism Diagnostic Observation Schedule (ADOS-2) and the Autism Diagnostic Interview (ADI-R).
• Have a Clinician's Global Impression-Severity (CGI-S) score ≥ 4 (moderately ill) at Screening.
• Verbal and performance scale Intelligence Quotient (IQ) ≥ 70 (both subtests of the Wechsler Abbreviated Scale of Intelligence (WASI-I or WASI-II ≥ 70).
• If already receiving stable concomitant medications affecting behavior, have continuous participation for 1 month prior to Screening (with the exception of fluoxetine, where a period of 6 weeks is needed), and not electively initiate new or modify ongoing medications for the duration of the study.
• If already receiving stable non-pharmacologic 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 not clinically significant 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 participant, if developmentally appropriate. If a participant does not have the capacity to consent, ability to obtain assent (if developmentally appropriate), as well as written informed consent from their parent(s)/legal guardian.

You may not qualify if:

• Patients born prior to 35 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 (except simple febrile seizures), movement disorder, tuberous sclerosis, fragile X, and any other known genetic syndromes, or known abnormal brain MRI/structural lesion.
• 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 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.
• Patients unable to tolerate venipuncture procedures for blood sampling.
• Patients in foster care for whom the province/state is defined as a legal guardian

Sponsors & Collaborators

• Holland Bloorview Kids Rehabilitation Hospital: lead
• United States Department of Defense: collaborator

Study Sites (2)

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, M4G 1R8, Canada

Location

Related Publications (1)

• Iffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2.

  PMID: 37811711 DERIVED

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder; Anxiety Disorders

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

Limitations and Caveats

Limitations include a small sample size, age range restricted to 10-17, and IQ restriction of 70+. This limits our ability to generalize our results to other individuals on the autism spectrum.

Results Point of Contact

• Title: Lisa Genore
• Organization: Holland Bloorview Kids Rehabilitation Hospital

lgenore@hollandbloorview.ca

416-425-6220

Study Officials

• Evdokia Anagnostou, M.D.

  Holland Bloorview Kids Rehabilitation Hospital

  PRINCIPAL INVESTIGATOR

• Suma Jacob, M.D., Ph.D.

  University of Minnesota

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

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: July 23, 2013
• First Posted: July 25, 2013
• Study Start: September 1, 2013
• Primary Completion: September 1, 2015
• Study Completion: March 1, 2016
• Last Updated: July 16, 2025
• Results First Posted: November 16, 2020

Record last verified: 2020-10

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); US (1)