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Autism Spectrum DisorderPhase 2OXY-RNCT01908205

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

A Phase 2 study testing Intranasal Oxytocin in people with Autism Spectrum Disorder, run by Holland Bloorview Kids Rehabilitation Hospital at 2 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 10 to 17 years
Conditions the study is for
Autism Spectrum Disorder
What is being tested?
Intranasal Oxytocin
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
60 participants
Where
2 sitesin 2 countries
Study dates
Sep 2013to Mar 2016
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 10 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.
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  • 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.

Who cannot join

  • 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.
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  • 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

Trial locations and contacts

2 sites in 2 countries.

2 sites in 2 countries

United States1 site

  • University of Minnesota

    Minneapolis, Minnesota, 55455

Other trials in United States

Canada1 site

  • Holland Bloorview Kids Rehabilitation Hospital

    Toronto, Ontario, M4G 1R8

Other trials in Canada

About this study

Official title: Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism

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.

Read the detailed description

Extensive data has been accumulated to suggest that central release of oxytocin is important for social cognition and function, as well as likely involved in anxiety modulation and repetitive behaviors. The Principal Investigator and Co-Principal Investigator of this study have previously documented: 1) an association between ASD and a single nuclear polymorphism of the oxytocin receptor gene, 2) ability to measure oxytocin levels in the blood by enzyme immunoassay and 3) preliminary data to support safety and efficacy of intranasal oxytocin in the treatment of social deficits and repetitive behaviors in adults with autism. A medication treatment targeting the core deficits of ASD in childhood is highly valuable because it could influence the developmental trajectory and make further psychosocial interventions possible. In this context, we propose a randomized placebo controlled trial of intranasal oxytocin in children and adolescents with ASD.

Being tested
Intranasal Oxytocin, Placebo
Keywords
Autism, Social, Functioning, Cognition, Repetitive Behaviors, Anxiety, Quality of Life
Medical subject headings
Autism Spectrum Disorder, Autistic Disorder, Anxiety Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Intranasal OxytocinDrug

    Also called Syntocinon

  • PlaceboDrug

Study groups in NCT01908205
Study groupRoleWhat they receive
Intranasal Oxytocin (Syntocinon)ExperimentalThe proposed dosing schedule is 0.4 IU/kg, taken twice daily, for a maximum of 24 IUs per dose
Receives: Drug: Intranasal Oxytocin
PlaceboPlacebo comparatorThe proposed dosing schedule is 0.4 IU/kg, taken twice daily, for a maximum of 24 IUs per dose
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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)

    Measured: 12 and 24 weeks

Secondary outcomes (10)

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

    This will be measured by a change in score the Revised Eyes Test (0- 28; where higher scores indicate better performance/improvement) Baseline to Week 12

    Measured: 12 Weeks

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

    This will be measured by improvement on the Let's Face it! Skills Battery from Baseline to Week 12 Social Cognition (higher score=better outcome)

    1. Let's Face It Skills Battery; i. Matchmaker (0-100); ii. Faces (0-100); iii. Houses (0-100)

    Measured: 12 Weeks

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

    This will be measured by improvement on the Behavioral Assessment System for Children (BASC-2) from Baseline to Week 12 Behavioral Assessment System for Children (higher score=positive response); i. *Social Skills: age 6 to 11 (18-69); age 12 to 17 (21-70); ii. Functional Communication: age 6 to 11 (10-66); age 12 to 17 (10-64); iii. Withdrawal age 6-11 (21- 62) ; age 12-17 (14-42)

    • only social subscales of BASC-2 reported

    Measured: 12 Weeks

  4. Number of Participant Considered Social Responders

    This will be measured by the Clinical Global Impressions - Improvement Scale - Social (CGI-I-Social)

    1. Clinical Global Impressions - Social Scale (1-7) (lower score=positive response). The results will be reported as the number of participants that were classified as a social responder (achieving a score of 1 or 2 on the scale).

    Measured: 12 Weeks

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

    This will be measured by improvement on the Child Yale-Brown Obsessive-Compulsive Scale (CY-BOCS) from Baseline to Week 12

    -lower score= positive response (0-20)

    Measured: 12 Weeks

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

    This will be measured by improvement on the Repetitive Behavior Scale (RBS-R)(0-129; where lower score= positive response) from Baseline to Week 12

    Measured: 12 Weeks

  7. Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Anxiety

    This will be measured by the Child and Adolescent Symptom Inventory (CASI-4R) Generalized anxiety score (male (40-101); female (41-96) -where lower score= positive response) from Baseline to Week 12

    Measured: 12 Weeks

  8. Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Quality of Life

    This will be measured by improvement on the Pediatric Quality of Life Inventory (PedsQL) (0-100, where higher scores indicate positive response) from Baseline to Week 12

    Measured: 12 Weeks

  9. Number of Participant Considered Overall Responders

    This will be measured by the Clinical Global Impressions - Improvement Scale - Global (CGI-I-Global) (1-7) (lower score=positive response). The results will be reported as the number of participants that were classified as an overall responder (achieving a score of 1 or 2 on the scale).

    Measured: 12 Weeks

  10. Safety and Tolerability of Intranasal Oxytocin in Children and Adolescents With ASD

    This will be measured by the Safety Monitoring Uniform Report Form (SMURF)

    Measured: 12 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Jul 2013 (estimated)
  2. Study startSep 2013
  3. Primary completionSep 2015
  4. Study completionMar 2016

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
60 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01908205
Sponsor's protocol ID
OXY-R07-2013
Acronym
OXY-R
Status last verified
Oct 2020
First submitted
23 Jul 2013
Last updated
16 Jul 2025
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • 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.

    PubMed 37811711

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Intranasal Oxytocin in people with Autism Spectrum Disorder, run by Holland Bloorview Kids Rehabilitation Hospital at 2 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 16 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 16 Nov 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Function (time frame: 12 and 24 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01908205, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2025.

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