Autism Spectrum DisorderPhase 2NCT01256060

Intranasal Oxytocin for the Treatment of Children and Adolescents With ASD (OXY)

A Phase 2 study testing Intranasal Oxytocin in people with Autism Spectrum Disorder, run by Evdokia Anagnostou at 1 site in Canada.

Completed

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

Official record

Search all trials

Trial summary

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?: No, everyone receives an active treatment
How many people: 15 participants
Where: 1 siteCanada
Study dates: Nov 2010to Mar 2013
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, Text Revision criteria for Autistic Disorder or Asperger's Disorder as established by a clinician and supported by the Autism Diagnostic Observation Schedule and the Autism Diagnostic Interview - Revised.
Have a Clinician's Global Impression-Severity score ≥ 4 (moderately ill) at Baseline.
Verbal Intelligent Quotient >/= 70.
If already receiving stable pharmacological and or non-pharmacologic educational, behavioral, and/or dietary interventions, have continuous participation during the preceding 3 months prior to Screening and will 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 Investigator.
The participant and caregiver must be able to speak and understand English sufficiently to allow for the completion of all study assessments.

Who cannot join

Patients born prior to 35 weeks gestational age.
Patients with any primary psychiatric diagnosis other than autism at Screening.
Patients with current 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 MRI/structural lesion of the brain.
Pregnant female patients, sexually active female patients on hormonal birth control and sexually active females who do not use two types of non-hormonal birth control
Patients with a medical condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. Patients with evidence or history of malignancy or any significant hematological, endocrine, cardiovascular (including any rhythm disorder), respiratory, renal, hepatic, or gastrointestinal disease.
Patients who are sensitive to Syntocinon or any components of its formulation
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.

Trial locations and contacts

1 site in 1 country.

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 Spectrum Disorders (ASD)

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 investigators of this study have previously documented: 1) an association between Autism Spectrum Disorder 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 Autism Spectrum Disorder in childhood is highly valuable because it could influence the developmental trajectory and make further psychosocial interventions possible. In this context, we propose a small dose finding study to confirm that the dose used in the adult study is not more than the maximum tolerated dose in youth. '

Conditions: Autism Spectrum Disorder
Being tested: Intranasal Oxytocin
Keywords: Autism Spectrum disorder, Oxytocin, Clinical Trial, Children, Pharmacology
Medical subject headings: Autism Spectrum Disorder

What participants receive

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

Intranasal OxytocinDrug
Also called Syntocinon
We are selecting morning and afternoon dosing to try to influence most hours where youth are in settings with increased potential for social interaction (school, after school). Medication will be administered by the parents before school and early afternoon. All patients will receive their first dose by the study physician to educate parents and themselves on proper administration and determine safety of first dose.

Study groups in NCT01256060
Study group	Role	What they receive
Intanasal Oxytocin	Experimental	A modified dose finding method will be used to determine safety among four dose levels for Intranasal Oxytocin. Half the dose (0.2 IU/kg /dose) is the minimum dose and two intermediate doses will also be evaluated (0.26 and 0.33 IU/kg / dose) Dose-finding escalations will be done in groups of three patients.Three patients will be studied at the first dose level. If none of these patients experience dose limiting toxicity, the dose will be escalated. If one experiences dose limiting toxicity, up to three more will be accrued at the same level. If none of these experience dose limiting toxicity, the dose will be escalated. If one or more of these experience dose-limiting toxicity, entry at that dose level will be stopped. Up to three more patients will be treated at the next lower dose. If zero out of these experience dose limiting toxicity, an additional three patients will be treated at that dose. Receives: Drug: Intranasal Oxytocin

What is being measured

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

Maximum Tolerated Dose (MTD)
The hypothesis is that the maximum tolerated dose in a range of 0.2-0.4 IU/kg / dose will be 0.4 IU/kg / dose, as was the case in the adult study, given that oxytocin is not stored in body fat and does not depend on liver or renal clearance.
Measured: 12 Weeks
Number of Participants With Serious Adverse Events
This will be reported as the number of participants who experienced a serious advert event throughout the study.
Measured: 24 Weeks

Secondary outcomes (2)

Baseline Levels of Oxytocin in Relation to Either Safety or Treatment Response
Children and adolescents with lower plasma oxytocin levels at baseline will show treatment related changes in social cognition. Children and adolescents with higher oxytocin plasma levels will show diminished or less dramatic treatment responses and may have more difficulty tolerating the treatment.
Measured: 12 Weeks
Blood Levels of Oxytocin During the Trial in Relation to Safety or Treatment Response
Children and adolescents with minimal changes in plasma level of oxytocin after treatment will be less responsive to treatment. Children and adolescents with atypical patterns of increase in oxytocin may be more sensitive to dose-related tolerability.
Measured: 12 Weeks

Timeline

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

Nov 2010Mar 2013

First posted8 Dec 2010 (estimated)
Study startNov 2010
Primary completionMar 2013
Study completionMar 2013

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 15 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01256060
Sponsor's protocol ID: 10-001
Status last verified: Jul 2016
First submitted: 22 Nov 2010
Last updated: 12 Aug 2016
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1
Cannabidiol (CBD) in Adults With ASD
Autism Spectrum Disorder
RecruitingPhase 3
A Study to Evaluate the Long-term Safety and Tolerability of Xanomeline and Trospium Chloride for the Treatment of Psychiatric Disorders in Children and Adolescents
Schizophrenia, Autism-Related Irritability
Not yet recruiting
Sensory-integration Therapy Verses Augumented Reality-based Training in ASD
Autism Spectrum Disorder
Recruiting
Cumulative and Booster Effects of Multisession Prefrontal tDCS in ASD Adolescents
Transcranial Direct Current Stimulation, Autistic Spectrum Disorder
Not yet recruiting
"Family Resilience And Mind-body Enhancement" (FRAME): A Randomised Controlled Trial
Autism

All Autism Spectrum Disorder trials

Study at a glance

From the registry record, last updated by the sponsor on 12 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism Spectrum Disorders (ASD)
ClinicalTrials.gov ID: NCT01256060
Acronym / sponsor ID: 10-001
Conditions: Autism Spectrum Disorder
Keywords: Autism Spectrum disorder, Oxytocin, Clinical Trial, Children, Pharmacology
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 15 participants (actual)
Lead sponsor: Evdokia Anagnostou
Collaborators: Holland Bloorview Kids Rehabilitation Hospital, The Hospital for Sick Children, University of Illinois Chicago
Study start: Nov 2010
Primary completion: Mar 2013 (actual)
Study completion: Mar 2013 (actual)
First posted: 8 Dec 2010 (estimated)
Results: Posted 15 Apr 2015

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Intanasal Oxytocin	Experimental	Drug: Intranasal OxytocinA modified dose finding method will be used to determine safety among four dose levels for Intranasal Oxytocin. Half the dose (0.2 IU/kg /dose) is the minimum dose and two intermediate doses will also be evaluated (0.26 and 0.33 IU/kg / dose) Dose-finding escalations will be done in groups of three patients.Three patients will be studied at the first dose level. If none of these patients experience dose limiting toxicity, the dose will be escalated. If one experiences dose limiting toxicity, up to three more will be accrued at the same level. If none of these experience dose limiting toxicity, the dose will be escalated. If one or more of these experience dose-limiting toxicity, entry at that dose level will be stopped. Up to three more patients will be treated at the next lower dose. If zero out of these experience dose limiting toxicity, an additional three patients will be treated at that dose.

Who may qualify

Ages: 10 to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (7 listed)

Male or female outpatients 10-17 years of age inclusive.
Meet Diagnostic and Statistical Manual of Mental Disorders, 4th Edition, Text Revision criteria for Autistic Disorder or Asperger's Disorder as established by a clinician and supported by the Autism Diagnostic Observation Schedule and the Autism Diagnostic Interview - Revised.
Have a Clinician's Global Impression-Severity score ≥ 4 (moderately ill) at Baseline.
Verbal Intelligent Quotient >/= 70.
If already receiving stable pharmacological and or non-pharmacologic educational, behavioral, and/or dietary interventions, have continuous participation during the preceding 3 months prior to Screening and will not electively initiate new or modify ongoing interventions for the duration of the study.

Key exclusion criteria (8 listed)

Patients born prior to 35 weeks gestational age.
Patients with any primary psychiatric diagnosis other than autism at Screening.
Patients with current 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 MRI/structural lesion of the brain.
Pregnant female patients, sexually active female patients on hormonal birth control and sexually active females who do not use two types of non-hormonal birth control
Patients with a medical condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. Patients with evidence or history of malignancy or any significant hematological, endocrine, cardiovascular (including any rhythm disorder), respiratory, renal, hepatic, or gastrointestinal disease.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Intranasal OxytocinAlso: SyntocinonWe are selecting morning and afternoon dosing to try to influence most hours where youth are in settings with increased potential for social interaction (school, after school). Medication will be administered by the parents before school and early afternoon. All patients will receive their first dose by the study physician to educate parents and themselves on proper administration and determine safety of first dose.	Drug	Intanasal Oxytocin

Primary outcome

Maximum Tolerated Dose (MTD)
Time frame: 12 Weeks
The hypothesis is that the maximum tolerated dose in a range of 0.2-0.4 IU/kg / dose will be 0.4 IU/kg / dose, as was the case in the adult study, given that oxytocin is not stored in body fat and does not depend on liver or renal clearance.
Number of Participants With Serious Adverse Events
Time frame: 24 Weeks
This will be reported as the number of participants who experienced a serious advert event throughout the study.

2 secondary outcomes

Baseline Levels of Oxytocin in Relation to Either Safety or Treatment Response
Time frame: 12 Weeks
Blood Levels of Oxytocin During the Trial in Relation to Safety or Treatment Response
Time frame: 12 Weeks

Results and safety

Results posted to the registry on 15 Apr 2015, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
0.2 IU / kg	3	3
0.26 IU / kg	3	2
0.33 IU / kg	3	3
0.4 IU / kg	6	6

Primary outcome: Maximum Tolerated Dose (MTD)

Time frame: 12 Weeks. Number. Unit: IU / kg

Measure	Intanasal Oxytocin n = 15
Value	0.40

Primary outcome: Number of Participants With Serious Adverse Events

Time frame: 24 Weeks. Number. Unit: participants

Measure	Intanasal Oxytocin n = 15
Value	0

2 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Intranasal Oxytocin	0 of 15 (0%)	15 of 15 (100%)	Not reported

Limitations and caveats (from the sponsor): The sample size is too small to view both safety and efficacy data. The design did not exceed the commonly used 24 IU / dose, so it is not clear that higher doses would not confer benefit. In the absence of placebo, adverse events may be inflated.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Autism Spectrum Disorder

RecruitingPhase 1
Cannabidiol (CBD) in Adults With ASD
Autism Spectrum Disorder
RecruitingPhase 3
A Study to Evaluate the Long-term Safety and Tolerability of Xanomeline and Trospium Chloride for the Treatment of Psychiatric Disorders in Children and Adolescents
Schizophrenia, Autism-Related Irritability
Not yet recruiting
Sensory-integration Therapy Verses Augumented Reality-based Training in ASD
Autism Spectrum Disorder
Recruiting
Cumulative and Booster Effects of Multisession Prefrontal tDCS in ASD Adolescents
Transcranial Direct Current Stimulation, Autistic Spectrum Disorder
Not yet recruiting
"Family Resilience And Mind-body Enhancement" (FRAME): A Randomised Controlled Trial
Autism

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Evdokia Anagnostou, M.D. principal investigator

Holland Bloorview Kids Rehabilitation Hospital

Suma Jacob, M.D., Ph.D. principal investigator

University of Illinois Chicago

Jessica Brian, Ph.D. principal investigator

Holland Bloorview Kids Rehabilitation Hospital

1 site in 1 country: see every site and its status.

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 Evdokia Anagnostou at 1 site in Canada.

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 7 inclusion and 8 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 12 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in 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. 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 15 Apr 2015). 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?

Maximum Tolerated Dose (MTD) (time frame: 12 Weeks). The study lists 2 primary outcomes in all. 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 NCT01256060.

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 12 Aug 2016.