Clinical trial NCT01256060: Intranasal Oxytocin for the Treatment of Children and Adolescents With ASD (OXY)
Official title: Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism Spectrum Disorders (ASD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Keywords: Autism Spectrum disorder
Oxytocin
Clinical Trial
Children
Pharmacology
Lead sponsor: Anagnostou, Evdokia, M.D.
Sponsor class: INDIV
Collaborators: name: Holland Bloorview Kids Rehabilitation Hospital
name: The Hospital for Sick Children
name: University of Illinois at Chicago
First posted: 2010-12-08
Enrollment: 15
Sex: ALL
Minimum age (years): 10
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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. '


Eligibility criteria: Inclusion Criteria:

1. Male or female outpatients 10-17 years of age inclusive.
2. 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.
3. Have a Clinician's Global Impression-Severity score ≥ 4 (moderately ill) at Baseline.
4. Verbal Intelligent Quotient \>/= 70.
5. 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.
6. Have normal physical examination and laboratory test results at Screening. If abnormal, the finding(s) must be deemed clinically insignificant by the Investigator.
7. The participant and caregiver must be able to speak and understand English sufficiently to allow for the completion of all study assessments.

Exclusion Criteria:

1. Patients born prior to 35 weeks gestational age.
2. Patients with any primary psychiatric diagnosis other than autism at Screening.
3. 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.
4. 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
5. 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.
6. Patients who are sensitive to Syntocinon or any components of its formulation
7. 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.
8. Patients unable to tolerate venipuncture procedures for blood sampling.

Primary outcomes:
measure: Maximum Tolerated Dose (MTD)
timeFrame: 12 Weeks
measure: Number of Participants With Serious Adverse Events
timeFrame: 24 Weeks
Secondary outcomes:
measure: Baseline Levels of Oxytocin in Relation to Either Safety or Treatment Response
timeFrame: 12 Weeks
measure: Blood Levels of Oxytocin During the Trial in Relation to Safety or Treatment Response
timeFrame: 12 Weeks
Other outcomes:
measure: Changes in Measures of Social Cognition, Social Function, Repetitive Behaviors, and Anxiety (Baseline to Week 12)
timeFrame: 12 Weeks
measure: Measures of Social Function - The Clinical Global Impressions - Social Scale (Baseline to Week 12)
timeFrame: 12 Weeks
Locations:
city: Toronto
country: Canada
facility: Holland Bloorview Kids Rehabilitation Hospital
state: Ontario
Study officials:
affiliation: Holland Bloorview Kids Rehabilitation Hospital
name: Evdokia Anagnostou, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Illinois at Chicago
name: Suma Jacob, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Holland Bloorview Kids Rehabilitation Hospital
name: Jessica Brian, Ph.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: The Hospital for Sick Children
name: Wendy Roberts, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Holland Bloorview Kids Rehabilitation Hospital
name: Sharon Smile, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Illinois at Chicago
name: Edwin Cook, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Holland Bloorview Kids Rehabilitation Hospital
name: Annie Dupuis, Ph.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: The Hospital for Sick Children
name: Margot Taylor, Ph.D.
role: PRINCIPAL_INVESTIGATOR
References: