Intranasal Oxytocin for the Treatment of Children and Adolescents With ASD (OXY)
Condition: Autism Spectrum Disorder
MeSH terms: Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive
Intervention: Intranasal Oxytocin
Intervention description: 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.
Intervention MeSH terms: Oxytocin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 15.0
Sex: All
Minimum age: 10 Years
Maximum age: 17 Years
Healthy volunteers: No
Keywords: Autism Spectrum disorder; Oxytocin; Clinical Trial; Children; Pharmacology

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.