Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism Spectrum Disorders (ASD)
Condition: Autism Spectrum Disorder
MeSH terms: Autistic Disorder; Autism Spectrum Disorder
Intervention: Intranasal Oxytocin; Placebo
Intervention MeSH terms: Oxytocin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 60.0
Sex: All
Minimum age: 10 Years
Maximum age: 17 Years
Healthy volunteers: No
Keywords: Autism; Social; Functioning; Cognition; Repetitive Behaviors; Anxiety; Quality of Life

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.
    

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. 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).

          3. Have a Clinician's Global Impression-Severity (CGI-S) score ≥ 4 (moderately ill) at
             Screening.

          4. Verbal and performance scale Intelligence Quotient (IQ) ≥ 70 (both subtests of the
             Wechsler Abbreviated Scale of Intelligence (WASI-I or WASI-II ≥ 70).

          5. 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.

          6. 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.

          7. 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.

          8. Ability to speak and understand English sufficiently to allow for the completion of
             all study assessments.

          9. 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.

        Exclusion Criteria

          1. Patients born prior to 35 weeks gestational age.

          2. Patients with a primary psychiatric diagnosis other than ASD.

          3. 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.

          4. 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.

          5. 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 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.

          7. Patients who are currently taking oxytocin or have taken intranasal oxytocin in the
             past with no response.

          8. Patients with a sensitivity to oxytocin or any components of its formulation.

          9. Patients unable to tolerate venipuncture procedures for blood sampling.

         10. Patients in foster care for whom the province/state is defined as a legal guardian