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

Overview

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.

Full Title of Study: “Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: September 2015

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.

Interventions

  • Drug: Intranasal Oxytocin
  • Drug: Placebo

Arms, Groups and Cohorts

  • Experimental: Intranasal Oxytocin (Syntocinon)
    • The proposed dosing schedule is 0.4 IU/kg, taken twice daily, for a maximum of 24 IUs per dose
  • Placebo Comparator: Placebo
    • The proposed dosing schedule is 0.4 IU/kg, taken twice daily, for a maximum of 24 IUs per dose

Clinical Trial Outcome Measures

Primary Measures

  • Efficacy of intranasal oxytocin vs. placebo on measures of social function
    • Time Frame: 12 Weeks
    • This will be measured by the Aberrant Behavior Checklist (ABC) – Social Withdrawal Subscale

Secondary Measures

  • Efficacy of intranasal oxytocin vs. placebo on measures of social cognition
    • Time Frame: 12 Weeks
    • This will be measured by the Revised Eyes Test
  • Efficacy of intranasal oxytocin vs. placebo on measures of social cognition
    • Time Frame: 12 Weeks
    • This will be measured by the Let’s Face it! Skills Battery
  • Efficacy of intranasal oxytocin vs. placebo on measures of social function
    • Time Frame: 12 Weeks
    • This will be measured by the Behavioral Assessment System for Children (BASC-2)
  • Efficacy of intranasal oxytocin vs. placebo on measures of social function
    • Time Frame: 12 Weeks
    • This will be measured by the Clinical Global Impressions – Improvement Scale – Social (CGI-I-Social)
  • Efficacy of intranasal oxytocin vs. placebo on measures of repetitive behaviors
    • Time Frame: 12 Weeks
    • This will be measured by the Child Yale-Brown Obsessive-Compulsive Scale (CY-BOCS)
  • Efficacy of intranasal oxytocin vs. placebo on measures of repetitive behaviors
    • Time Frame: 12 Weeks
    • This will be measured by the Repetitive Behavior Scale (RBS-R)
  • Efficacy of intranasal oxytocin vs. placebo on measures of anxiety
    • Time Frame: 12 Weeks
    • This will be measured by the Child and Adolescent Symptom Inventory (CASI-4R)
  • Efficacy of intranasal oxytocin vs. placebo on measures of quality of life
    • Time Frame: 12 Weeks
    • This will be measured by the Pediatric Quality of Life Inventory (PedsQL)
  • Safety and tolerability of intranasal oxytocin in children and adolescents with ASD
    • Time Frame: 12 Weeks
    • This will be measured by the Clinical Global Impressions – Improvement Scale – Global (CGI-I-Global)
  • Safety and tolerability of intranasal oxytocin in children and adolescents with ASD
    • Time Frame: 12 Weeks
    • This will be measured by the Safety Monitoring Uniform Report Form (SMURF)

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 10 Years

Maximum Age: 17 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Evdokia Anagnostou
  • Collaborator
    • United States Department of Defense
  • Provider of Information About this Clinical Study
    • Sponsor-Investigator: Evdokia Anagnostou, Principal Investigator – Anagnostou, Evdokia, M.D.
  • Overall Official(s)
    • Evdokia Anagnostou, M.D., Principal Investigator, Holland Bloorview Kids Rehabilitation Hospital
    • Suma Jacob, M.D., Ph.D., Principal Investigator, University of Minnesota

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