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

This study has been completed.
Sponsor:
Collaborator:
United States Department of Defense
Information provided by (Responsible Party):
Evdokia Anagnostou, Anagnostou, Evdokia, M.D.
ClinicalTrials.gov Identifier:
NCT01908205
First received: July 23, 2013
Last updated: April 12, 2016
Last verified: April 2016
  Purpose
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.

Condition Intervention Phase
Autism Spectrum Disorder
Drug: Intranasal Oxytocin
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism

Resource links provided by NLM:


Further study details as provided by Anagnostou, Evdokia, M.D.:

Primary Outcome Measures:
  • Efficacy of intranasal oxytocin vs. placebo on measures of social function [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
    This will be measured by the Aberrant Behavior Checklist (ABC) - Social Withdrawal Subscale


Secondary Outcome Measures:
  • Efficacy of intranasal oxytocin vs. placebo on measures of social cognition [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
    This will be measured by the Revised Eyes Test

  • Efficacy of intranasal oxytocin vs. placebo on measures of social cognition [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: Yes ]
    This will be measured by the Safety Monitoring Uniform Report Form (SMURF)


Enrollment: 60
Study Start Date: September 2013
Study Completion Date: March 2016
Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Intranasal Oxytocin (Syntocinon)
The proposed dosing schedule is 0.4 IU/kg, taken twice daily, for a maximum of 24 IUs per dose
Drug: Intranasal Oxytocin
Other Name: Syntocinon
Placebo Comparator: Placebo
The proposed dosing schedule is 0.4 IU/kg, taken twice daily, for a maximum of 24 IUs per dose
Drug: Placebo

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.
  Eligibility

Ages Eligible for Study:   10 Years to 17 Years   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01908205

Locations
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Canada, Ontario
Holland Bloorview Kids Rehabilitation Hospital
Toronto, Ontario, Canada, M4G 1R8
Sponsors and Collaborators
Evdokia Anagnostou
United States Department of Defense
Investigators
Principal Investigator: Evdokia Anagnostou, M.D. Holland Bloorview Kids Rehabilitation Hospital
Principal Investigator: Suma Jacob, M.D., Ph.D. University of Minnesota - Clinical and Translational Science Institute
  More Information

Responsible Party: Evdokia Anagnostou, Principal Investigator, Anagnostou, Evdokia, M.D.
ClinicalTrials.gov Identifier: NCT01908205     History of Changes
Other Study ID Numbers: OXY-R07-2013 
Study First Received: July 23, 2013
Last Updated: April 12, 2016
Health Authority: Canada: Health Canada
United States: Food and Drug Administration
Individual Participant Data  
Plan to Share IPD: No

Keywords provided by Anagnostou, Evdokia, M.D.:
Autism
Social
Functioning
Cognition
Repetitive Behaviors
Anxiety
Quality of Life

Additional relevant MeSH terms:
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
Oxytocin
Oxytocics
Reproductive Control Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 29, 2016