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

Phase 2

Completed

Conditions: Autism Spectrum Disorder

Interventions: Drug: Intranasal Oxytocin
Drug: Placebo

Registration Number: NCT01908205

Lead Sponsor: Evdokia Anagnostou

Brief 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.

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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 60

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Intranasal Oxytocin (Syntocinon)	Intranasal Oxytocin	The proposed dosing schedule is 0.4 IU/kg, taken twice daily, for a maximum of 24 IUs per dose
Placebo	Placebo	The proposed dosing schedule is 0.4 IU/kg, taken twice daily, for a maximum of 24 IUs per dose

Primary Outcome Measures

Name	Time	Method
Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Function	12 and 24 weeks	This will be measured by a change in score on the Aberrant Behavior Checklist (ABC) - Social Withdrawal Subscale (0-48, where lower scores indicate improvement)

Secondary Outcome Measures

Name	Time	Method
Number of Participant Considered Social Responders	12 Weeks	This will be measured by the Clinical Global Impressions - Improvement Scale - Social (CGI-I-Social) a) Clinical Global Impressions - Social Scale (1-7) (lower score=positive response). The results will be reported as the number of participants that were classified as a social responder (achieving a score of 1 or 2 on the scale).
Number of Participant Considered Overall Responders	12 Weeks	This will be measured by the Clinical Global Impressions - Improvement Scale - Global (CGI-I-Global) (1-7) (lower score=positive response). The results will be reported as the number of participants that were classified as an overall responder (achieving a score of 1 or 2 on the scale).
Safety and Tolerability of Intranasal Oxytocin in Children and Adolescents With ASD	12 Weeks	This will be measured by the Safety Monitoring Uniform Report Form (SMURF)
Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Cognition	12 Weeks	This will be measured by improvement on the Let's Face it! Skills Battery from Baseline to Week 12 Social Cognition (higher score=better outcome) a. Let's Face It Skills Battery; i. Matchmaker (0-100); ii. Faces (0-100); iii. Houses (0-100)
Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Function	12 Weeks	This will be measured by improvement on the Behavioral Assessment System for Children (BASC-2) from Baseline to Week 12 Behavioral Assessment System for Children (higher score=positive response); i. \Social Skills: age 6 to 11 (18-69); age 12 to 17 (21-70); ii. Functional Communication: age 6 to 11 (10-66); age 12 to 17 (10-64); iii. Withdrawal age 6-11 (21- 62) ; age 12-17 (14-42) \ only social subscales of BASC-2 reported
Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Anxiety	12 Weeks	This will be measured by the Child and Adolescent Symptom Inventory (CASI-4R) Generalized anxiety score (male (40-101); female (41-96) -where lower score= positive response) from Baseline to Week 12
Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Repetitive Behaviors	12 Weeks	This will be measured by improvement on the Repetitive Behavior Scale (RBS-R)(0-129; where lower score= positive response) from Baseline to Week 12
Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Quality of Life	12 Weeks	This will be measured by improvement on the Pediatric Quality of Life Inventory (PedsQL) (0-100, where higher scores indicate positive response) from Baseline to Week 12

Trial Locations

Locations (2): University of Minnesota
🇺🇸
Minneapolis, Minnesota, United States
Holland Bloorview Kids Rehabilitation Hospital
🇨🇦
Toronto, Ontario, Canada