Phase 2 N=290 Randomized Quadruple-blind Treatment

Study of Oxytocin in Autism to Improve Reciprocal Social Behaviors

Autism Spectrum Disorders

Bottom Line

View on ClinicalTrials.gov: NCT01944046 ↗

Enrolled (actual)

290

Serious AEs

1.3%

Results posted

Jan 2021

Primary outcome: Primary: Change in Aberrant Behavior Checklist-Modified Social Withdrawal Subscale ABC-mSW, a Measure of Social Reciprocity — 8.81; 8.68; 8.97; 8.68 score on a scale — p=0.503

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Double blind phase Placebo Nasal Spray (Drug); double Blind Oxytocin Nasal Spray (Drug); Open Label intranasal oxytocin (Drug)
Age: Pediatric · 3+ yrs
Sex: All
Sponsor: Linmarie Sikich
Primary completion: Nov 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY Change in Aberrant Behavior Checklist-Modified Social Withdrawal Subscale ABC-mSW, a Measure of Social Reciprocity	7.83; 7.81; 6.99; 6.78	0.61
PRIMARY Change in Aberrant Behavior Checklist-Modified Social Withdrawal Subscale ABC-mSW, a Measure of Social Reciprocity	7.83; 7.81; 6.99; 6.78	0.61
SECONDARY Change in Sociability Factor (SF)	35.8; 36.1; -5.57; -5.81; -5.99; -6.30	—
SECONDARY Change in Social Responsiveness Scale-2 (SRS-2) Social Motivation Subscale Score	-4.58; -5.72; -4.49; -5.42	—
SECONDARY Change in Stanford Binet-5th Edition (SB-5) IQ Score	0.40; 1.0	—

Summary

The purpose of this research study is to learn about the effects of supplemental intranasal oxytocin as a treatment for improving social difficulties in children and adolescents with autism. This study will also provide additional information about the safety and tolerability of intranasal oxytocin. Investigators expect oxytocin will increase social motivation, improving daily living skills and quality of life.

Eligibility Criteria

Inclusion Criteria

Be between the ages of 3 years 0 months and 17 years 11 months at the time of randomization
Be diagnosed by clinician experienced in assessment of ASD with autistic disorder, Asperger's syndrome, or PDD-NOS using DSM-V-TR criteria
Must have clinical diagnosis of ASD confirmed using the Autism Diagnostic Observation Scale (ADOS, Lord et al., 2001)
Must have clinical diagnosis of ASD confirmed using the Autism Diagnostic Interview-Revised (ADI-R, Rutter, 2003). ASD criteria proposed by Risi (2006). Specifically, subject must be within 1 point of autism criteria on both social and communication domains of the ADI or meet autism criteria in one of these ADI domains and come within 2 points of autism criteria in the other
Have a guardian who is able to provide informed consent
If cognitively able, subject must be able to provide informed assent/consent

Exclusion Criteria

Have a known diagnosis of Rett Syndrome or Childhood Disintegrative Disorder, or have marked sensory impairment such as deafness or blindness
Have active cardiovascular disease or renal disease that is not controlled by medication
Subjects who are pregnant, lactating, or who refuse to practice contraception if sexually active
Subjects who have had changes in allied health therapies, behavioral or educational interventions within the two months prior to randomization other than those associated with school holidays
Subjects who have had changes in psychiatric medications within 4 weeks of randomization
Subjects who have had previous chronic treatment with oxytocin
Subjects who have caretakers who are unable to speak English, be consistently present at visits to report on symptoms, or are otherwise judged as unable to comply with the protocol by the data collection site team
Subjects with active seizures within the 6 months preceding screening or baseline -added part way through study in response to subject death.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01944046). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.