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Phase 2 N=34 Randomized Quadruple-blind Treatment

Intranasal Oxytocin Treatment for Social Deficits in Children With Autism

Autism

Enrolled (actual)
34
Serious AEs
0.0%
Results posted
Jun 2018
Primary outcome: Primary: Change From Baseline in Parent Rated Social Responsiveness Scale (SRS) Scores During Treatment. — 9.73; 3.18 units on a scale — p=0.0275

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Oxytocin nasal spray (Drug); Placebo (Drug)
Age
Pediatric · 6+ yrs
Sex
All
Sponsor
Stanford University
Primary completion
May 2016

Outcome Measures

OutcomeResultp-value
PRIMARY
Change From Baseline in Parent Rated Social Responsiveness Scale (SRS) Scores During Treatment.
9.73; 3.18 0.0275 sig
SECONDARY
Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment
0; 1; 0; 1; 1; 0 >0.05
SECONDARY
Change From Baseline in Height.
134; 129; 134; 129 >0.05
SECONDARY
Clinical Global Impression-Improvement (CGI-I) Score at Week 4
0; 0; 3; 3; 2; 5
SECONDARY
Parent Rated Aberrant Behavior Checklist (ABC) Irritability Scores at Baseline and Week 4
13.20; 13.94; 8.86; 11.78; 14.13; 11.7
SECONDARY
Change From Baseline in Parent Rated Spence Children's Anxiety Scale (SCAS) During Treatment.
1.66; 4.53 0.3395
SECONDARY
Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition - Social and Communication Subscales During Treatment.
61.4; 67.75; 66.11; 70.92; 66.91; 71.80
SECONDARY
Change From Baseline in Laboratory Based Facial Emotion Recognition Abilities During Treatment.
SECONDARY
Change From Baseline in Laboratory Based Eye-gaze to Social Cues During Treatment.
SECONDARY
Change From Baseline in Reading the Mind in the Eyes Test, Child Version (RMET-child) Scores During Treatment.
SECONDARY
Change From Baseline in Laboratory Based Social Mimicry Abilities During Treatment.
SECONDARY
Change From Baseline in Developmental NEuroPSYchological Assessment (NEPSY-II) Affect Recognition Scores During Treatment.
10; 9.43; 8.75; 6.43
SECONDARY
Change From Baseline in Plasma Oxytocin Levels During Treatment.
8.82; 8.66; 10.36; 8.40
SECONDARY
Change From Baseline in Parent Rated Repetitive Behavior Scale- Revised (RBS-R) Scores During Treatment.
5.68; 5.79 0.9757
SECONDARY
Change From Baseline in Weight
32.7; 28.0; 33.5; 28.4 >0.05
SECONDARY
Change From Baseline in Heart Rate
92.5; 92.7; 97.1; 101.6 >0.05
SECONDARY
Change From Baseline in Blood Pressure
104.2; 101.0; 111.1; 110.9; 64.3; 64.6 >0.05

Summary

Autism is a pervasive developmental disorder characterized by core deficits in social behavior and communication, and the presence of repetitive or stereotyped behaviors. It is one of three recognized disorders in the autism spectrum which affects an estimated 1 in 88 children in the United States. At present, pharmacotherapies target only associated features of autism, with no effective drug treatments for the social impairments. Several lines of evidence now suggest that the neuropeptide oxytocin (OT) may be an effective treatment for the core social deficits in autism. Here we will test the effects of twice daily intranasal OT (24 IU) over a 4-week period for enhancing social deficits in male and female children aged 6-12 years with autism. This research has high potential to lead to the development of more effective treatments and earlier interventions for children with autism.

Eligibility Criteria

Inclusion Criteria

  • Medically healthy outpatients between 6 and 12 years of age (cut off 12 years and 11 months)
  • Intelligence Quotient > 40
  • Diagnosis of autism spectrum disorder based on the Autism Diagnostic Interview - Revised, Autism Diagnostic Observation Schedule, and DSM-IV criteria
  • Clinical Global Impression severity rating of 4 or higher
  • Care provider who can reliably bring subject to clinic visits, provide trustworthy ratings, and interacts with the subject on a regular basis
  • Stable medications for at least 4 weeks
  • No planned changes in psychosocial interventions during the trial
  • Willingness to provide blood samples.

Exclusion Criteria

  • Diagnostics and Statistical Manual of Mental Disorders, 4th edition (DSM-IV) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder
  • Regular nasal obstruction or nosebleeds
  • Active medical problems: unstable seizures, significant physical illness (e.g., serious liver, renal, or cardiac pathology)
  • Sensitivity to preservatives (in particular E 216, E 218, and chlorobutanol hemihydrate)
  • A genetic abnormality (e.g., Fragile X Syndrome)
  • Significant hearing or vision impairments
  • Habitually drinks large volumes of water
  • Pregnancy, breastfeeding, or child birth within the last 6 months
  • Sexually active females not using a reliable method of contraception.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01624194). 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.

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