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Trial · Interventional

CompletedPhase 2Results postedRegistry data

Intranasal Oxytocin Treatment for Social Deficits in Children With Autism

NCTNCT01624194 (opens ClinicalTrials.gov in a new tab)Other idSU-12132011-8827
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 2
Enrollment
54 participants (actual)
Sponsor
Stanford University · Other
Design
Randomized · Quadruple masking
Countries
1 country

United States

Start
Jun 2012
Primary completion
May 2016
Completion
May 2016
First posted
20 Jun 2012 (estimated)
Last update posted
15 Jul 2019
Conditions
Autism

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
201220132014201520162017
  1. Ph 2

    Intranasal Oxytocin Treatment for Social Deficits in Children With Autism

    NCT01624194

    Jun 2012 to May 2016

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • Oxytocin nasal sprayDrug
  • PlaceboDrug

Arms2

  • Active comparator

    Oxytocin nasal spray

    Prior to randomization, all subjects will participate in a 1-week open-label placebo lead-in trial. Each subject will be administered the placebo nasal spray at Stanford University and then their parent will continue administering the nasal spray to the subject for 1 week at home. Each subject will then be randomly assigned either to the active group or to the placebo (stratified by gender) and will be given the appropriate nasal spray bottle and their parents will be responsible for administering 3 puffs per nostril (4 IU/puff) to their child for a total dose of 24 IU oxytocin or placebo twice daily (BID; morning and evening) for 4-weeks. On completion of this 4-week treatment trial subjects will have the option of participating in a second double-blind trial in which they will be assigned to the alternate nasal spray, to that which they received during the first 4-week trial, for an additional 4-week period.

  • Placebo comparator

    Placebo nasal spray

    The placebo nasal spray bottles will be prepared by adding all of the ingredients used in the Syntocinon nasal sprays with the exception of the concentrated oxytocin solution.

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Change From Baseline in Parent Rated Social Responsiveness Scale (SRS) Scores During Treatment.

    Time frame · Baseline; Week 4

    Registry description

    Social Responsiveness Scale (SRS) raw scores measure social abilities with lower raw scores meaning better social abilities. (Raw Score Range: 0 - 195)

Secondary outcomes(16)

  1. Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment

    Time frame · Baseline through Week 4

    Registry description

    Dosage Record Treatment Emergent Symptom Scale (DOTES) side effects reported by parents during 4-weeks of treatment. Participant Counts are used.

  2. Change From Baseline in Height.

    Time frame · Baseline; Week 4

  3. Clinical Global Impression-Improvement (CGI-I) Score at Week 4

    Time frame · Baseline to Week 4

    Registry description

    This outcome is reported as the count of participants in each CGI-I rating category at the week 4 visit, assessing change over the 4-week period. CGI-I rating of 1=Very Much Improved, 2=Much Improved, 3=Minimally Improved, 4=No Change, 5=Minimally Worse, 6=Much Worse, and 7=Very Much Worse.

  4. Parent Rated Aberrant Behavior Checklist (ABC) Irritability Scores at Baseline and Week 4

    Time frame · Baseline; Week 4

    Registry description

    Higher scores indicate more symptoms, lower scores indicate fewer symptoms. Irritability scores can range from 0-45. Lethargy scores can range from 0-48. Stereotypy scores can range from 0-21. Hyperactivity scores can range from 0-48. Inappropriate speech scores can from 0-12.

  5. Change From Baseline in Parent Rated Spence Children's Anxiety Scale (SCAS) During Treatment.

    Time frame · Baseline; Week 4

    Registry description

    Scale measuring severity of anxiety symptoms. Higher scores mean higher levels of anxiety, lower scores mean lower levels of anxiety. (Raw Score Range: 0 - 114)

  6. Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition - Social and Communication Subscales During Treatment.

    Time frame · Baseline; Week 4

    Registry description

    Higher Social Standard Score means better social skills, lower Social Standard Score means worse social skills. Higher Communication Standard Score means better communication skills, lower Communication Standard Score means worse communication skills. Standard Scores can range from 20 to 160.

  7. Change From Baseline in Laboratory Based Facial Emotion Recognition Abilities During Treatment.

    Time frame · Up to 4 weeks

  8. Change From Baseline in Laboratory Based Eye-gaze to Social Cues During Treatment.

    Time frame · Baseline; Week 4

  9. Change From Baseline in Reading the Mind in the Eyes Test, Child Version (RMET-child) Scores During Treatment.

    Time frame · Up to 4 weeks

  10. Change From Baseline in Laboratory Based Social Mimicry Abilities During Treatment.

    Time frame · Up to 4 weeks

  11. Change From Baseline in Developmental NEuroPSYchological Assessment (NEPSY-II) Affect Recognition Scores During Treatment.

    Time frame · Baseline; Week 4

    Registry description

    Higher Affect Recognition scores mean better affect recognition abilities, lower Affect Recognition scores mean worse affect recognition abilities. Scores can range from 1 to 19.

  12. Change From Baseline in Plasma Oxytocin Levels During Treatment.

    Time frame · Up to 4 weeks

    Registry description

    This outcome originally specified that oxytocin, vasopressin, and cortisol levels would be assessed; however, data on vasopressin and cortisol levels were not collected during the study. There are no clinical laboratory tests that establish a normative range for oxytocin. Measurements prior to and following treatment were intended to evaluate oxytocin level as a predictor of response.

  13. Change From Baseline in Parent Rated Repetitive Behavior Scale- Revised (RBS-R) Scores During Treatment.

    Time frame · Baseline; Week 4

    Registry description

    Higher scores on the Repetitive Behavior Scale- Revised mean higher levels of repetitive and restricted behaviors. (Raw Score Total Range: 0 - 129)

  14. Change From Baseline in Weight

    Time frame · Baseline; Week 4

  15. Change From Baseline in Heart Rate

    Time frame · Baseline; Week 4

  16. Change From Baseline in Blood Pressure

    Time frame · Baseline; Week 4

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeLeast squares mean· Baseline; Week 4

Change From Baseline in Parent Rated Social Responsiveness Scale (SRS) Scores During Treatment.

GroupValue (units on a scale)Spread
Oxytocin Nasal Spray9.732.64
Placebo Nasal Spray3.182.18
Secondary and other outcome results(7)
Secondary outcomeNumber· Baseline through Week 4

Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment

GroupValue (participants)
Oxytocin Nasal Spray0
Placebo Nasal Spray1
Secondary outcomeLeast squares mean· Baseline; Week 4

Change From Baseline in Height.

GroupValue (cm)
Oxytocin Nasal Spray134
Placebo Nasal Spray129
Secondary outcomeCount of participants· Baseline to Week 4

Clinical Global Impression-Improvement (CGI-I) Score at Week 4

GroupValue (Participants)
Oxytocin Nasal Spray0
Placebo Nasal Spray0
Secondary outcomeMean· Baseline; Week 4

Parent Rated Aberrant Behavior Checklist (ABC) Irritability Scores at Baseline and Week 4

GroupValue (units on a scale)Spread
Oxytocin Nasal Spray13.2011.91
Placebo Nasal Spray13.948.71
Secondary outcomeLeast squares mean· Baseline; Week 4

Change From Baseline in Parent Rated Spence Children's Anxiety Scale (SCAS) During Treatment.

GroupValue (units on a scale)Spread
Oxytocin Nasal Spray1.663.05
Placebo Nasal Spray4.532.21
Secondary outcomeMean· Baseline; Week 4

Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition - Social and Communication Subscales During Treatment.

GroupValue (units on a scale)Spread
Oxytocin Nasal Spray61.412.82
Placebo Nasal Spray67.7512.45
Secondary outcome· Up to 4 weeks

Change From Baseline in Laboratory Based Facial Emotion Recognition Abilities During Treatment.

GroupValue
Oxytocin Nasal Spraynot posted
Placebo Nasal Spraynot posted

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Oxytocin Nasal SprayOxytocin nasal spray: 24IU BID (3 x 0.1 mL \[4IU\] sprays per nostril twice for 4-weeks.0 / 140 / 1411 / 14
Placebo Nasal SprayPlacebo: 3 x 0.1 mL sprays per nostril twice daily for 4-weeks.0 / 180 / 1812 / 18

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies1

Studies that cite this trial.

Studies with results

1 study on this page

Indexing hint: Randomized controlled trial · 2017

6 results

Intranasal oxytocin treatment for social deficits and biomarkers of response in children with autism.

Parker et al. 2017Proceedings of the National Academy of Sciences of the United States of Americacited by 287

PMID 28696286PMC5544319DOI 10.1073/pnas.1705521114PUB-28696286

Rights unknown6 results
Source linkSee results (6)

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

  • DerivedIffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2. PMID 37811711

Eligibility

Who could join, as posted.

Sex
All
Age
from 6 Years up to 12 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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.

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