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Intranasal Oxytocin for the Treatment of Children and Adolescents With ASD (OXY)

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ClinicalTrials.gov Identifier: NCT01256060
Recruitment Status : Completed
First Posted : December 8, 2010
Results First Posted : April 15, 2015
Last Update Posted : August 12, 2016
Sponsor:
Collaborators:
Holland Bloorview Kids Rehabilitation Hospital
The Hospital for Sick Children
University of Illinois at Chicago
Information provided by (Responsible Party):
Evdokia Anagnostou, Anagnostou, Evdokia, M.D.

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Autism Spectrum Disorder
Intervention Drug: Intranasal Oxytocin
Enrollment 15
Recruitment Details Participants were recruited from the hospital clinical database, as well as presentations at local conferences and media. Diagnosis was established using the Diagnostic and Statistical Manual, Fourth Edition for an Autism Spectrum Disorder supported by the Autism Diagnostic Observations Schedule and the Autism Diagnostic Interview - Revised.
Pre-assignment Details Cohort 1 Dosage: 0.20 IU/kg - 3 participants Cohort 2 Dosage: 0.26 IU/kg - 3 participants Cohort 3 Dosage: 0.33 IU/kg - 3 participants Cohort 4 Dosage: 0.40 IU/kg - 6 participants
Arm/Group Title 0.2 IU / kg 0.26 IU / kg 0.33 IU / kg 0.4 IU / kg
Hide Arm/Group Description [Not Specified] [Not Specified] [Not Specified] [Not Specified]
Period Title: Overall Study
Started 3 3 3 6
Completed 3 2 3 6
Not Completed 0 1 0 0
Reason Not Completed
Lost to Follow-up             0             1             0             0
Arm/Group Title Intanasal Oxytocin
Hide Arm/Group Description

A modified dose finding method was used to determine safety among four dose levels. Half the dose (0.2 IU/kg /dose) was the minimum dose and two intermediate doses were also evaluated (0.26 and 0.33 IU/kg / dose) Dose-finding escalations were done in groups of three patients.

  1. Three patients were studied at the first dose level
  2. If none of these patients experienced dose limiting toxicity, the dose was escalated.
  3. If one patient experienced dose limiting toxicity, up to three more patients were accrued at the same level. (a) If none of these patients experienced dose limiting toxicity, the dose was escalated. (b) If one or more experienced dose-limiting toxicity, entry at that dose level would be stopped, the maximum tolerated dose exceeded, and dose escalation would be stopped. Up to three more patients would be treated at the next lower dose. If zero out of three patients experience dose limiting toxicity, an additional three patients were to be treated at that dose.
Overall Number of Baseline Participants 15
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants
<=18 years
15
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants
Female
4
  26.7%
Male
11
  73.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Canada Number Analyzed 15 participants
15
1.Primary Outcome
Title Maximum Tolerated Dose (MTD)
Hide Description The hypothesis is that the maximum tolerated dose in a range of 0.2-0.4 IU/kg / dose will be 0.4 IU/kg / dose, as was the case in the adult study, given that oxytocin is not stored in body fat and does not depend on liver or renal clearance.
Time Frame 12 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intanasal Oxytocin
Hide Arm/Group Description:
Cohort 1 Dosage: 0.20 IU/kg - 3 participants Cohort 2 Dosage: 0.26 IU/kg - 3 participants Cohort 3 Dosage: 0.33 IU/kg - 3 participants Cohort 4 Dosage: 0.40 IU/kg - 6 participants
Overall Number of Participants Analyzed 15
Measure Type: Number
Unit of Measure: IU / kg
0.40
2.Primary Outcome
Title Number of Participants With Serious Adverse Events
Hide Description This will be reported as the number of participants who experienced a serious advert event throughout the study.
Time Frame 24 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intanasal Oxytocin
Hide Arm/Group Description:
Number of Participants Experiencing a Serious Adverse Event
Overall Number of Participants Analyzed 15
Measure Type: Number
Unit of Measure: participants
0
3.Secondary Outcome
Title Baseline Levels of Oxytocin in Relation to Either Safety or Treatment Response
Hide Description Children and adolescents with lower plasma oxytocin levels at baseline will show treatment related changes in social cognition. Children and adolescents with higher oxytocin plasma levels will show diminished or less dramatic treatment responses and may have more difficulty tolerating the treatment.
Time Frame 12 Weeks
Outcome Measure Data Not Reported
4.Secondary Outcome
Title Blood Levels of Oxytocin During the Trial in Relation to Safety or Treatment Response
Hide Description Children and adolescents with minimal changes in plasma level of oxytocin after treatment will be less responsive to treatment. Children and adolescents with atypical patterns of increase in oxytocin may be more sensitive to dose-related tolerability.
Time Frame 12 Weeks
Outcome Measure Data Not Reported
5.Other Pre-specified Outcome
Title Changes in Measures of Social Cognition, Social Function, Repetitive Behaviors, and Anxiety (Baseline to Week 12)
Hide Description

Social Cognition (higher score=positive response)

  1. Let's Face It Skills Battery; i. Matchmaker (0-100); ii. Faces (0-100); iii. Houses (0-100)
  2. Eyes Test (0-28)
  3. Strange Stories (0-16)
  4. Irony and Empathy (0-24)

Social Function

  1. Aberrant Behavior Checklist (0-48) (lower score=positive response)
  2. Behavioral Assessment System for Children (higher score=positive response); i. Social: age 8 to 11 & 15 to 18 (18-69); age 12 to 14 (21-70); ii. Functional: age 8 to 14 (10-66); age 15 to 18 (10-64)
  3. Social Responsiveness Scale (higher score=positive response); male (34-127); female (35-142)

Anxiety (lower score=positive response)

a. Child Symptom Inventory; i. Separation: male (44-106); female (44-101); ii. Generalized: male (40-101); female (41-96)

Repetitive Behaviors (lower score=positive response)

  1. Child Yale-Brown Obsessive-Compulsive Scale (0-20)
  2. Repetitive Behavior Scale (0-129)

Measures insensitive to change will be omitted from results.

Time Frame 12 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intranasal Oxytocin
Hide Arm/Group Description:
Oxytocin: Intranasal Oxytocin
Overall Number of Participants Analyzed 15
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
Let's Face It! Skills Battery-Matchmaker
-10.9
(-19.3 to -2.6)
Let's Face It! Skills Battery-Faces
-9.21
(-15.26 to -3.15)
Let's Face It! Skills Battery-Houses
-10.73
(-18.5 to -2.91)
The Revised Eyes Test
-0.14
(-0.82 to 0.53)
Strange Stories Task
-1.36
(-2.72 to 0.0008)
Irony and Empathy Task
-1.64
(-3.02 to -0.27)
Aberrant Behavior Checklist
3.00
(0.05 to 5.96)
Social Responsiveness Scale
9.8
(3.8 to 15.8)
Behavioral Assessment System for Children-Social
-2.84
(-5.31 to -0.38)
Behavioral Assessment System for Children-Function
-4.10
(-6.23 to -1.97)
Child Yale-Brown Obsessive-Compulsive Scale
2.93
(0.95 to 4.91)
Repetitive Behavior Scale-Revised
17.3
(6.3 to 28.3)
Child and Adolescent Symptom Inventory-Separation
7.5
(0.2 to 14.8)
Child and Adolescent Symptom Inventory-General
11.0
(4.0 to 18.0)
6.Other Pre-specified Outcome
Title Measures of Social Function - The Clinical Global Impressions - Social Scale (Baseline to Week 12)
Hide Description

Social Function

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).

Time Frame 12 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intranasal Oxytocin
Hide Arm/Group Description:
Oxytocin: Intranasal Oxytocin
Overall Number of Participants Analyzed 15
Measure Type: Number
Unit of Measure: participants
7
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Intranasal Oxytocin
Hide Arm/Group Description Oxytocin: Intranasal Oxytocin. Please note that the Adverse Events are not presented per dose level received, as this is not a dose-finding study, it is a modified maximum tolerated dose study. This means that a small number of participants were exposed to increasing dose levels to assess for Adverse Events. It is not meaningful to analyze adverse events by cohorts due to the extremely small sample size.
All-Cause Mortality
Intranasal Oxytocin
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Intranasal Oxytocin
Affected / at Risk (%) # Events
Total   0/15 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Intranasal Oxytocin
Affected / at Risk (%) # Events
Total   15/15 (100.00%)    
Gastrointestinal disorders   
Abdominal Discomfort  3/15 (20.00%)  3
Nausea  1/15 (6.67%)  2
General disorders   
Fatigue  4/15 (26.67%)  5
Tooth Sensitivity/Pain  2/15 (13.33%)  2
Infections and infestations   
Upper Respiratory Infections  6/15 (40.00%)  7
Metabolism and nutrition disorders   
Decrease Appetite  3/15 (20.00%)  5
Nervous system disorders   
Emotional Lability   6/15 (40.00%)  7
Headache  7/15 (46.67%)  11
Migraine  1/15 (6.67%)  3
Psychiatric disorders   
Irritability  4/15 (26.67%)  6
Respiratory, thoracic and mediastinal disorders   
Asthma Attack  1/15 (6.67%)  2
Skin and subcutaneous tissue disorders   
Rash  3/15 (20.00%)  3
Itchy Nose  1/15 (6.67%)  2
Indicates events were collected by systematic assessment
The sample size is too small to view both safety and efficacy data. The design did not exceed the commonly used 24 IU / dose, so it is not clear that higher doses would not confer benefit. In the absence of placebo, adverse events may be inflated.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Evdokia Anagnostou
Organization: Holland Bloorview Kids Rehabilitation Hospital
Phone: 416-753-6005
EMail: eanagnostou@hollandbloorview.ca
Layout table for additonal information
Responsible Party: Evdokia Anagnostou, Anagnostou, Evdokia, M.D.
ClinicalTrials.gov Identifier: NCT01256060     History of Changes
Other Study ID Numbers: 10-001
First Submitted: November 22, 2010
First Posted: December 8, 2010
Results First Submitted: January 13, 2015
Results First Posted: April 15, 2015
Last Update Posted: August 12, 2016