Trial Outcomes & Findings for Intranasal Oxytocin for the Treatment of Autism Spectrum Disorders (NCT NCT01337687)

Results Overview

Rating Scale that utilizes direct observation, scales and patient report to inform clinical judgment. A form of CGI that targets social functioning will be used as the primary outcome measure. Semi-structured interview.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

19 participants

Primary outcome timeframe

6 Weeks

Results posted on

2020-02-27

Participant Flow

Participant milestones

Participant milestones
Measure	Oxytocin Intranasal Oxytocin: Oxytocin administered intranasally twice a day via 1 12 unit puff to each nostril, totaling 48 IU a day.	Placebo Placebo: Saline will be administered intranasally twice a day via 1 puff per nostril, totaling 48 IU a day.
Overall Study STARTED	10	9
Overall Study COMPLETED	10	9
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Intranasal Oxytocin for the Treatment of Autism Spectrum Disorders

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Oxytocin n=10 Participants Intranasal Oxytocin: Oxytocin administered intranasally twice a day via 1 12 unit puff to each nostril, totaling 48 IU a day.	Placebo n=9 Participants Placebo: Saline will be administered intranasally twice a day via 1 puff per nostril, totaling 48 IU a day.	Total n=19 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants	9 Participants n=7 Participants	19 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	33.8 years n=5 Participants	32.9 years n=7 Participants	33.2 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants	7 Participants n=7 Participants	16 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants	9 participants n=7 Participants	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 Weeks

Population: Participants rated as improved on the CGI-I scale

Rating Scale that utilizes direct observation, scales and patient report to inform clinical judgment. A form of CGI that targets social functioning will be used as the primary outcome measure. Semi-structured interview.

Outcome measures

Outcome measures
Measure	Oxytocin n=10 Participants Intranasal Oxytocin: Oxytocin administered intranasally twice a day via 1 12 unit puff to each nostril, totaling 48 IU a day.	Placebo n=9 Participants Placebo: Saline will be administered intranasally twice a day via 1 puff per nostril, totaling 48 IU a day.
Number of Participants With Improvement on the Clinical Global Impression-Improvement (CGI-I) Scale	3 Participants	1 Participants

SECONDARY outcome

Timeframe: Baseline, Week 6

Clinician-Rated questionnaire measuring the time spent, distress,interference, resistance, and control in relation to obsessions and compulsions based on a 5 point scale ranging from 0-5 for each category with 0 being most desirable and 5 being least. Sub-scales are added and averaged to obtain total scores. -scale range (total): 0 - 40 total, 0 - 7 subclinical, 8-15 mild, 16 - 23 moderate, 24 - 31 severe, 32 - 40 extreme Questions are rated on a 5-point scale, 0-4, increasing in severity. Obsession Rating Subscale (5 questions, ranging from 0 - 20 total) and Compulsion Rating subscale (5 questions, ranging from 0 - 20 total) are totaled and added to obtain total scores. -score interpretation: Higher overall scores reflect increasing symptom severity.

Outcome measures

Outcome measures
Measure	Oxytocin n=10 Participants Intranasal Oxytocin: Oxytocin administered intranasally twice a day via 1 12 unit puff to each nostril, totaling 48 IU a day.	Placebo n=9 Participants Placebo: Saline will be administered intranasally twice a day via 1 puff per nostril, totaling 48 IU a day.
Change in Obsessive- Compulsive Behavior Based on Yale Brown Obsessive Compulsive Scale (YBOCS) Baseline	12.0 score on a scale Standard Deviation 3.9	10.3 score on a scale Standard Deviation 2.5
Change in Obsessive- Compulsive Behavior Based on Yale Brown Obsessive Compulsive Scale (YBOCS) Week 6	9.4 score on a scale Standard Deviation 2.9	8.1 score on a scale Standard Deviation 2.5

SECONDARY outcome

Timeframe: Baseline, week 6

Subscale ranges: stereotypic behavior (0 - 27);self-injurious behavior (0 - 24); compulsive behavior (0 - 18); ritualistic/Sameness Behavior (0 - 36); restricted Interests (0 - 9). Higher score in all subscales reflect increasing severity. Questions rate behaviors on a 4-point scale: 0 = does not occur; 1 = occurs, mild problem; 2 = occurs, moderate problem; 3 = occurs, severe problem. Subscale scores consist of sum of ratings within each question subset. Lower order behaviors-- stereotypy and self-injury, higher order behaviors--compulsions, rituals /sameness, restricted interests. Lower Order total range: 0-51, Higher Order total range: 0-63 Stereotypic behavior and Self-injurious behavior subscales were summed to create a total range for Lower Order behavior; and Compulsive behavior, ritualistic /sameness behavior, and restricted interests subscales were summed to create a total range for Higher Order behavior.

Outcome measures

Outcome measures
Measure	Oxytocin n=10 Participants Intranasal Oxytocin: Oxytocin administered intranasally twice a day via 1 12 unit puff to each nostril, totaling 48 IU a day.	Placebo n=9 Participants Placebo: Saline will be administered intranasally twice a day via 1 puff per nostril, totaling 48 IU a day.
Change in Repetitive Behavior Based Repetitive Behavior Scale - Revised (RBS-R) Baseline: Higher Order	17.0 score on a scale Standard Deviation 10.6	20.0 score on a scale Standard Deviation 10.8
Change in Repetitive Behavior Based Repetitive Behavior Scale - Revised (RBS-R) Week 6:Higher Order	17.7 score on a scale Standard Deviation 16.2	17.8 score on a scale Standard Deviation 13.3
Change in Repetitive Behavior Based Repetitive Behavior Scale - Revised (RBS-R) Baseline: Lower Order	5.8 score on a scale Standard Deviation 4.6	4.9 score on a scale Standard Deviation 3.7
Change in Repetitive Behavior Based Repetitive Behavior Scale - Revised (RBS-R) Week 6: Lower Order	2.4 score on a scale Standard Deviation 2.3	3.7 score on a scale Standard Deviation 2.6

SECONDARY outcome

Timeframe: baseline, week 6

A clinical tool measuring emotion recognition through facial expression, voice and posture. 1. Child faces 2 (range 0 - 100, higher values reflecting higher % of errors) 2. Adult faces 2 (range 0 - 100, higher values reflecting higher % of errors) 3. Child paralanguage 2 (range 0 - 100, higher values reflecting higher % of errors) 4. Adult paralanguage 2 (range 0 - 100, higher values reflecting higher % of errors) Errors are counted and organized by pre-determined affect and intensity. Subtests considered separately.

Outcome measures

Outcome measures
Measure	Oxytocin n=10 Participants Intranasal Oxytocin: Oxytocin administered intranasally twice a day via 1 12 unit puff to each nostril, totaling 48 IU a day.	Placebo n=9 Participants Placebo: Saline will be administered intranasally twice a day via 1 puff per nostril, totaling 48 IU a day.
Change in Facial Emotion Recognition Based on Diagnostic Analysis of Nonverbal Accuracy - 2 (DANVA-2) DANVA -Face Baseline	32.6 units on a scale Standard Deviation 8.8	39.1 units on a scale Standard Deviation 8.4
Change in Facial Emotion Recognition Based on Diagnostic Analysis of Nonverbal Accuracy - 2 (DANVA-2) DANVA- FACE Week 6	33.5 units on a scale Standard Deviation 6.4	37.4 units on a scale Standard Deviation 7.4
Change in Facial Emotion Recognition Based on Diagnostic Analysis of Nonverbal Accuracy - 2 (DANVA-2) DANVA- Paralanguage Baseline	27.8 units on a scale Standard Deviation 5.7	34.1 units on a scale Standard Deviation 3.0
Change in Facial Emotion Recognition Based on Diagnostic Analysis of Nonverbal Accuracy - 2 (DANVA-2) DANVA- Paralanguage Week 6	30.5 units on a scale Standard Deviation 2.7	35.2 units on a scale Standard Deviation 4.7

Adverse Events

Oxytocin

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Oxytocin n=10 participants at risk Intranasal Oxytocin: Oxytocin administered intranasally twice a day via 1 12 unit puff to each nostril, totaling 48 IU a day.	Placebo n=9 participants at risk Placebo: Saline will be administered intranasally twice a day via 1 puff per nostril, totaling 48 IU a day.
General disorders Forgetfulness	10.0% 1/10 • Number of events 2	0.00% 0/9

Additional Information

Casara Jean Ferretti

Montefiore Medical Center, Albert Einstein College of Medicine

Phone: 3472499694

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place