Trial Outcomes & Findings for Combined Effects of Early Behavioral Intervention and Propranolol on ASD (NCT NCT02428205)
NCT ID: NCT02428205
Last Updated: 2024-03-04
Results Overview
The GSOM is a brief evaluation tool that measures a participant's level of social functioning and how it changes with intervention. There GSOM includes 4 different tasks: conversational reciprocity, ability to recognize facial expressions, social problem solving, affect demonstration, and emotional perspective taking. The experimenter administers each component of the GSOM to the participant and then scores their responses according to a pre-determined scoring rubric. Each component is scored on a 1-5 or 0 - 2 scale, with higher total scores indicating better social functioning. A video camera will be used to record the participant's behavior during GSOM administration for the purposes of having two independent raters score the tasks after the study session is complete. A camera will also be used during the affect demonstration task of the GSOM. The overall score is used. The range is 6-132, with higher scores being better.
COMPLETED
NA
10 participants
Week 12
2024-03-04
Participant Flow
Participant milestones
| Measure |
Allocated to Propranolol
Allocated to propranolol (n=5)
Lost to follow-up (n=2)
* Withdrew after baseline visit due to not feeling seeing a benefit (n=1)
* Withdrew after 6-week visit -no reason given (n=1)
Completed (n=4 at 6 weeks) (n=3 at 12 weeks)
|
Allocated to Placebo
Allocated to placebo (n=4)
Lost to follow-up (n=2)
* Withdrew after baseline visit - no reason given (n=1)
* Withdrew after baseline visit due to worsening behaviors (n=1)
Completed (n=2 at 6 weeks) (n=2 at 12 weeks)
|
|---|---|---|
|
Overall Study
STARTED
|
5
|
4
|
|
Overall Study
COMPLETED
|
3
|
2
|
|
Overall Study
NOT COMPLETED
|
2
|
2
|
Reasons for withdrawal
| Measure |
Allocated to Propranolol
Allocated to propranolol (n=5)
Lost to follow-up (n=2)
* Withdrew after baseline visit due to not feeling seeing a benefit (n=1)
* Withdrew after 6-week visit -no reason given (n=1)
Completed (n=4 at 6 weeks) (n=3 at 12 weeks)
|
Allocated to Placebo
Allocated to placebo (n=4)
Lost to follow-up (n=2)
* Withdrew after baseline visit - no reason given (n=1)
* Withdrew after baseline visit due to worsening behaviors (n=1)
Completed (n=2 at 6 weeks) (n=2 at 12 weeks)
|
|---|---|---|
|
Overall Study
Lack of Efficacy
|
1
|
0
|
|
Overall Study
Lost to Follow-up
|
1
|
1
|
|
Overall Study
Worse behavior
|
0
|
1
|
Baseline Characteristics
Combined Effects of Early Behavioral Intervention and Propranolol on ASD
Baseline characteristics by cohort
| Measure |
Propranolol Group
n=5 Participants
participants assigned to propranolol
|
Placebo Group
n=4 Participants
participants assigned to placebo
|
Total
n=9 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
5 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
5 participants
n=99 Participants
|
4 participants
n=107 Participants
|
9 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Week 12Population: This is a feasibility study so results are limited.
The GSOM is a brief evaluation tool that measures a participant's level of social functioning and how it changes with intervention. There GSOM includes 4 different tasks: conversational reciprocity, ability to recognize facial expressions, social problem solving, affect demonstration, and emotional perspective taking. The experimenter administers each component of the GSOM to the participant and then scores their responses according to a pre-determined scoring rubric. Each component is scored on a 1-5 or 0 - 2 scale, with higher total scores indicating better social functioning. A video camera will be used to record the participant's behavior during GSOM administration for the purposes of having two independent raters score the tasks after the study session is complete. A camera will also be used during the affect demonstration task of the GSOM. The overall score is used. The range is 6-132, with higher scores being better.
Outcome measures
| Measure |
Propranolol Arm
n=3 Participants
Propranolol will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session. To minimize risk, the bodyweight adjusted minimum dose of propranolol used safely for test anxiety in healthy adults (10mg) will be used: participants weighing \> 30 kg will be given 4 mg, participants weighing 22.5 - 30 kg will be given 3 mg, participants weighing 15 - 22.5 kg will be given 2 mg. Participants weighing \< 15 kg will be excluded for safety reasons.
Propranolol: Participant will receive Propranolol prior to each EIBI session
|
Placebo Arm
n=2 Participants
Placebo will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session.The same bodyweight adjusted doses specified in the propranolol arm will be used for this arm.
Placebo: Participant will receive placebo prior to each EIBI session
|
|---|---|---|
|
Change in General Social Outcome Measure (GSOM)
|
48.3 score on a scale
Standard Deviation 14.0
|
20.0 score on a scale
Standard Deviation 15.6
|
PRIMARY outcome
Timeframe: Week 12Population: Feasibility only
This 65-item, parent-report measure asks questions about a participants' social awareness, social information processing, capacity for reciprocal social responses, social anxiety or avoidance, and characteristic autistic preoccupations or traits. The total score is used with a range of 0-195, with lower scores being better.
Outcome measures
| Measure |
Propranolol Arm
n=3 Participants
Propranolol will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session. To minimize risk, the bodyweight adjusted minimum dose of propranolol used safely for test anxiety in healthy adults (10mg) will be used: participants weighing \> 30 kg will be given 4 mg, participants weighing 22.5 - 30 kg will be given 3 mg, participants weighing 15 - 22.5 kg will be given 2 mg. Participants weighing \< 15 kg will be excluded for safety reasons.
Propranolol: Participant will receive Propranolol prior to each EIBI session
|
Placebo Arm
n=2 Participants
Placebo will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session.The same bodyweight adjusted doses specified in the propranolol arm will be used for this arm.
Placebo: Participant will receive placebo prior to each EIBI session
|
|---|---|---|
|
Change in Social Responsiveness Scale (SRS)
|
79.3 score on a scale
Standard Deviation 17.0
|
72.0 score on a scale
Standard Deviation 2.8
|
SECONDARY outcome
Timeframe: Week 12Population: Feasibility only
To assess anxiety, the PAS will be completed by the parents/caregivers of participants. This self-report measure yields a total score in addition to six subscales (separation anxiety, social phobia, obsessive compulsive, panic/agoraphobia, physical injury fears, and generalized anxiety). The overall score is used. Range is 0-112 with lower score meaning less anxiety.
Outcome measures
| Measure |
Propranolol Arm
n=3 Participants
Propranolol will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session. To minimize risk, the bodyweight adjusted minimum dose of propranolol used safely for test anxiety in healthy adults (10mg) will be used: participants weighing \> 30 kg will be given 4 mg, participants weighing 22.5 - 30 kg will be given 3 mg, participants weighing 15 - 22.5 kg will be given 2 mg. Participants weighing \< 15 kg will be excluded for safety reasons.
Propranolol: Participant will receive Propranolol prior to each EIBI session
|
Placebo Arm
n=2 Participants
Placebo will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session.The same bodyweight adjusted doses specified in the propranolol arm will be used for this arm.
Placebo: Participant will receive placebo prior to each EIBI session
|
|---|---|---|
|
Change in Preschool Anxiety Scale (PAS)
|
87.7 score on a scale
Standard Deviation 32.9
|
71.5 score on a scale
Standard Deviation 30.4
|
SECONDARY outcome
Timeframe: Week 12To assess overall behavioral disturbances, a parent/caregiver of the participants will complete ABC. This 58-item questionnaire is a well validated, reliable, and widely used assessment tool for interventions for a range of cognitive disorders. The full score is used, with a range of 0-174, with higher scores meaning more behaviors.
Outcome measures
| Measure |
Propranolol Arm
n=3 Participants
Propranolol will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session. To minimize risk, the bodyweight adjusted minimum dose of propranolol used safely for test anxiety in healthy adults (10mg) will be used: participants weighing \> 30 kg will be given 4 mg, participants weighing 22.5 - 30 kg will be given 3 mg, participants weighing 15 - 22.5 kg will be given 2 mg. Participants weighing \< 15 kg will be excluded for safety reasons.
Propranolol: Participant will receive Propranolol prior to each EIBI session
|
Placebo Arm
n=2 Participants
Placebo will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session.The same bodyweight adjusted doses specified in the propranolol arm will be used for this arm.
Placebo: Participant will receive placebo prior to each EIBI session
|
|---|---|---|
|
Change in Aberrant Behavior Checklist (ABC)
|
72.7 score on a scale
Standard Deviation 65.5
|
53.0 score on a scale
Standard Deviation 39.6
|
SECONDARY outcome
Timeframe: Week 12Population: Social domain
To assess overall adaptive functioning, the VABS will be administered in the form of a structured interview with the parent/caregiver. The VABS is a well validated assessment used for the full range of our subjects' ages and yields standard scores in Communication, Daily Living Skills, Socialization, and Motor Skills. VABS scores for Socialization will be monitored in this study. The range is 20-160 with higher scores better.
Outcome measures
| Measure |
Propranolol Arm
n=3 Participants
Propranolol will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session. To minimize risk, the bodyweight adjusted minimum dose of propranolol used safely for test anxiety in healthy adults (10mg) will be used: participants weighing \> 30 kg will be given 4 mg, participants weighing 22.5 - 30 kg will be given 3 mg, participants weighing 15 - 22.5 kg will be given 2 mg. Participants weighing \< 15 kg will be excluded for safety reasons.
Propranolol: Participant will receive Propranolol prior to each EIBI session
|
Placebo Arm
n=2 Participants
Placebo will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session.The same bodyweight adjusted doses specified in the propranolol arm will be used for this arm.
Placebo: Participant will receive placebo prior to each EIBI session
|
|---|---|---|
|
Change in Vineland Adaptive Behavior Scales-II (VABS)
|
70.7 score on a scale
Standard Deviation 10.2
|
66.5 score on a scale
Standard Deviation 23.3
|
SECONDARY outcome
Timeframe: Week 12Population: Peer interaction subdomain
The AIM will be administered to assess the frequency and impact of a participant's ASD-related symptoms. A parent/caregiver is asked a series of 41 questions regarding the frequency and the impact, or interference resulting from, a series of autism-associated behaviors. Peer interaction from the AIM is the primary domain obtained from this measure. For this domain the range is 0 to 40, with higher numbers more severe.
Outcome measures
| Measure |
Propranolol Arm
n=3 Participants
Propranolol will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session. To minimize risk, the bodyweight adjusted minimum dose of propranolol used safely for test anxiety in healthy adults (10mg) will be used: participants weighing \> 30 kg will be given 4 mg, participants weighing 22.5 - 30 kg will be given 3 mg, participants weighing 15 - 22.5 kg will be given 2 mg. Participants weighing \< 15 kg will be excluded for safety reasons.
Propranolol: Participant will receive Propranolol prior to each EIBI session
|
Placebo Arm
n=2 Participants
Placebo will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session.The same bodyweight adjusted doses specified in the propranolol arm will be used for this arm.
Placebo: Participant will receive placebo prior to each EIBI session
|
|---|---|---|
|
Change in Autism Impact Measure (AIM)
|
23.7 score on a scale
Standard Deviation 8.7
|
24.5 score on a scale
Standard Deviation 6.4
|
SECONDARY outcome
Timeframe: Week 12To evaluate language, we will administer the PLS, which has been developed for use in younger children (birth through age 7), and is appropriate for children of all ability levels, including nonverbal children. This play-based, interactive assessment is designed to assess receptive and expressive language skills and their change over time. The range is 50-150 with higher scores being better.
Outcome measures
| Measure |
Propranolol Arm
n=3 Participants
Propranolol will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session. To minimize risk, the bodyweight adjusted minimum dose of propranolol used safely for test anxiety in healthy adults (10mg) will be used: participants weighing \> 30 kg will be given 4 mg, participants weighing 22.5 - 30 kg will be given 3 mg, participants weighing 15 - 22.5 kg will be given 2 mg. Participants weighing \< 15 kg will be excluded for safety reasons.
Propranolol: Participant will receive Propranolol prior to each EIBI session
|
Placebo Arm
n=2 Participants
Placebo will be administered in the form of a liquid dose via oral syringe by the participants' parent/caregiver an hour before each EIBI session.The same bodyweight adjusted doses specified in the propranolol arm will be used for this arm.
Placebo: Participant will receive placebo prior to each EIBI session
|
|---|---|---|
|
Change in Preschool Language Scale (PLS):
|
87.7 score on a scale
Standard Deviation 32.9
|
71.5 score on a scale
Standard Deviation 30.4
|
Adverse Events
Propranolol Arm
Placebo Arm
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Propranolol Arm
n=5 participants at risk
Allocated to propranolol (n=5)
Lost to follow-up (n=2)
* Withdrew after baseline visit due to not feeling seeing a benefit (n=1)
* Withdrew after 6-week visit -no reason given (n=1)
Completed (n=4 at 6 weeks) (n=3 at 12 weeks)
|
Placebo Arm
n=4 participants at risk
Allocated to placebo (n=4)
Lost to follow-up (n=2)
* Withdrew after baseline visit - no reason given (n=1)
* Withdrew after baseline visit due to worsening behaviors (n=1)
Completed (n=2 at 6 weeks) (n=2 at 12 weeks)
|
|---|---|---|
|
General disorders
Fatigue
|
20.0%
1/5 • Number of events 1 • Adverse event data collected for duration of medication administration- 12 weeks
|
0.00%
0/4 • Adverse event data collected for duration of medication administration- 12 weeks
|
|
General disorders
aggression
|
0.00%
0/5 • Adverse event data collected for duration of medication administration- 12 weeks
|
25.0%
1/4 • Number of events 1 • Adverse event data collected for duration of medication administration- 12 weeks
|
|
General disorders
irritability
|
0.00%
0/5 • Adverse event data collected for duration of medication administration- 12 weeks
|
25.0%
1/4 • Number of events 1 • Adverse event data collected for duration of medication administration- 12 weeks
|
|
General disorders
insomina
|
0.00%
0/5 • Adverse event data collected for duration of medication administration- 12 weeks
|
25.0%
1/4 • Number of events 1 • Adverse event data collected for duration of medication administration- 12 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place