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| Name | Class |
|---|---|
| Autism Science Foundation | OTHER |
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The purpose of this study is to explore the effects of propranolol in combination with early intensive behavioral intervention on social interaction, and secondarily on language, anxiety, adaptive behaviors, and global function in children with autism in a double-blinded, placebo-controlled pilot trial.
The investigators' specific aim is to examine the effects of serial doses of propranolol in combination with early intensive behavioral intervention on social interaction, and secondarily on language tasks, anxiety, adaptive behaviors, and global function in children with autism in a double-blinded, placebo-controlled trial. The investigators will also examine whether response to treatment can be predicted based upon markers of autonomic functioning, such as pupillary light reflex and heart rate variability, and whether anxiety predicts treatment response. The investigators' hypothesis is that propranolol will augment the effects of behavioral therapy on social functioning and language abilities. The investigators have demonstrated benefits of propranolol on social functioning and language in previous single-dose studies in adults and adolescents with autism. The investigators also predict that those with the greatest degree of autonomic dysregulation will demonstrate the greatest benefit from the drug.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Propranolol arm | Experimental | 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. |
|
| Placebo arm | Placebo Comparator | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Propranolol | Drug | Participant will receive Propranolol prior to each EIBI session |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in General Social Outcome Measure (GSOM) | 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. | Week 12 |
| Change in Social Responsiveness Scale (SRS) | 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. | Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Preschool Anxiety Scale (PAS) | 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| David Q Beversdorf, MD | University of Missouri-Columbia | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Thompson Center for Autism & Neurodevelopmental Disorders | Columbia | Missouri | 65211 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Allocated to Propranolol | Allocated to propranolol (n=5) Lost to follow-up (n=2)
Completed (n=4 at 6 weeks) (n=3 at 12 weeks) |
| FG001 | Allocated to Placebo | Allocated to placebo (n=4) Lost to follow-up (n=2)
Completed (n=2 at 6 weeks) (n=2 at 12 weeks) |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Propranolol Group | participants assigned to propranolol |
| BG001 | Placebo Group | participants assigned to placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in General Social Outcome Measure (GSOM) | 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. | This is a feasibility study so results are limited. | Posted | Mean | Standard Deviation | score on a scale | Week 12 |
|
Adverse event data collected for duration of medication administration- 12 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Propranolol Arm | Allocated to propranolol (n=5) Lost to follow-up (n=2)
Completed (n=4 at 6 weeks) (n=3 at 12 weeks) |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fatigue | General disorders | Non-systematic Assessment | Fatigue |
This was only a feasibility trial. We do not recommend emphasis be placed on the results due to the very small sample.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr David Beversdorf | University of Missouri | 5738826081 | beversdorfd@health.missouri.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Mar 19, 2015 | Feb 2, 2018 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D001321 | Autistic Disorder |
| ID | Term |
|---|---|
| D000067877 | Autism Spectrum Disorder |
| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
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| ID | Term |
|---|---|
| D011433 | Propranolol |
| ID | Term |
|---|---|
| D050198 | Phenoxypropanolamines |
| D011412 | Propanolamines |
| D000605 | Amino Alcohols |
| D000438 | Alcohols |
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| Placebo | Drug | Participant will receive placebo prior to each EIBI session |
|
| Week 12 |
| Change in Aberrant Behavior Checklist (ABC) | To 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. | Week 12 |
| Change in Vineland Adaptive Behavior Scales-II (VABS) | 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. | Week 12 |
| Change in Autism Impact Measure (AIM) | 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. | Week 12 |
| Change in Preschool Language Scale (PLS): | To 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. | Week 12 |
| Worse behavior |
|
| BG002 |
| Total |
Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| OG000 | Propranolol Arm | 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 |
| OG001 | Placebo Arm | 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 |
|
|
| Primary | Change in Social Responsiveness Scale (SRS) | 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. | Feasibility only | Posted | Mean | Standard Deviation | score on a scale | Week 12 |
|
|
|
| Secondary | Change in Preschool Anxiety Scale (PAS) | 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. | Feasibility only | Posted | Mean | Standard Deviation | score on a scale | Week 12 |
|
|
|
| Secondary | Change in Aberrant Behavior Checklist (ABC) | To 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. | Posted | Mean | Standard Deviation | score on a scale | Week 12 |
|
|
|
| Secondary | Change in Vineland Adaptive Behavior Scales-II (VABS) | 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. | Social domain | Posted | Mean | Standard Deviation | score on a scale | Week 12 |
|
|
|
| Secondary | Change in Autism Impact Measure (AIM) | 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. | Peer interaction subdomain | Posted | Mean | Standard Deviation | score on a scale | Week 12 |
|
|
|
| Secondary | Change in Preschool Language Scale (PLS): | To 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. | Posted | Mean | Standard Deviation | score on a scale | Week 12 |
|
|
|
| 0 |
| 5 |
| 0 |
| 5 |
| 1 |
| 5 |
| EG001 | Placebo Arm | Allocated to placebo (n=4) Lost to follow-up (n=2)
Completed (n=2 at 6 weeks) (n=2 at 12 weeks) | 0 | 4 | 0 | 4 | 1 | 4 |
| aggression | General disorders | Non-systematic Assessment | aggression |
|
| irritability | General disorders | Non-systematic Assessment | irritability |
|
| insomina | General disorders | Non-systematic Assessment | insomnia |
|
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| D009930 |
| Organic Chemicals |
| D020005 | Propanols |
| D000588 | Amines |
| D009281 | Naphthalenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D011083 | Polycyclic Compounds |