Study to Assess the Janssen Autism Knowledge Engine in Participants With Autism Spectrum Disorder and in a Normally Developing Cohort

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the utility of and to clinically validate the Autism Behavior Inventory (ABI) in measuring clinical symptoms of Autism Spectrum Disorder (ASD) compared with other gold standard measures.

Detailed Description
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This is an observational study (in which participants identified as belonging to study groups are assessed for biomedical or health outcomes) to evaluate the utility of Janssen Autism Knowledge Engine (JAKE) in measuring clinical symptoms of ASD in children and adults with ASD. The study will consist of 3 cohorts of participants. For Cohort 1 and 2, there will be 14-day Screening phase and 8-week (Cohort 1) or 10 week (Cohort 2) data collection phase extending from Day 0 (Baseline) to Day 56 or Day 70, respectively. For Cohort 3, the study will consist of a screening visit and a single testing visit, which may be combined. Primarily, the usability of JAKE as a system to monitor clinical outcomes in ASD will be assessed. Participants' safety will be monitored throughout the study.

Official Title
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A Study to Assess the Janssen Autism Knowledge Engine in Children and Adults With Autism Spectrum Disorder and in a Normally Developing Cohort

Conditions
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Autism Spectrum Disorder

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Cohort 1 and 2: Participants must be males or females aged 6 years and older with at least one female for every five male participants Diagnosis of ASD made or confirmed using the Autism Diagnostic Observation Schedule, 2nd edition (ADOS-2) Participants can receive behavioral and/or pharmacologic treatments for ASD and comorbid disorders during the course of the study, or not (Cohort 1) In cohort 2, as part of their standard care participants must be about to begin a behavioral intervention within 2 weeks after the Baseline visit Parents or legally authorized representatives must speak and understand English Cohort 3: Participants must be males or females aged 6 years and older with at least one female for every five male participants. Parents or legally authorized representatives must speak and understand English In the opinion of the investigator, subject and parents must be capable of completing all procedures and tasks of the study Each participant or their legally authorized representative must sign an informed consent form (ICF) A score in the normal range on the Social Communication Questionnaire (SCQ) and must not have any Diagnostic and Statistical Manual of Mental Disorders Exclusion Criteria: Cohort 1 and 2: Measured composite score on the Kaufmann Brief Intelligence Test-2 (KBIT-2) of less than 60 History of, or current significant medical illness that the Investigator considers should exclude the participant Psychological and/or emotional problems which would render the informed consent invalid or limit the ability of the subject to comply with the study requirements Any condition that in the opinion of the investigator would compromise the study, or the wellbeing of the subject; for example, visual problems that would impede eye-tracking or viewing the stimuli or hearing problems that would impede hearing auditory stimuli Participant is an employee of the investigator or study site, with direct involvement in the proposed study or other studies under the direction of that investigator or study site, as well as family members of the employees or the investigator Cohort 3: History of or current significant medical illness that the Investigator considers should exclude the participant Psychological and/or emotional problems which would render the informed consent invalid or limit the ability of the participant to comply with the study requirements Any condition that in the opinion of the investigator would compromise the study, or the wellbeing of the subject; for example, visual problems that would impede eye-tracking or viewing the stimuli or hearing problems that would impede hearing auditory stimuli Participant is an employee of the investigator or study site, with direct involvement in the proposed study or other studies under the direction of that investigator or study site, as well as family members of the employees or the investigator Participant should not take any sedating medications on the day of the test battery and should not consume caffeine within 2 hours prior to the battery

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Cohort 1<br>It will consist of planned 100 children and adults with Autism Spectrum Disorder (ASD) aged 6 years and older with no requirements regarding concurrent therapies or treatments. | |
| Cohort 2<br>It will consist of planned 50 children and adults aged 6 and older who, as part of their standard care, happen to be beginning a behavioral intervention within 2 weeks after the baseline visit of the study. This intervention can be an applied behavior analysis (ABA) program or similar or a social skills program or a school-based autism program and must be intended to last at least throughout the duration of the study. | |
| Cohort 3<br>It will consist of planned 30 normally developing children and adults. These participants will only have a single visit wherein they will undergo a single session with the task battery and biosensors. There should be 5 participants aged 6-9 years, 5 aged 10-12 years, 5 aged 13-17 years, and 5 aged 18 years and older. This cohort should approximate the male:female ratio in Cohorts 1 & 2, with approximately 1 female for every 5 males. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Utility of Autism Behavior Inventory(ABI) in Measuring Clinical Symptoms of Autism Spectrum Disorder (ASD) | The ABI is a series of 73 questions related to the core and associated symptoms of ASD. Questions are answered on two 4-point scales, consisting of ratings of frequency and intensity, frequency and context, or quality and context. | up to Week 10 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Usability of the Janssen Autism Knowledge Engine (JAKE) in Measuring Clinical Symptoms of ASD | The JAKE personal healthcare record (pHR) encompasses various modules for use by clinicians, caregivers, participants and the sponsor. The JAKE is accessible both through a web interface for computers and applications for mobile devices. The modules are Medical/Developmental History, Autism Behavior Inventory (ABI), Journal/ASD events, Treatment Tracker, Dashboard, HealthVault, Research Data Warehouse and Workbench. Results of several parent and clinician questionnaires will be compared with the findings of the mentis ABI and biosensors. An exit survey will assess overall usability of the system. | up to Week 10 | 
| Correlation Between Key Biosensors and Autism Spectrum Disorder (ASD) Symptoms | The utility of the biosensors will be determined by measuring the associations between the biosensors and the ASD symptoms (response to tasks and stimuli in lab, the ABI, standard scales, and the event tracker). | up to Week 10 | 
| Correlation Between Genomic Characteristics and Autism Phenotypes | Genomic analysis will be done by analyzing DNA samples related to the JAKE System or autism spectrum disorder. | up to Week 10 | 
| Usability of the JAKE System to detect changes in response to standard | Change is symptoms in response to coincident courses of treatment with standard behavioral therapy as measured with the JAKE system. | up to Week 10 | 
| Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) |  | up to Week 10 | 
| Normative data on the JAKE Biosensor Array (Continuous and Periodic) | Normative data on the JAKE biosensor array (Continuous and Periodic) will be collected. Comparisons between Cohort 3 and Cohorts 1 and 2 will be made to examine differences between normally developing and ASD participants. | up to Week 10 | 
| Normative data on the JAKE Task Battery | Normative data on the JAKE task battery will be collected. Comparisons between Cohort 3 and Cohorts 1 and 2 will be made to examine differences between normally developing and ASD participants. | up to Week 10 | 
| Ease of use of JAKE for use in Prospective Clinical Trials | The JAKE personal healthcare record (pHR) encompasses various modules for use by clinicians, caregivers, participants and the sponsor. The JAKE is accessible both through a web interface for computers and applications for mobile devices. The modules are Medical/Developmental History, ABI, Journal/ASD events, Treatment Tracker, Dashboard, Health Vault, Research Data Warehouse and Workbench. | up to Week 10 | 
| Utility of JAKE for use in Prospective Clinical Trials | The JAKE personal healthcare record (pHR) encompasses various modules for use by clinicians, caregivers, participants and the sponsor. The JAKE is accessible both through a web interface for computers and applications for mobile devices. The modules are Medical/Developmental History, ABI, Journal/ASD events, Treatment Tracker, Dashboard, Health Vault, Research Data Warehouse and Workbench. | up to Week 10 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Autism Spectrum Disorder, Janssen Autism Knowledge Engine, Autism Behavior Inventory