A Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multicenter Study Investigating the Efficacy, Safety, and Tolerability of JNJ-42165279 in Adolescent and Adult Subjects With Autism Spectrum Disorder
Overview
- Phase
- Phase 2
- Intervention
- JNJ-42165279
- Conditions
- Autism Spectrum Disorder
- Sponsor
- Janssen Research & Development, LLC
- Enrollment
- 78
- Locations
- 9
- Primary Endpoint
- Change From Baseline to Day 85 in the Autism Behavior Inventory (ABI) Core Domain Score (Social Communication and Restrictive Behaviour)
- Status
- Completed
- Last Updated
- last year
Overview
Brief Summary
The purpose of this study is to evaluate the efficacy of JNJ-42165279 compared with placebo in the improvement of symptoms of Autism Spectrum Disorder (ASD) during 12 weeks of treatment using the Autism Behavior Inventory (ABI).
Investigators
Eligibility Criteria
Inclusion Criteria
- •Diagnosis of Autism Spectrum Disorder (ASD) according to Diagnostic and Statistical Manual of Mental Disorders - 5th Edition (DSM-5) criteria and made or confirmed using the Autism Diagnostic Observation Schedule, 2nd edition (ADOS-2) (minimum score of 8 \[autism spectrum\])
- •Otherwise healthy for their age group or medically stable with or without medication on the basis of physical examination, medical history, vital signs, 12-lead electrocardiogram (ECG), and clinical laboratory tests
- •Have a composite score on Kaufman Brief Intelligence Test, Second Edition (KBIT-2) of at least 60
- •Must live with a parent or primary caregiver or, if not, during each week he/she must either (A) spend at least 3 hours a day for at least 4 days or, (B) spend the weekend with a parent or primary caregiver
- •Any pharmacologic, diet, or behavioral intervention for ASD must have begun at least 1 month prior to the baseline visit and continue unchanged through the treatment period, or have ended at least 1 month prior to the baseline visit
- •Must be able to swallow the study medication whole and self-administer medication if living independently or have a parent or caregiver be able to administer medication
- •Must agree to abide by the birth control requirements during the study and for 3 months after the last dose
Exclusion Criteria
- •Current or recent history of clinically significant suicidal ideation within the past 6 months, or a history of suicidal behavior within the past year
- •Use of a drug with moderate/strong cytochrome P450 (CYP)3A4 inhibiting or inducing properties at, or prior to, screening that is not discontinued at least within 1 month prior to Day 1
- •History of drug or alcohol use disorder according to DSM-5 criteria within 6 months before screening or positive test result(s) for alcohol or drugs of abuse (except if related to current treatment)
- •Currently taking or has taken within the past month recreational or medically prescribed cannabis
Arms & Interventions
JNJ-42165279
Participants will self-administer 25 milligram (mg) JNJ-42165279 tablets orally twice daily for 12 weeks.
Intervention: JNJ-42165279
Placebo
Participants will self-administer matching placebo tablets orally twice daily for 12 weeks.
Intervention: Placebo
Outcomes
Primary Outcomes
Change From Baseline to Day 85 in the Autism Behavior Inventory (ABI) Core Domain Score (Social Communication and Restrictive Behaviour)
Time Frame: Baseline (Day 1) to Day 85
ABI:62-item questionnaire to track outcomes in autism spectrum disorder (ASD). Each item was answered on 1 of 2 possible dimensions: quality (how well person carries out particular behavior; 1 to 13 items) or frequency (how often particular behavior occurs; 14 to 62 items). Each item was rated on scale of 0 (never) to 3 (very often) for frequency and 0 (without help) to 3 (not at all) for quality. Higher score=severe symptoms/more frequency. ABI core domain score was from 2 domains: social communication (23 items; 3 sub-domains) and repetitive/restrictive behavior (RRB;15 items; 4 sub-domains). Domain and sub-domain scores: calculated as average of non-missing items (ie, sum of all non-missing items divided by number of non-missing items); scores ranged from 0 to 3, higher scores=more severe symptoms of ASD. ABI core domain score=sum of social communication and RRB domain scores divided by total number of items in these 2 domains. Negative changes in ABI core domain score=improvement.
Change From Baseline to Day 85 in the ABI Social Communication Domain Score
Time Frame: Baseline (Day 1) to Day 85
ABI was 62-item questionnaire completed on a web/mobile application or on paper. It tracks outcomes in ASD. Each ABI item was answered on 1 of 2 possible dimensions, quality (how well a person carries out a particular behavior; 1 to 13 items) or frequency (how often a particular behavior occurs; 14 to 62 items). Each item was rated on scale of 0 (never) to 3 (very often) for frequency and 0 (without help) to 3 (not at all) for quality. Higher score indicated severe symptoms/more frequency. The ABI social communication domain score consisted of 23 items; with 3 sub-domains. The domain and sub-domain scores were calculated as the average of the non-missing items (ie, sum of all non-missing items divided by the number of non-missing items). For both domains and its sub-domains, scores ranged from 0 to 3 with higher scores indicating more severe symptoms of ASD. Negative change in score indicates improvement.
Change From Baseline to Day 85 in the ABI Repetitive/Restrictive Behavior (RRB) Domain Score
Time Frame: Baseline (Day 1) to Day 85
ABI is 62-item questionnaire completed on a web/mobile application or on paper. It tracks outcomes in ASD. Each ABI item was answered on 1 of 2 possible dimensions, quality (how well a person carries out a particular behavior; 1 to 13 items) or frequency (how often a particular behavior occurs; 14 to 62 items). Each item was rated on scale of 0 (never) to 3 (very often) for frequency and 0 (without help) to 3 (not at all) for quality. Higher score indicated severe symptoms/more frequency. The ABI RRB domain score consisted of 15 items; with 3 sub-domains. The domain and sub-domain scores were calculated as the average of the non-missing items (ie, sum of all non-missing items divided by the number of non-missing items). For both domains and its sub-domains, scores ranged from 0 to 3 with higher scores indicating more severe symptoms of ASD. Negative change in score indicates improvement.
Change From Baseline to Day 85 in the Social Responsiveness Scale 2 (SRS-2) Total T-Score
Time Frame: Baseline (Day 1) to Day 85
SRS-2: 65-item scale measured extent of autistic social impairment and included 5 subscales: social awareness, social cognition, social communication, social motivation, and restricted interests and repetitive behavior. Each of 65 items had 4 responses: not true, sometimes true, often true, and almost always true. Scoring value for each item was 0 to 3. If a response to an item was missing, then pre-defined median value for item (0 or 1) was imputed. SRS-2 was not scored if 7 or more item responses were missed. Total raw score was sum of item response values. Each subscale was obtained by adding response values and converted total raw score to standardized T-score based on gender and rater (parent or caregiver). Total T-score was categorized: within normal limits (\<=59), mild (60 to 65), moderate (66 to 75) and severe (\>=76). For total score T-score, higher scores=more severe symptoms. SRS T-score had mean of 50 and standard deviation of 10. Negative changes in T-scores=improvement.
Secondary Outcomes
- Change From Baseline to Day 85 in the ABI Mood and Anxiety Domain Score(Baseline (Day 1) to Day 85)
- Change From Baseline to Day 85 in the ABI Challenging Behavior Domain Score(Baseline (Day 1) to Day 85)
- Change From Baseline to Day 85 in the ABI Self-Regulation Domain Score(Baseline (Day 1) to Day 85)
- Change From Baseline to Day 85 in the Aberrant Behavior Checklist (ABC) Subscales Scores(Baseline (Day 1) to Day 85)
- Change From Baseline to Day 71 in the Autism Behavior Inventory-Short Form (ABI-S) Domains Scores(Baseline (Day 1) to Day 71)
- Change From Baseline to Day 85 in the Autism Behavior Inventory-Clinician Interview (ABI-C) Domains Scores(Baseline (Day 1) to Day 85)
- Change From Baseline to Day 85 in the Clinical Global Impression-Severity (CGI-S) Scale Score(Baseline (Day 1) to Day 85)
- Change From Baseline to Day 85 in the Zarit Burden Interview (ZBI) Global Score(Baseline (Day 1) to Day 85)
- Change From Baseline to Day 85 in the Repetitive Behavior Scale - Revised (RBS-R) Subscale Score(Baseline (Day 1) to Day 85)
- Change From Baseline to Day 85 in the Child Adolescent Symptom Inventory - Anxiety (CASI-Anx) Scale Score(Baseline (Day 1) to Day 85)
- Change From Baseline to Day 85 in the Caregiver Global Impression of Severity (Caregiver GI-S) Scale Score(Baseline (Day 1) to Day 85)
- Caregiver Assessment of Treatment: Question 1: Number of Participants With Overall Status (Improvement) of Autism at Day 85(At Day 85)
- Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85(At Day 85)
- Caregiver Assessment of Treatment: Question 3: Number of Participants With Interest in Continuing Treatment at Day 85(At Day 85)
- Self Global Impression of Improvement (Self GI-I) Scale Score at Day 85(At Day 85)
- Clinical Global Impression-Improvement (CGI-I) Score (Frequency Distribution) at Day 85(At Day 85)