Trial Outcomes & Findings for A Study to Investigate the Efficacy, Safety, and Tolerability of JNJ-42165279 in Adolescent and Adult Participants With Autism Spectrum Disorder (NCT NCT03664232)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

78 participants

Primary outcome timeframe

Baseline (Day 1) to Day 85

Results posted on

2025-04-23

Participant Flow

Participant milestones

Participant milestones
Measure	Placebo QD Participants enrolled prior to protocol amendment-5, received placebo matched to JNJ-42165279 tablets orally once daily (QD) from Day 1 to Day 85.	Placebo BID Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg QD Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Overall Study STARTED	7	31	8	32
Overall Study Treated	7	30	8	32
Overall Study COMPLETED	7	26	7	27
Overall Study NOT COMPLETED	0	5	1	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo BID Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg QD Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Overall Study Withdrawal by Subject	1	0	1
Overall Study Adverse Event	0	1	0
Overall Study Other-Non-compliance with study drug	1	0	2
Overall Study Other-Unspecified	3	0	1
Overall Study - Withdrawal by Parent/Guardian	0	0	1

Baseline Characteristics

A Study to Investigate the Efficacy, Safety, and Tolerability of JNJ-42165279 in Adolescent and Adult Participants With Autism Spectrum Disorder

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo QD n=7 Participants Participants enrolled prior to protocol amendment-5, received placebo matched to JNJ-42165279 tablets orally once daily (QD) from Day 1 to Day 85.	Placebo BID n=30 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=8 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=31 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	Total n=76 Participants Total of all reporting groups
Age, Continuous	22.4 years STANDARD_DEVIATION 5.13 • n=5 Participants	19.5 years STANDARD_DEVIATION 4.62 • n=7 Participants	23.5 years STANDARD_DEVIATION 4.96 • n=5 Participants	20.9 years STANDARD_DEVIATION 5.98 • n=4 Participants	20.8 years STANDARD_DEVIATION 5.35 • n=21 Participants
Age, Customized Adolescent: 13 to 17 Years	0 Participants n=5 Participants	12 Participants n=7 Participants	2 Participants n=5 Participants	12 Participants n=4 Participants	26 Participants n=21 Participants
Age, Customized Adult: 18 to 35 Years	7 Participants n=5 Participants	18 Participants n=7 Participants	6 Participants n=5 Participants	19 Participants n=4 Participants	50 Participants n=21 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	8 Participants n=7 Participants	1 Participants n=5 Participants	8 Participants n=4 Participants	18 Participants n=21 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	22 Participants n=7 Participants	7 Participants n=5 Participants	23 Participants n=4 Participants	58 Participants n=21 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	2 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	3 Participants n=21 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	3 Participants n=7 Participants	2 Participants n=5 Participants	4 Participants n=4 Participants	9 Participants n=21 Participants
Race (NIH/OMB) White	7 Participants n=5 Participants	25 Participants n=7 Participants	6 Participants n=5 Participants	23 Participants n=4 Participants	61 Participants n=21 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants	2 Participants n=21 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	1 Participants n=21 Participants
Region of Enrollment United States	7 Participants n=5 Participants	30 Participants n=7 Participants	8 Participants n=5 Participants	31 Participants n=4 Participants	76 Participants n=21 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The full analysis set twice daily (FAS-BID) included all randomized participants who had received at least 1 dose of study agent at the BID dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure. As per study statistical analysis plan (SAP), data for this outcome measure was planned to be collected and analyzed only on the participants who received BID dosing.

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.

Outcome measures

Outcome measures
Measure	Placebo BID n=25 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=26 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the Autism Behavior Inventory (ABI) Core Domain Score (Social Communication and Restrictive Behaviour)	-0.20 Score on a scale Standard Deviation 0.400	-0.26 Score on a scale Standard Deviation 0.274	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS-BID included all randomized participants who had received at least 1 dose of study agent at the BID dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure. As per study SAP, data for this outcome measure was planned to be collected and analyzed only on the participants who received BID dosing.

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.

Outcome measures

Outcome measures
Measure	Placebo BID n=25 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=26 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the ABI Social Communication Domain Score	-0.22 Score on a scale Standard Deviation 0.379	-0.27 Score on a scale Standard Deviation 0.332	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS-BID included all randomized participants who had received at least 1 dose of study agent at the BID dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure. As per study SAP, data for this outcome measure was planned to be collected and analyzed only on the participants who received BID dosing.

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.

Outcome measures

Outcome measures
Measure	Placebo BID n=25 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=26 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the ABI Repetitive/Restrictive Behavior (RRB) Domain Score	-0.17 Score on a scale Standard Deviation 0.525	-0.23 Score on a scale Standard Deviation 0.299	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

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.

Outcome measures

Outcome measures
Measure	Placebo BID n=7 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=27 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=7 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=29 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the Social Responsiveness Scale 2 (SRS-2) Total T-Score	-6.4 T-score Standard Deviation 4.79	-4.6 T-score Standard Deviation 7.91	-4.1 T-score Standard Deviation 6.99	-7.1 T-score Standard Deviation 7.73

SECONDARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

ABI was 62-item questionnaire to track 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. ABI mood and anxiety domain had 9 items with 5 sub-domains. ABI mood and anxiety domain and its sub-domains, scores ranged from 0 to 3 with higher scores indicating more severe symptoms of ASD. 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). Negative changes in ABI mood and anxiety domain score indicated improvement.

Outcome measures

Outcome measures
Measure	Placebo BID n=6 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=25 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=7 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=26 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the ABI Mood and Anxiety Domain Score	-0.30 Score on a scale Standard Deviation 0.408	-0.23 Score on a scale Standard Deviation 0.622	-0.19 Score on a scale Standard Deviation 0.551	-0.26 Score on a scale Standard Deviation 0.552

SECONDARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

ABI was 62-item questionnaire to track 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. ABI challenging behavior domain had 7 items with 4 sub-domains. ABI challenging behavior domain and its sub-domains, scores ranged from 0 to 3 with higher scores indicating more severe symptoms of ASD. 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). Negative changes in ABI challenging behavior domain score indicated improvement.

Outcome measures

Outcome measures
Measure	Placebo BID n=6 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=25 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=7 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=26 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the ABI Challenging Behavior Domain Score	-0.27 Score on a scale Standard Deviation 0.329	-0.22 Score on a scale Standard Deviation 0.533	-0.20 Score on a scale Standard Deviation 0.411	-0.20 Score on a scale Standard Deviation 0.532

SECONDARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

ABI was 62-item questionnaire to track 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. ABI self-regulation domain had 8 items with 2 sub-domains. ABI self-regulation domain and its sub-domains, scores ranged from 0 to 3 with higher scores indicating more severe symptoms of ASD. 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). Negative changes in ABI self-regulation domain score indicated improvement.

Outcome measures

Outcome measures
Measure	Placebo BID n=6 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=25 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=7 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=26 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the ABI Self-Regulation Domain Score	-0.31 Score on a scale Standard Deviation 0.272	-0.20 Score on a scale Standard Deviation 0.510	-0.11 Score on a scale Standard Deviation 0.364	-0.18 Score on a scale Standard Deviation 0.380

SECONDARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

The ABC was 58-item behavior rating scale used to measure behavior problems across 5 subscales: irritability (15 items), lethargy (social withdrawal; 16 items), stereotypic behavior (7 items), hyperactivity/ noncompliance (16 items), and inappropriate speech (4 items). Items were rated on a 4-point scale score ranging from 0 (not at all a problem) to 3 (the problem is severe in degree). The total subscale scores ranged from 0 to 45, 0 to 48, 0 to 21, 0 to 48 and 0 to 12 for irritability, lethargy (social withdrawal), stereotypic behavior, hyperactivity, and inappropriate speech respectively. The higher scores indicating more severe behavior problems. Negative change in score indicated improvement.

Outcome measures

Outcome measures
Measure	Placebo BID n=7 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=26 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=7 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=28 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the Aberrant Behavior Checklist (ABC) Subscales Scores Irritability	-5.86 Score on a scale Standard Deviation 5.398	-3.80 Score on a scale Standard Deviation 6.408	-2.71 Score on a scale Standard Deviation 6.291	-4.18 Score on a scale Standard Deviation 8.940
Change From Baseline to Day 85 in the Aberrant Behavior Checklist (ABC) Subscales Scores Lethargy/Social Withdrawal	-6.86 Score on a scale Standard Deviation 5.014	-3.31 Score on a scale Standard Deviation 7.092	-2.71 Score on a scale Standard Deviation 4.680	-5.14 Score on a scale Standard Deviation 7.271
Change From Baseline to Day 85 in the Aberrant Behavior Checklist (ABC) Subscales Scores Stereotypic Behavior	-1.86 Score on a scale Standard Deviation 2.340	-1.19 Score on a scale Standard Deviation 3.359	-0.14 Score on a scale Standard Deviation 1.676	-1.68 Score on a scale Standard Deviation 3.128
Change From Baseline to Day 85 in the Aberrant Behavior Checklist (ABC) Subscales Scores Hyperactivity/Noncompliance	-6.00 Score on a scale Standard Deviation 3.916	-4.77 Score on a scale Standard Deviation 7.010	-2.00 Score on a scale Standard Deviation 6.351	-4.07 Score on a scale Standard Deviation 6.532
Change From Baseline to Day 85 in the Aberrant Behavior Checklist (ABC) Subscales Scores Inappropriate Speech	-1.43 Score on a scale Standard Deviation 2.573	-0.85 Score on a scale Standard Deviation 1.826	-0.43 Score on a scale Standard Deviation 0.787	-0.86 Score on a scale Standard Deviation 2.138

SECONDARY outcome

Timeframe: Baseline (Day 1) to Day 71

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

The ABI-S was the shorter version of ABI. It consists of 24 items across 5 areas: challenging behavior (3 items), mood and anxiety (5 items), restrictive behaviors (7 items), social communication (6 items), and self-regulation (3 items). Each item was answered on 1 of 2 possible dimensions, quality (how well a person carries out a particular behavior or frequency (how often a particular behavior occurs). Each ABI-S domain score was calculated as the sum of the scores in the ABI-S domain divided by the total number of items in this domain. Each item was rated on scales of 0 (never) to 3 (very often) for frequency and 0 (without help) to 3 (not at all) for quality. The total subscale scores ranged from 0 to 9, 0 to 15, 0 to 21, 0 to 18, and 0 to 9, for challenging behavior, mood and anxiety, restrictive behaviors, social communication, and self-regulation respectively. Lower score indicated better performance. Negative change in score indicates improvement.

Outcome measures

Outcome measures
Measure	Placebo BID n=5 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=18 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=5 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=22 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 71 in the Autism Behavior Inventory-Short Form (ABI-S) Domains Scores Challenging behavior	0.07 Score on a scale Standard Deviation 0.548	-0.17 Score on a scale Standard Deviation 0.618	-0.33 Score on a scale Standard Deviation 0.707	-0.32 Score on a scale Standard Deviation 0.477
Change From Baseline to Day 71 in the Autism Behavior Inventory-Short Form (ABI-S) Domains Scores Mood and anxiety	-0.48 Score on a scale Standard Deviation 0.782	-0.24 Score on a scale Standard Deviation 0.473	0.32 Score on a scale Standard Deviation 0.179	-0.21 Score on a scale Standard Deviation 0.624
Change From Baseline to Day 71 in the Autism Behavior Inventory-Short Form (ABI-S) Domains Scores Restrictive behaviors	-0.14 Score on a scale Standard Deviation 0.440	-0.12 Score on a scale Standard Deviation 0.580	0.11 Score on a scale Standard Deviation 0.275	-0.23 Score on a scale Standard Deviation 0.383
Change From Baseline to Day 71 in the Autism Behavior Inventory-Short Form (ABI-S) Domains Scores Social communication	-0.37 Score on a scale Standard Deviation 0.431	-0.38 Score on a scale Standard Deviation 0.520	-0.23 Score on a scale Standard Deviation 0.480	-0.31 Score on a scale Standard Deviation 0.425
Change From Baseline to Day 71 in the Autism Behavior Inventory-Short Form (ABI-S) Domains Scores Self regulation	-0.47 Score on a scale Standard Deviation 0.506	0.04 Score on a scale Standard Deviation 0.740	0.13 Score on a scale Standard Deviation 0.447	-0.29 Score on a scale Standard Deviation 0.637

SECONDARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

The ABI-C covers the domains of ABI and was intended for completion by the clinician following an interview with caregiver and observation or interview with the individual with ASD, as appropriate. The ABI-C was designed to capture the behaviors of a person with ASD that have occurred over the past week. There were 14 items across each of the 5 domains: challenging behavior (2 items), mood and anxiety (2 items), restrictive behaviors (5 items), social communication (3 items), and self-regulation (2 items). The clinician rates the severity of behaviors or level of impairment on a scale of 1 (no impairment) to 7 (very severe difficulties) with a higher score indicating more severe symptoms of ASD. Total subscale scores ranged from 0 to 14, 0 to 14, 0 to 35, 0 to 21 and 0 to 14, for challenging behavior, mood and anxiety, restrictive behaviors, social communication and self-regulation respectively. Lower score indicated better performance. Negative change in score indicates improvement.

Outcome measures

Outcome measures
Measure	Placebo BID n=7 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=27 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=7 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=29 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the Autism Behavior Inventory-Clinician Interview (ABI-C) Domains Scores Challenging behavior	-0.64 Score on a scale Standard Deviation 1.107	-0.26 Score on a scale Standard Deviation 0.739	-0.07 Score on a scale Standard Deviation 1.018	-0.40 Score on a scale Standard Deviation 1.030
Change From Baseline to Day 85 in the Autism Behavior Inventory-Clinician Interview (ABI-C) Domains Scores Mood and anxiety	-0.36 Score on a scale Standard Deviation 0.690	-0.44 Score on a scale Standard Deviation 0.859	-0.07 Score on a scale Standard Deviation 0.450	-0.60 Score on a scale Standard Deviation 1.270
Change From Baseline to Day 85 in the Autism Behavior Inventory-Clinician Interview (ABI-C) Domains Scores Restrictive behaviors	-0.29 Score on a scale Standard Deviation 0.430	-0.47 Score on a scale Standard Deviation 0.686	-0.43 Score on a scale Standard Deviation 0.582	-0.52 Score on a scale Standard Deviation 0.863
Change From Baseline to Day 85 in the Autism Behavior Inventory-Clinician Interview (ABI-C) Domains Scores Social communication	0.05 Score on a scale Standard Deviation 0.300	-0.59 Score on a scale Standard Deviation 0.669	-0.05 Score on a scale Standard Deviation 0.525	-0.70 Score on a scale Standard Deviation 0.993
Change From Baseline to Day 85 in the Autism Behavior Inventory-Clinician Interview (ABI-C) Domains Scores Self regulation	-0.14 Score on a scale Standard Deviation 0.627	-0.41 Score on a scale Standard Deviation 1.029	-0.07 Score on a scale Standard Deviation 0.189	-0.22 Score on a scale Standard Deviation 0.862

SECONDARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS-BID included all randomized participants who had received at least 1 dose of study agent at the BID dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure. As per study SAP, data for this outcome measure was planned to be collected and analyzed only on the participants who received BID dosing.

The CGI-S 7-point scale was a global assessment that measures the clinician's impression of the severity of illness of the participant. A rating scale of 1 to 7 was used, with 1="normal, not at all ill" and 7="among the most extremely ill". Higher score indicated more severe illness.

Outcome measures

Outcome measures
Measure	Placebo BID n=26 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=28 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the Clinical Global Impression-Severity (CGI-S) Scale Score	0.0 Score on a scale Interval -2.0 to 1.0	0.0 Score on a scale Interval -3.0 to 1.0	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

The RBS-R was a 43-item report scale to indicate the occurrence of repetitive behaviors and degree to which a behavior was a problem scored on a range between 0 (behavior did not occur) to 3 (behavior occurred and was a severe problem). There are 6 subscale scores: Stereotyped Behavior (items 1 to 6), self-injurious behavior (items 7 to 14), compulsive behavior (items 15 to 22), ritualistic behavior (items 23 to 28), sameness behavior (items 29 to 39), and restricted behavior (items 40 to 43). The ranges for RBS-R subscale scores were stereotyped behavior: 0 to 18; self-injurious behavior: 0 to 24; compulsive behavior: 0 to 24; ritualistic behavior: 0 to 18; sameness behavior: 0 to 33; and restricted behavior: 0 to 12. Higher scores indicate more severe problems with repetitive behaviors and a decrease (a negative change) from baseline represents improvement.

Outcome measures

Outcome measures
Measure	Placebo BID n=7 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=27 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=7 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=29 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the Repetitive Behavior Scale - Revised (RBS-R) Subscale Score stereotyped behavior	-1.6 Score on a scale Standard Deviation 2.07	-0.6 Score on a scale Standard Deviation 2.34	-1.0 Score on a scale Standard Deviation 1.83	-0.8 Score on a scale Standard Deviation 1.89
Change From Baseline to Day 85 in the Repetitive Behavior Scale - Revised (RBS-R) Subscale Score self-injurious behavior	-1.4 Score on a scale Standard Deviation 1.99	0.2 Score on a scale Standard Deviation 2.15	-0.7 Score on a scale Standard Deviation 1.60	-1.2 Score on a scale Standard Deviation 2.05
Change From Baseline to Day 85 in the Repetitive Behavior Scale - Revised (RBS-R) Subscale Score compulsive behavior	-1.7 Score on a scale Standard Deviation 4.61	-0.4 Score on a scale Standard Deviation 2.95	-1.0 Score on a scale Standard Deviation 1.53	-0.8 Score on a scale Standard Deviation 2.33
Change From Baseline to Day 85 in the Repetitive Behavior Scale - Revised (RBS-R) Subscale Score ritualistic behavior	-2.6 Score on a scale Standard Deviation 3.21	-0.5 Score on a scale Standard Deviation 3.71	-1.6 Score on a scale Standard Deviation 2.15	-1.1 Score on a scale Standard Deviation 3.63
Change From Baseline to Day 85 in the Repetitive Behavior Scale - Revised (RBS-R) Subscale Score sameness behavior	-6.3 Score on a scale Standard Deviation 4.89	-2.0 Score on a scale Standard Deviation 4.98	-2.3 Score on a scale Standard Deviation 3.15	-2.9 Score on a scale Standard Deviation 6.39
Change From Baseline to Day 85 in the Repetitive Behavior Scale - Revised (RBS-R) Subscale Score restricted behavior	-0.4 Score on a scale Standard Deviation 3.05	-0.5 Score on a scale Standard Deviation 1.83	-0.3 Score on a scale Standard Deviation 2.81	-0.7 Score on a scale Standard Deviation 2.55

SECONDARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

The ZBI short version scale consists of 22 items designed to assess the psychological burden experienced by a caregiver. Items ask how the caregiver felt and their responses range from 0 to 4, where 0 = never and 4 = nearly always. The ZBI global score was the sum of all item score and ranges from 0 to 88 with higher scores indicating a higher burden. A negative change in the ZBI global score from baseline representing improvement.

Outcome measures

Outcome measures
Measure	Placebo BID n=7 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=30 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=8 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=29 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the Zarit Burden Interview (ZBI) Global Score	-2.0 Score on a scale Standard Deviation 12.12	-2.2 Score on a scale Standard Deviation 10.89	-4.3 Score on a scale Standard Deviation 7.83	-3.9 Score on a scale Standard Deviation 10.20

SECONDARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

The CASI assesses symptoms of the following disorders: attention deficit hyperactivity disorder, oppositional defiant disorder, conduct disorder, generalized anxiety disorder, social phobia, separation anxiety disorder, disruptive mood dysregulation disorder, major depressive episode, manic episode, dysthymic disorder, schizophrenia, autistic/Asperger's disorder, anorexia, and bulimia. CASI-Anx scale total is a 21-point anxiety subscale of the full CASI. Responses were ranged from 0 to 3, where 0 = never and 3 = very often. The CASI-Anx scale total score ranges from 0 to 63 with higher scores indicating more severe anxiety. Negative changes in Symptom Severity scores indicate improvement.

Outcome measures

Outcome measures
Measure	Placebo BID n=7 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=27 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=7 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=29 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the Child Adolescent Symptom Inventory - Anxiety (CASI-Anx) Scale Score	-5.14 Score on a scale Standard Deviation 7.647	-4.89 Score on a scale Standard Deviation 8.135	-1.86 Score on a scale Standard Deviation 2.610	-5.19 Score on a scale Standard Deviation 8.020

SECONDARY outcome

Timeframe: Baseline (Day 1) to Day 85

Population: The FAS-BID included all randomized participants who had received at least 1 dose of study agent at the BID dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure. As per study SAP, data for this outcome measure was planned to be collected and analyzed only on the participants who received BID dosing.

The caregiver GI-S is a single-item instrument that asks caregivers to rate their overall impression of the severity of their child's ASD symptoms. A rating scale of 1 to 7 was used, with 1= none and 7=severe illness. Higher score indicated more severe illness.

Outcome measures

Outcome measures
Measure	Placebo BID n=26 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=28 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Change From Baseline to Day 85 in the Caregiver Global Impression of Severity (Caregiver GI-S) Scale Score	0.0 Score on a scale Interval -3.0 to 2.0	0.0 Score on a scale Interval -4.0 to 2.0	—	—

SECONDARY outcome

Timeframe: At Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

The caregiver assessment of treatment is a 3-item questionnaire completed at the end of the treatment period. The first question asks caregivers to rate their overall impression of improvement in their child's autism since starting the study medication, with 7 response options ranging from 1 "very much improved" to 7 "very much worse".

Outcome measures

Outcome measures
Measure	Placebo BID n=7 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=29 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=8 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=29 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Caregiver Assessment of Treatment: Question 1: Number of Participants With Overall Status (Improvement) of Autism at Day 85 Very much improved	0 Participants	2 Participants	0 Participants	3 Participants
Caregiver Assessment of Treatment: Question 1: Number of Participants With Overall Status (Improvement) of Autism at Day 85 Much improved	2 Participants	3 Participants	0 Participants	2 Participants
Caregiver Assessment of Treatment: Question 1: Number of Participants With Overall Status (Improvement) of Autism at Day 85 Improved	3 Participants	7 Participants	5 Participants	10 Participants
Caregiver Assessment of Treatment: Question 1: Number of Participants With Overall Status (Improvement) of Autism at Day 85 No change	2 Participants	15 Participants	3 Participants	13 Participants
Caregiver Assessment of Treatment: Question 1: Number of Participants With Overall Status (Improvement) of Autism at Day 85 Worse	0 Participants	1 Participants	0 Participants	1 Participants
Caregiver Assessment of Treatment: Question 1: Number of Participants With Overall Status (Improvement) of Autism at Day 85 Much worse	0 Participants	1 Participants	0 Participants	0 Participants
Caregiver Assessment of Treatment: Question 1: Number of Participants With Overall Status (Improvement) of Autism at Day 85 Very Much Worse	0 Participants	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: At Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure and n (number analyzed): number of participants evaluable at each specified category.

The caregiver assessment of treatment was a 3-item questionnaire completed at the end of the treatment period. The second question asks caregivers whether there was improvement in 9 specific symptoms (child communicated more, child's repetitive behaviors improvement, child's restricted interests, child's mood improved, child was less anxious, child better perform daily activities, child more interest social activities, child's sleep improved, child less distracted), with responses options of Yes or No.

Outcome measures

Outcome measures
Measure	Placebo BID n=7 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=29 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=8 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=29 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child communicated more- No	4 Participants	16 Participants	4 Participants	12 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child communicated more- Yes	3 Participants	13 Participants	4 Participants	17 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child repetitive behaviors improvement -No	3 Participants	24 Participants	6 Participants	19 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child repetitive behaviors improvement -Yes	3 Participants	5 Participants	2 Participants	10 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Restricted Interests Improved- No	3 Participants	24 Participants	6 Participants	16 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Restricted Interests Improved- Yes	3 Participants	5 Participants	2 Participants	13 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Mood Improved- No	2 Participants	17 Participants	2 Participants	15 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Mood Improved- Yes	5 Participants	11 Participants	6 Participants	14 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Was Less Anxious- No	5 Participants	18 Participants	2 Participants	18 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Was Less Anxious-Yes	2 Participants	10 Participants	6 Participants	11 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Better Perform Daily Activities- No	3 Participants	17 Participants	3 Participants	16 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Better Perform Daily Activities- yes	4 Participants	12 Participants	5 Participants	13 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child More Interest Social Activities- No	3 Participants	17 Participants	4 Participants	17 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child More Interest Social Activities- Yes	4 Participants	12 Participants	4 Participants	12 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Sleep Improved -No	5 Participants	21 Participants	6 Participants	18 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Sleep Improved -Yes	2 Participants	8 Participants	2 Participants	11 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Less Distracted- No	5 Participants	24 Participants	5 Participants	19 Participants
Caregiver Assessment of Treatment: Question 2: Number of Participants With Improvement in Specific Symptoms During Treatment at Day 85 Child Less Distracted-Yes	1 Participants	5 Participants	3 Participants	10 Participants

SECONDARY outcome

Timeframe: At Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure.

The caregiver assessment of treatment is a 3-item questionnaire completed at the end of the treatment period. The third question asked caregivers their interest in having their child continue the study medication. There were 5 response options ranging from "Not at all interested" to "Extremely interested".

Outcome measures

Outcome measures
Measure	Placebo BID n=7 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=29 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=8 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=29 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Caregiver Assessment of Treatment: Question 3: Number of Participants With Interest in Continuing Treatment at Day 85 Extremely Interested	2 Participants	10 Participants	0 Participants	6 Participants
Caregiver Assessment of Treatment: Question 3: Number of Participants With Interest in Continuing Treatment at Day 85 Very Interested	1 Participants	3 Participants	2 Participants	5 Participants
Caregiver Assessment of Treatment: Question 3: Number of Participants With Interest in Continuing Treatment at Day 85 Moderately Interested	1 Participants	7 Participants	1 Participants	6 Participants
Caregiver Assessment of Treatment: Question 3: Number of Participants With Interest in Continuing Treatment at Day 85 A Little Interested	2 Participants	4 Participants	3 Participants	4 Participants
Caregiver Assessment of Treatment: Question 3: Number of Participants With Interest in Continuing Treatment at Day 85 Not At All Interested	1 Participants	5 Participants	2 Participants	8 Participants

SECONDARY outcome

Timeframe: At Day 85

Population: The FAS-BID included all randomized participants who had received at least 1 dose of study agent at the BID dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure. As per study SAP, data for this outcome measure was planned to be collected and analyzed only on the participants who received BID dosing.

Self-Global impression of improvement (Self GI-I) scale score at Day 85 was reported. At the end of the treatment period, the participant was asked to give his/her impression of overall improvement in ASD symptoms using a single-item instrument, the Self GI-I. The scale ranges from 1 (very much better) to 7 (very much worse), with higher scores indicating worsening of symptoms.

Outcome measures

Outcome measures
Measure	Placebo BID n=30 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=27 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Self Global Impression of Improvement (Self GI-I) Scale Score at Day 85	3.03 Score on a scale Standard Deviation 1.098	3.41 Score on a scale Standard Deviation 0.931	—	—

SECONDARY outcome

Timeframe: At Day 85

Population: The FAS analysis set included all randomized participants who had received at least 1 dose of study agent at either dose and had both a baseline and at least 1 postbaseline efficacy assessment. Here, N (number of participants analyzed) signifies participants who were analyzed for this outcome measure and n (number analyzed): number of participants evaluable at each specified category.

The CGI-I was single-item instrument that measures the clinician's global impression of improvement in the participant from the initiation of treatment. The CGI-I was 7-point scale to measure improvement in illness, where 1=very much improved, 2=much improved, 3=minimally improved, 4=no change from baseline, 5=minimally worse, 6=much worse, 7=very much worse.

Outcome measures

Outcome measures
Measure	Placebo BID n=1 Participants Participants enrolled after protocol amendment-5 received placebo matched to JNJ-42165279 tablets orally twice daily (BID) from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=26 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.	JNJ-42165279 25 mg QD n=2 Participants Participants enrolled prior to protocol amendment-5 received JNJ-42165279 25 milligrams (mg) tablets orally QD from Day 1 to Day 85.	JNJ-42165279 25 mg BID n=27 Participants Participants enrolled after protocol amendment-5 received JNJ-42165279 25 mg tablets orally BID from Day 1 to Day 85.
Clinical Global Impression-Improvement (CGI-I) Score (Frequency Distribution) at Day 85 Very much improved	0 Participants	0 Participants	0 Participants	0 Participants
Clinical Global Impression-Improvement (CGI-I) Score (Frequency Distribution) at Day 85 Much improved	0 Participants	3 Participants	0 Participants	7 Participants
Clinical Global Impression-Improvement (CGI-I) Score (Frequency Distribution) at Day 85 Minimally improved	1 Participants	9 Participants	0 Participants	5 Participants
Clinical Global Impression-Improvement (CGI-I) Score (Frequency Distribution) at Day 85 No change	0 Participants	14 Participants	2 Participants	15 Participants
Clinical Global Impression-Improvement (CGI-I) Score (Frequency Distribution) at Day 85 Minimally worse	0 Participants	0 Participants	0 Participants	0 Participants
Clinical Global Impression-Improvement (CGI-I) Score (Frequency Distribution) at Day 85 Much worse	0 Participants	0 Participants	0 Participants	0 Participants
Clinical Global Impression-Improvement (CGI-I) Score (Frequency Distribution) at Day 85 Very much worse	0 Participants	0 Participants	0 Participants	0 Participants

Adverse Events

Placebo QD

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Placebo BID

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

JNJ-42165279 25 mg QD

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

JNJ-42165279 25 mg BID

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Study Director

Janssen Research & Development, LLC

Phone: 844-434-4210

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee If an investigator wishes to publish information from the study, a copy of the manuscript must be provided to the sponsor for review at least 60 days before submission for publication or presentation. If requested by the sponsor in writing, the investigator will withhold such publication for up to an additional 60 days to allow for filing of a patent application.
Publication restrictions are in place

Restriction type: OTHER