Trial Outcomes & Findings for Lurasidone Pediatric Autism Study (NCT NCT01911442)

NCT ID: NCT01911442

Last Updated: 2016-02-25

Results Overview

The ABC irritability subscale score is the sum of 15 items, each rated among 0 = Not at all; 1 = Slight in degree; 2 = Moderately serious; and 3 = Severe in degree. The ABC irritability subscale score ranges from 0 to 45. Higher values of ABC subscale scores represent greater severity of illness.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

150 participants

Primary outcome timeframe

Baseline to 6 Weeks

Results posted on

2016-02-25

Participant Flow

Participants were recruited from 46 centers in the United States between August 2013 and October 2014

Participants were screened and washed out up to 21 days.

Participant milestones

Participant milestones
Measure	Lurasidone 20 mg Once Daily Subjects received 20 mg/day from Day 1 to Week 6 Visit	Lurasidone 60 mg Once Daily Subjects received lurasidone 20 mg/day from Days 1-3, 40 mg/day from Days 4-6 and 60 mg/day from Day 7 to Week 6 Visit. One-time dose reduction to Lurasidone 40 mg/day may occur in Weeks 2, 3 or 4 (ie, between Day 8 to Day 29, inclusive).	Placebo Subject received placebo to match lurasidone from Day 1 to Week 6 Visit
Overall Study STARTED	49	51	50
Overall Study COMPLETED	43	47	38
Overall Study NOT COMPLETED	6	4	12

Reasons for withdrawal

Reasons for withdrawal
Measure	Lurasidone 20 mg Once Daily Subjects received 20 mg/day from Day 1 to Week 6 Visit	Lurasidone 60 mg Once Daily Subjects received lurasidone 20 mg/day from Days 1-3, 40 mg/day from Days 4-6 and 60 mg/day from Day 7 to Week 6 Visit. One-time dose reduction to Lurasidone 40 mg/day may occur in Weeks 2, 3 or 4 (ie, between Day 8 to Day 29, inclusive).	Placebo Subject received placebo to match lurasidone from Day 1 to Week 6 Visit
Overall Study Adverse Event	2	2	4
Overall Study Lack of Efficacy	1	1	1
Overall Study Lost to Follow-up	2	0	1
Overall Study Withdrawal by Subject	1	0	6
Overall Study Mother's Work Schedule	0	1	0

Baseline Characteristics

Lurasidone Pediatric Autism Study

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Lurasidone 20 mg Once Daily n=49 Participants Subject received placebo to match lurasidone from Day 1 to Week 6 Visit	Lurasidone 60 mg Once Daily n=51 Participants Subjects received lurasidone 20 mg/day from Days 1-3, 40 mg/day from Days 4-6 and 60 mg/day from Day 7 to Week 6 Visit. One-time dose reduction to Lurasidone 40 mg/day may occur in Weeks 2, 3 or 4 (ie, between Day 8 to Day 29, inclusive).	Placebo n=49 Participants Subject received placebo to match lurasidone from Day 1 to Week 6 Visit	Total n=149 Participants Total of all reporting groups
Age, Categorical <=18 years	49 Participants n=93 Participants	51 Participants n=4 Participants	49 Participants n=27 Participants	149 Participants n=483 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Sex: Female, Male Female	10 Participants n=93 Participants	8 Participants n=4 Participants	9 Participants n=27 Participants	27 Participants n=483 Participants
Sex: Female, Male Male	39 Participants n=93 Participants	43 Participants n=4 Participants	40 Participants n=27 Participants	122 Participants n=483 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	11 Participants n=93 Participants	7 Participants n=4 Participants	7 Participants n=27 Participants	25 Participants n=483 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	38 Participants n=93 Participants	44 Participants n=4 Participants	42 Participants n=27 Participants	124 Participants n=483 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=93 Participants	1 Participants n=4 Participants	0 Participants n=27 Participants	2 Participants n=483 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants	2 Participants n=483 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Race (NIH/OMB) Black or African American	10 Participants n=93 Participants	9 Participants n=4 Participants	5 Participants n=27 Participants	24 Participants n=483 Participants
Race (NIH/OMB) White	35 Participants n=93 Participants	38 Participants n=4 Participants	42 Participants n=27 Participants	115 Participants n=483 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants	2 Participants n=483 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=93 Participants	1 Participants n=4 Participants	0 Participants n=27 Participants	4 Participants n=483 Participants
Region of Enrollment United States	49 participants n=93 Participants	51 participants n=4 Participants	49 participants n=27 Participants	149 participants n=483 Participants

PRIMARY outcome

Timeframe: Baseline to 6 Weeks

Population: Intent to treat (ITT) population includes all randomized subjects who receive at least one dose of study medication and have at least one post-baseline assessment in any efficacy variable.

Outcome measures

Outcome measures
Measure	Lurasidone 20 mg Once Daily n=48 Participants Subjects received 20 mg/day from Day 1 to Week 6 Visit	Lurasidone 60 mg Once Daily n=51 Participants Subjects received lurasidone 20 mg/day from Days 1-3, 40 mg/day from Days 4-6 and 60 mg/day from Day 7 to Week 6 Visit. One-time dose reduction to Lurasidone 40 mg/day may occur in Weeks 2, 3 or 4 (ie, between Day 8 to Day 29, inclusive).	Placebo n=49 Participants Subject received placebo to match lurasidone from Day 1 to Week 6 Visit
Change in Aberrant Behavior Checklist (ABC) Irritability Subscale Score at Week 6	8.8 units on a scale Standard Error 1.5	9.4 units on a scale Standard Error 1.43	7.5 units on a scale Standard Error 1.52

SECONDARY outcome

Timeframe: Baseline to 6 Weeks

Population: Intent to treat population. 49 in the placebo arm is correct. One subject in the placebo group did not receive the study medication, and therefore that subject was removed from the ITT population. A total of 50 subjects were randomized and 49 subjects were included in the placebo group of the ITT population.

The Clinical Global Impression - Severity of Illness (CGI-S) Scale is rated on a 7-point scale of severity with 1 = Normal, not at all ill to 7 = Among the most extremely ill patients. Higher values of CGI-S scores represent greater severity of illness.

Outcome measures

Outcome measures
Measure	Lurasidone 20 mg Once Daily n=48 Participants Subjects received 20 mg/day from Day 1 to Week 6 Visit	Lurasidone 60 mg Once Daily n=51 Participants Subjects received lurasidone 20 mg/day from Days 1-3, 40 mg/day from Days 4-6 and 60 mg/day from Day 7 to Week 6 Visit. One-time dose reduction to Lurasidone 40 mg/day may occur in Weeks 2, 3 or 4 (ie, between Day 8 to Day 29, inclusive).	Placebo n=49 Participants Subject received placebo to match lurasidone from Day 1 to Week 6 Visit
Change From Baseline in Clinical Global Impression-Severity (CGI-S) at Week 6	-1.1 units on a scale Standard Error 0.17	-1.0 units on a scale Standard Error 0.16	-0.7 units on a scale Standard Error 0.17

SECONDARY outcome

Timeframe: Baseline to 6 Weeks

Population: ITT

The ABC hyperactivity and noncompliance subscale score is the sum of 16 items, each rated among 0 = Not at all; 1 = Slight in degree; 2 = Moderately serious; and 3 = Severe in degree. The ABC hyperactivity and noncompliance subscale score may range from 0 to 48. In general, higher values of ABC subscale scores represent greater severity of illness.

Outcome measures

Outcome measures
Measure	Lurasidone 20 mg Once Daily n=48 Participants Subjects received 20 mg/day from Day 1 to Week 6 Visit	Lurasidone 60 mg Once Daily n=51 Participants Subjects received lurasidone 20 mg/day from Days 1-3, 40 mg/day from Days 4-6 and 60 mg/day from Day 7 to Week 6 Visit. One-time dose reduction to Lurasidone 40 mg/day may occur in Weeks 2, 3 or 4 (ie, between Day 8 to Day 29, inclusive).	Placebo n=49 Participants Subject received placebo to match lurasidone from Day 1 to Week 6 Visit
Change From Baseline in Aberrant Behavior Checklist (ABC) Hyperactivity Subscale Score at Week 6	-9.7 units on a scale Standard Error 1.50	-6.6 units on a scale Standard Error 1.43	-7.1 units on a scale Standard Error 1.52

SECONDARY outcome

Timeframe: 6 Weeks

Population: ITT

CY-BOCS total score ranges from 0 to 20. The higher value of CY-BOCS scores the greater severity of illness. This table is a summary of Y-BOCS compulsion total score.

Outcome measures

Outcome measures
Measure	Lurasidone 20 mg Once Daily n=48 Participants Subjects received 20 mg/day from Day 1 to Week 6 Visit	Lurasidone 60 mg Once Daily n=51 Participants Subjects received lurasidone 20 mg/day from Days 1-3, 40 mg/day from Days 4-6 and 60 mg/day from Day 7 to Week 6 Visit. One-time dose reduction to Lurasidone 40 mg/day may occur in Weeks 2, 3 or 4 (ie, between Day 8 to Day 29, inclusive).	Placebo n=49 Participants Subject received placebo to match lurasidone from Day 1 to Week 6 Visit
Change From Baseline in Children's Yale-Brown Obsessive Compulsive Scales (CY-BOCS) Modified for Pervasive Developmental Disorders (PDDs)	-1.0 units on a scale Standard Error 0.46	-1.0 units on a scale Standard Error 0.44	-1.2 units on a scale Standard Error 0.49

SECONDARY outcome

Timeframe: 6 Weeks

Population: ITT

CGSQ is a caregiver reported assessment to assesses extent to which caregivers are affected by special demands associated with caring for a child with emotional/behavioral problems. CGSQ is comprised of three subscales which range in severity from 1 to 5 (Objective Strain, Subjective Externalized Strain, Subjective Internalized Strain), The 3 subscales are calculated as the averages of the corresponding individual items. Higher scores on each indicates greater strain. A Global Strain score is calculated by summing the three subscales (Objective Strain, Subjective Externalized Strain, Subjective Internalized Strain) to provide an indication of the total impact of the special demands on the family. Global Strain scores range from 3 to 15. As with the individual subscales, higher scores indicate greater strain.

Outcome measures

Outcome measures
Measure	Lurasidone 20 mg Once Daily n=48 Participants Subjects received 20 mg/day from Day 1 to Week 6 Visit	Lurasidone 60 mg Once Daily n=51 Participants Subjects received lurasidone 20 mg/day from Days 1-3, 40 mg/day from Days 4-6 and 60 mg/day from Day 7 to Week 6 Visit. One-time dose reduction to Lurasidone 40 mg/day may occur in Weeks 2, 3 or 4 (ie, between Day 8 to Day 29, inclusive).	Placebo n=49 Participants Subject received placebo to match lurasidone from Day 1 to Week 6 Visit
Change From Baseline in the Caregiver Strain Questionnaire (CGSQ)	-1.49 units on a scale Standard Error 0.292	-1.66 units on a scale Standard Error 0.274	-1.35 units on a scale Standard Error 0.300

SECONDARY outcome

Timeframe: 6 Weeks

Population: ITT - the current data presented is at week 6.

Outcome measures

Outcome measures
Measure	Lurasidone 20 mg Once Daily n=43 Participants Subjects received 20 mg/day from Day 1 to Week 6 Visit	Lurasidone 60 mg Once Daily n=47 Participants Subjects received lurasidone 20 mg/day from Days 1-3, 40 mg/day from Days 4-6 and 60 mg/day from Day 7 to Week 6 Visit. One-time dose reduction to Lurasidone 40 mg/day may occur in Weeks 2, 3 or 4 (ie, between Day 8 to Day 29, inclusive).	Placebo n=38 Participants Subject received placebo to match lurasidone from Day 1 to Week 6 Visit
Proportion of Subjects Who Have CGI-I Score of 1 (Very Much Improved) or 2 (Much Improved) at Week 6	17 percentage of subjects	17 percentage of subjects	14 percentage of subjects

SECONDARY outcome

Timeframe: 6 Weeks

Population: ITT

Outcome measures

Outcome measures
Measure	Lurasidone 20 mg Once Daily n=43 Participants Subjects received 20 mg/day from Day 1 to Week 6 Visit	Lurasidone 60 mg Once Daily n=47 Participants Subjects received lurasidone 20 mg/day from Days 1-3, 40 mg/day from Days 4-6 and 60 mg/day from Day 7 to Week 6 Visit. One-time dose reduction to Lurasidone 40 mg/day may occur in Weeks 2, 3 or 4 (ie, between Day 8 to Day 29, inclusive).	Placebo n=38 Participants Subject received placebo to match lurasidone from Day 1 to Week 6 Visit
Proportion of Subjects Who Have at Least 25% Reduction From Baseline to Week 6 in the ABC Irritability Subscale Score.	25 percentage of subjects	26 percentage of subjects	23 percentage of subjects

Adverse Events

Lurasidone 20 mg Once Daily

Serious events: 3 serious events

Other events: 33 other events

Deaths: 0 deaths

Lurasidone 60 mg Once Daily

Serious events: 2 serious events

Other events: 38 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 28 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Lurasidone 20 mg Once Daily n=49 participants at risk Subjects received 20 mg/day from Day 1 to Week 6 Visit	Lurasidone 60 mg Once Daily n=51 participants at risk Subjects received lurasidone 20 mg/day from Days 1-3, 40 mg/day from Days 4-6 and 60 mg/day from Day 7 to Week 6 Visit. One-time dose reduction to Lurasidone 40 mg/day may occur in Weeks 2, 3 or 4 (ie, between Day 8 to Day 29, inclusive).	Placebo n=49 participants at risk Subject received placebo to match lurasidone from Day 1 to Week 6 Visit
Injury, poisoning and procedural complications Torus Fracture	2.0% 1/49 • Number of events 1 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.	2.0% 1/51 • Number of events 1 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.	0.00% 0/49 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.
Injury, poisoning and procedural complications Upper Limb Fracture	2.0% 1/49 • Number of events 1 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.	2.0% 1/51 • Number of events 1 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.	0.00% 0/49 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.
General disorders Appendicitis	0.00% 0/49 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.	2.0% 1/51 • Number of events 1 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.	0.00% 0/49 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.
General disorders Irritability	2.0% 1/49 • Number of events 1 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.	0.00% 0/51 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.	0.00% 0/49 • Adverse event data were collected as spontaneous reports at all visits Participant flow module presents all subjects randomized. The summary of AEs is based on safety population. As explained earlier, there were 49 placebo subjects included in the safety population.

Other adverse events

Additional Information

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Sunovion

Phone: 1-866-503-6351

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee There IS agreement between Principal Investigator and Sponsor that restricts PI's rights to discuss or publish trial results after trial is completed. In addition to the \<60-180 day restriction above, since this is a multicenter study, 1st publication of study results shall be made with other participating study sites as a multicenter publication provided, if a multicenter publication is not forthcoming within 24 months post completion of study at all sites, PI shall be free to publish.
Publication restrictions are in place

Restriction type: OTHER