Trial Outcomes & Findings for A Clinical Trial That Will Study the Efficacy and Safety of an Investigational Drug in Acutely Psychotic People With Schizophrenia (NCT NCT04092686)
NCT ID: NCT04092686
Last Updated: 2026-06-18
Results Overview
PANSS was an interview-based assessment comprised of 30 items and 3 subscales (Positive, Negative, General Psychopathology). The Positive subscale assessed hallucinations, delusions, and related symptoms; the Negative subscale assessed emotional withdrawal, lack of motivation, and similar symptoms; and the General Psychopathology subscale addressed other symptoms such as anxiety, somatic concern, and disorientation. An anchored Likert scale from 1 - 7, where values of 2 and above indicated the presence of progressively more severe symptoms, was used to score each item. Individual items were then summed to determine scores for the 3 subscales, as well as a total score. PANSS total score ranges from: 30-210, where a higher score indicates greater severity. A negative change from baseline indicates improvement.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
464 participants
Primary outcome timeframe
Baseline, Week 6
Results posted on
2026-06-18
Participant Flow
Participants took part in the study at investigational sites in Bulgaria, Croatia, Latvia, Serbia, Ukraine, Russia, Colombia, and the United States (US) from 14 Oct 2019 to 14 June 2023.
A total of 660 participants were screened, of which 464 participants were enrolled and randomized to SEP-363856 75 milligrams (mg), SEP-363856 100 mg or placebo in 1:1:1 ratio.
Participant milestones
| Measure |
Placebo
Participants received matched SEP-363856 placebo tablets, orally, once daily, from Day 1 up to Week 6
|
SEP-363856 75mg
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 up to Week 6.
|
SEP-363856 100mg
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 to Day 7 and then the dose was increased to 100 mg from Day 8 up to Week 6.
|
|---|---|---|---|
|
Overall Study
STARTED
|
155
|
155
|
154
|
|
Overall Study
Modified-intent-to-treat Population (mITT)
|
155
|
153
|
152
|
|
Overall Study
COMPLETED
|
128
|
121
|
116
|
|
Overall Study
NOT COMPLETED
|
27
|
34
|
38
|
Reasons for withdrawal
| Measure |
Placebo
Participants received matched SEP-363856 placebo tablets, orally, once daily, from Day 1 up to Week 6
|
SEP-363856 75mg
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 up to Week 6.
|
SEP-363856 100mg
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 to Day 7 and then the dose was increased to 100 mg from Day 8 up to Week 6.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
10
|
14
|
19
|
|
Overall Study
Lack of Efficacy
|
1
|
5
|
3
|
|
Overall Study
Withdrawal by participant
|
14
|
14
|
15
|
|
Overall Study
Reason not specified
|
2
|
1
|
1
|
Baseline Characteristics
A Clinical Trial That Will Study the Efficacy and Safety of an Investigational Drug in Acutely Psychotic People With Schizophrenia
Baseline characteristics by cohort
| Measure |
Placebo
n=155 Participants
Participants received matched SEP-363856 placebo tablets, orally, once daily, from Day 1 up to Week 6.
|
SEP-363856 75mg
n=155 Participants
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 up to Week 6.
|
SEP-363856 100mg
n=154 Participants
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 to Day 7 and then the dose was increased to 100 mg from Day 8 up to Week 6.
|
Total
n=464 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Region of Enrollment
Russia
|
29 participants
n=20 Participants
|
28 participants
n=20 Participants
|
28 participants
n=40 Participants
|
85 participants
n=5 Participants
|
|
Age, Continuous
|
38.6 years
STANDARD_DEVIATION 10.82 • n=20 Participants
|
38.5 years
STANDARD_DEVIATION 11.24 • n=20 Participants
|
37.5 years
STANDARD_DEVIATION 10.33 • n=40 Participants
|
38.2 years
STANDARD_DEVIATION 10.79 • n=5 Participants
|
|
Sex: Female, Male
Female
|
65 Participants
n=20 Participants
|
71 Participants
n=20 Participants
|
56 Participants
n=40 Participants
|
192 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
90 Participants
n=20 Participants
|
84 Participants
n=20 Participants
|
98 Participants
n=40 Participants
|
272 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
6 Participants
n=20 Participants
|
14 Participants
n=20 Participants
|
7 Participants
n=40 Participants
|
27 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
149 Participants
n=20 Participants
|
141 Participants
n=20 Participants
|
147 Participants
n=40 Participants
|
437 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=20 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=20 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=20 Participants
|
0 Participants
n=20 Participants
|
1 Participants
n=40 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
3 Participants
n=20 Participants
|
1 Participants
n=20 Participants
|
3 Participants
n=40 Participants
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
39 Participants
n=20 Participants
|
33 Participants
n=20 Participants
|
40 Participants
n=40 Participants
|
112 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
108 Participants
n=20 Participants
|
118 Participants
n=20 Participants
|
108 Participants
n=40 Participants
|
334 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=20 Participants
|
1 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=20 Participants
|
2 Participants
n=20 Participants
|
2 Participants
n=40 Participants
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
Latvia
|
2 participants
n=20 Participants
|
2 participants
n=20 Participants
|
2 participants
n=40 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
49 participants
n=20 Participants
|
50 participants
n=20 Participants
|
48 participants
n=40 Participants
|
147 participants
n=5 Participants
|
|
Region of Enrollment
Ukraine
|
21 participants
n=20 Participants
|
22 participants
n=20 Participants
|
22 participants
n=40 Participants
|
65 participants
n=5 Participants
|
|
Region of Enrollment
Bulgaria
|
6 participants
n=20 Participants
|
7 participants
n=20 Participants
|
8 participants
n=40 Participants
|
21 participants
n=5 Participants
|
|
Region of Enrollment
Serbia
|
45 participants
n=20 Participants
|
44 participants
n=20 Participants
|
44 participants
n=40 Participants
|
133 participants
n=5 Participants
|
|
Region of Enrollment
Croatia
|
2 participants
n=20 Participants
|
0 participants
n=20 Participants
|
1 participants
n=40 Participants
|
3 participants
n=5 Participants
|
|
Region of Enrollment
Colombia
|
1 participants
n=20 Participants
|
2 participants
n=20 Participants
|
1 participants
n=40 Participants
|
4 participants
n=5 Participants
|
|
Baseline PANSS total score
|
100.2 Score on a scale
STANDARD_DEVIATION 8.23 • n=20 Participants
|
101.0 Score on a scale
STANDARD_DEVIATION 10.50 • n=20 Participants
|
100.0 Score on a scale
STANDARD_DEVIATION 9.28 • n=40 Participants
|
100.4 Score on a scale
STANDARD_DEVIATION 9.37 • n=5 Participants
|
|
Baseline CGI-S score
|
5.05 score on a scale
STANDARD_DEVIATION 0.438 • n=20 Participants
|
5.02 score on a scale
STANDARD_DEVIATION 0.503 • n=20 Participants
|
4.99 score on a scale
STANDARD_DEVIATION 0.518 • n=40 Participants
|
5.02 score on a scale
STANDARD_DEVIATION 0.487 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 6Population: The mITT population included all participants that were randomized, received at least 1 dose of study drug, and had a baseline and at least 1 post-baseline efficacy measurement in PANSS or CGI-S.
PANSS was an interview-based assessment comprised of 30 items and 3 subscales (Positive, Negative, General Psychopathology). The Positive subscale assessed hallucinations, delusions, and related symptoms; the Negative subscale assessed emotional withdrawal, lack of motivation, and similar symptoms; and the General Psychopathology subscale addressed other symptoms such as anxiety, somatic concern, and disorientation. An anchored Likert scale from 1 - 7, where values of 2 and above indicated the presence of progressively more severe symptoms, was used to score each item. Individual items were then summed to determine scores for the 3 subscales, as well as a total score. PANSS total score ranges from: 30-210, where a higher score indicates greater severity. A negative change from baseline indicates improvement.
Outcome measures
| Measure |
Placebo
n=155 Participants
Participants received matched SEP-363856 placebo tablets, orally, once daily, from Day 1 up to Week 6.
|
SEP-363856 75mg
n=153 Participants
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 up to Week 6.
|
SEP-363856 100mg
n=152 Participants
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 to Day 7 and then the dose was increased to 100 mg from Day 8 up to Week 6.
|
|---|---|---|---|
|
Change From Baseline in PANSS Total Score at Week 6
|
-14.3 score on a scale
Standard Error 1.47
|
-16.4 score on a scale
Standard Error 1.49
|
-18.1 score on a scale
Standard Error 1.50
|
SECONDARY outcome
Timeframe: Baseline, Week 6Population: The mITT population included all participants that were randomized, received at least 1 dose of study drug, and had a baseline and at least 1 post-baseline efficacy measurement in PANSS or CGI-S.
The CGI-S is a single-item clinician-rated assessment of the participant's current illness state on a 7-point scale (score range: 1-7), where a higher score is associated with greater illness severity. A negative change from baseline indicates improvement.
Outcome measures
| Measure |
Placebo
n=155 Participants
Participants received matched SEP-363856 placebo tablets, orally, once daily, from Day 1 up to Week 6.
|
SEP-363856 75mg
n=153 Participants
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 up to Week 6.
|
SEP-363856 100mg
n=152 Participants
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 to Day 7 and then the dose was increased to 100 mg from Day 8 up to Week 6.
|
|---|---|---|---|
|
Change From Baseline in CGI-S Score at Week 6
|
-0.78 score on a scale
Standard Error 0.088
|
-0.91 score on a scale
Standard Error 0.089
|
-0.93 score on a scale
Standard Error 0.090
|
Adverse Events
Placebo
Serious events: 6 serious events
Other events: 36 other events
Deaths: 0 deaths
SEP-363856 75mg
Serious events: 14 serious events
Other events: 53 other events
Deaths: 0 deaths
SEP-363856 100mg
Serious events: 11 serious events
Other events: 52 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=155 participants at risk
Participants received matched SEP-363856 placebo tablets, orally, once daily, from Day 1 up to Week 6.
|
SEP-363856 75mg
n=155 participants at risk
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 up to Week 6.
|
SEP-363856 100mg
n=154 participants at risk
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 to Day 7 and then the dose was increased to 100 mg from Day 8 up to Week 6.
|
|---|---|---|---|
|
Psychiatric disorders
Schizophrenia
|
2.6%
4/155 • Number of events 4 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
8.4%
13/155 • Number of events 13 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
5.8%
9/154 • Number of events 9 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
|
Infections and infestations
Corona virus infection
|
0.65%
1/155 • Number of events 1 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.00%
0/155 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.65%
1/154 • Number of events 1 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
|
Psychiatric disorders
Malignant catatonia
|
0.00%
0/155 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.65%
1/155 • Number of events 1 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.00%
0/154 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Food poisoning
|
0.00%
0/155 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.65%
1/155 • Number of events 1 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.00%
0/154 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
0.00%
0/155 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.65%
1/155 • Number of events 1 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.00%
0/154 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
|
Investigations
Coronavirus test positive
|
0.65%
1/155 • Number of events 1 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.00%
0/155 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.00%
0/154 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
|
Injury, poisoning and procedural complications
Toxicity to various agents
|
0.00%
0/155 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.00%
0/155 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
0.65%
1/154 • Number of events 1 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
Other adverse events
| Measure |
Placebo
n=155 participants at risk
Participants received matched SEP-363856 placebo tablets, orally, once daily, from Day 1 up to Week 6.
|
SEP-363856 75mg
n=155 participants at risk
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 up to Week 6.
|
SEP-363856 100mg
n=154 participants at risk
Participants received SEP-363856 tablet, orally, once daily at a starting dose of 50 mg on Day 1 through Day 3 followed by dose-escalation to 75 mg from Day 4 to Day 7 and then the dose was increased to 100 mg from Day 8 up to Week 6.
|
|---|---|---|---|
|
Psychiatric disorders
Insomnia
|
5.8%
9/155 • Number of events 13 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
9.7%
15/155 • Number of events 18 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
8.4%
13/154 • Number of events 13 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
|
Psychiatric disorders
Schizophrenia
|
4.5%
7/155 • Number of events 7 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
7.1%
11/155 • Number of events 11 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
8.4%
13/154 • Number of events 15 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
|
Psychiatric disorders
Anxiety
|
3.9%
6/155 • Number of events 10 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
11.0%
17/155 • Number of events 23 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
7.8%
12/154 • Number of events 17 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
|
Psychiatric disorders
Agitation
|
5.2%
8/155 • Number of events 10 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
5.8%
9/155 • Number of events 21 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
3.2%
5/154 • Number of events 7 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
|
Nervous system disorders
Headache
|
9.0%
14/155 • Number of events 17 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
11.6%
18/155 • Number of events 22 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
11.0%
17/154 • Number of events 18 • From the first dose of study drug up to 7 days after the last dose of the study drug (up to approximately 7 weeks)
Safety population included all participants that were randomized and received at least 1 dose of study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place