Trial Outcomes & Findings for Study of the Safety and Efficacy of Fixed-dose Brexpiprazole (OPC-34712) as Adjunctive Therapy in the Treatment of Adults With Major Depressive Disorder With and Without Anxious Distress (NCT NCT02196506)

NCT ID: NCT02196506

Last Updated: 2018-08-21

Results Overview

To assess the change in the Montgomery-Asberg Depression Rating Scale (MADRS) Total Score from Baseline (End of Phase A \[Week 8\]) to Week 14. The MADRS was utilized as the primary efficacy assessment of the participant's level of depression and was administered utilizing the Structured Interview Guide for the MADRS (SIGMA). The MADRS consisted of 10 items each with 7 defined grades of severity. The rater decided whether the rating lied on predefined scale steps (0, 2, 4, 6) or between them (1, 3, 5). The 10 items were apparent sadness, reported sadness, inner tension, reduced sleep, reduced appetite, concentration difficulties, lassitude, inability to feel, pessimistic thoughts, suicidal thoughts. Each item was scored using a scale of 0 to 6 (a higher score indicates increased severity). The maximum total score was 60; 0, no symptom; 60, severely affected.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

837 participants

Primary outcome timeframe

From baseline (end of Phase A [Week 8]) to week 14

Results posted on

2018-08-21

Participant Flow

This trial was conducted in 837 participants at 51 trial sites in the following 5 countries:Germany,Hungary,Poland,Slovakia,andUnited States (US).A total of 1144 participants with major depressive disorder were screened for the trial, 837 enrolled into Phase A,394 were randomized into Phase B and 322 continued treatment with placebo+ADT in Phase A+

Trial consisted of a screening phase and 3 phases. In phase A (8-week single-blind prospective treatment phase) and in phase A+ (Single-blind phase A Responder), there was single treatment group. In phase B (6-week double-blind randomization phase), there were 2 treatment groups. All Outcome Measures were assessed in phase B.

Participant milestones

Participant milestones
Measure
Phase A: All ADT (Antidepressant Therapy)
Participants meeting entrance criteria who were experiencing a major depressive episode with a HAM-D17 Total Score of greater than or equal 18 at baseline were enrolled into an 8-week Single-blind Prospective Treatment Phase (Phase A). All participants received single-blind placebo plus an investigator determined, open-label, ADT. Once assigned to an ADT by the investigator, participants remained on the same ADT for the duration of the trial. At the Week 8 visit, the IWRS (Interactive web response system) determined based on scores entered by the investigator, whether a participant was a "Phase A Responder" or a "Phase A Inadequate Responder."
Phase B: Brexpiprazole + ADT
Participants received brexpiprazole 2 mg and ADT orally once daily for 6 weeks.
Phase B: Placebo + ADT
Participants received matching placebo and ADT orally once daily for 6 weeks.
Phase A +: ALL ADT
Phase A+ included participants who met criteria for a response at the end of the prospective treatment phase (Week 8 visit of Phase A) and participants who were not suitable for randomization in Phase B per the judgment of the investigator or medical monitor. Treatment response in Phase A was determined at the Week 8 visit based on improvement or lack of improvement of the participant's depressive symptoms, which was confirmed by clinical criteria that prospectively defined response. Participant response was determined from clinical data that were entered into the IWRS at each visit. Participants in Phase A+ received single-blind placebo+ADT for an additional 6 weeks, for a total of 14 weeks, and attended visits at Weeks 11 and 14.
Phase A
STARTED
837
0
0
0
Phase A
COMPLETED
716
0
0
0
Phase A
NOT COMPLETED
121
0
0
0
Phase B
STARTED
0
192
202
0
Phase B
COMPLETED
0
177
196
0
Phase B
NOT COMPLETED
0
15
6
0
Phase A+
STARTED
0
0
0
322
Phase A+
COMPLETED
0
0
0
308
Phase A+
NOT COMPLETED
0
0
0
14

Reasons for withdrawal

Reasons for withdrawal
Measure
Phase A: All ADT (Antidepressant Therapy)
Participants meeting entrance criteria who were experiencing a major depressive episode with a HAM-D17 Total Score of greater than or equal 18 at baseline were enrolled into an 8-week Single-blind Prospective Treatment Phase (Phase A). All participants received single-blind placebo plus an investigator determined, open-label, ADT. Once assigned to an ADT by the investigator, participants remained on the same ADT for the duration of the trial. At the Week 8 visit, the IWRS (Interactive web response system) determined based on scores entered by the investigator, whether a participant was a "Phase A Responder" or a "Phase A Inadequate Responder."
Phase B: Brexpiprazole + ADT
Participants received brexpiprazole 2 mg and ADT orally once daily for 6 weeks.
Phase B: Placebo + ADT
Participants received matching placebo and ADT orally once daily for 6 weeks.
Phase A +: ALL ADT
Phase A+ included participants who met criteria for a response at the end of the prospective treatment phase (Week 8 visit of Phase A) and participants who were not suitable for randomization in Phase B per the judgment of the investigator or medical monitor. Treatment response in Phase A was determined at the Week 8 visit based on improvement or lack of improvement of the participant's depressive symptoms, which was confirmed by clinical criteria that prospectively defined response. Participant response was determined from clinical data that were entered into the IWRS at each visit. Participants in Phase A+ received single-blind placebo+ADT for an additional 6 weeks, for a total of 14 weeks, and attended visits at Weeks 11 and 14.
Phase A
Participant Met Withdrawal Criteria
34
0
0
0
Phase A
Withdrawal by Subject
34
0
0
0
Phase A
Adverse Event
22
0
0
0
Phase A
Lost to Follow-up
9
0
0
0
Phase A
Protocol deviation
12
0
0
0
Phase A
Participant withdrawn by investigator
10
0
0
0
Phase B
Participant Met Withdrawal Criteria
0
0
1
0
Phase B
Lack of Efficacy
0
1
2
0
Phase B
Withdrawal by Participant
0
8
1
0
Phase B
Adverse Event
0
4
1
0
Phase B
Lost to Follow-up
0
2
1
0
Phase A+
Participant Met Withdrawal Criteria
0
0
0
1
Phase A+
Withdrawal by Participant
0
0
0
5
Phase A+
Adverse Event
0
0
0
1
Phase A+
Lost to Follow-up
0
0
0
6
Phase A+
Participant withdrawn by investigator
0
0
0
1

Baseline Characteristics

Study of the Safety and Efficacy of Fixed-dose Brexpiprazole (OPC-34712) as Adjunctive Therapy in the Treatment of Adults With Major Depressive Disorder With and Without Anxious Distress

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Brexpiprazole + ADT
n=192 Participants
Phase B: Brexpiprazole +Antidepressant therapy (ADT): Participants received brexpiprazole 2mg and ADT orally once daily for 6 weeks.
Placebo + ADT
n=202 Participants
Phase B: Placebo + Antidepressant therapy (ADT): Participants received matching placebo and ADT orally once daily for 6 weeks
Total
n=394 Participants
Total of all reporting groups
Age, Continuous
43.0 years
STANDARD_DEVIATION 12.7 • n=5 Participants
42.7 years
STANDARD_DEVIATION 12.5 • n=7 Participants
42.9 years
STANDARD_DEVIATION 12.6 • n=5 Participants
Sex: Female, Male
Female
147 Participants
n=5 Participants
144 Participants
n=7 Participants
291 Participants
n=5 Participants
Sex: Female, Male
Male
45 Participants
n=5 Participants
58 Participants
n=7 Participants
103 Participants
n=5 Participants
Race/Ethnicity, Customized
Hispanic or Latino
11 Participants
n=5 Participants
9 Participants
n=7 Participants
20 Participants
n=5 Participants
Race/Ethnicity, Customized
Not Hispanic or Latino
181 Participants
n=5 Participants
192 Participants
n=7 Participants
373 Participants
n=5 Participants
Race/Ethnicity, Customized
Unknown
0 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants
Region of Enrollment
Hungary
16 participants
n=5 Participants
16 participants
n=7 Participants
32 participants
n=5 Participants
Region of Enrollment
United States
100 participants
n=5 Participants
106 participants
n=7 Participants
206 participants
n=5 Participants
Region of Enrollment
Poland
26 participants
n=5 Participants
26 participants
n=7 Participants
52 participants
n=5 Participants
Region of Enrollment
Slovakia
23 participants
n=5 Participants
25 participants
n=7 Participants
48 participants
n=5 Participants
Region of Enrollment
Germany
27 participants
n=5 Participants
29 participants
n=7 Participants
56 participants
n=5 Participants

PRIMARY outcome

Timeframe: From baseline (end of Phase A [Week 8]) to week 14

Population: The primary analysis was performed on Efficacy Sample which included all randomized participants who took at least one dose of trial medication in Phase B and who have both an end of Phase A (Week 8) and at least one post-randomization MADRS Total Score during Phase B.

To assess the change in the Montgomery-Asberg Depression Rating Scale (MADRS) Total Score from Baseline (End of Phase A \[Week 8\]) to Week 14. The MADRS was utilized as the primary efficacy assessment of the participant's level of depression and was administered utilizing the Structured Interview Guide for the MADRS (SIGMA). The MADRS consisted of 10 items each with 7 defined grades of severity. The rater decided whether the rating lied on predefined scale steps (0, 2, 4, 6) or between them (1, 3, 5). The 10 items were apparent sadness, reported sadness, inner tension, reduced sleep, reduced appetite, concentration difficulties, lassitude, inability to feel, pessimistic thoughts, suicidal thoughts. Each item was scored using a scale of 0 to 6 (a higher score indicates increased severity). The maximum total score was 60; 0, no symptom; 60, severely affected.

Outcome measures

Outcome measures
Measure
Brexpiprazole + ADT
n=191 Participants
Phase B: Brexpiprazole +Antidepressant therapy (ADT): Participants received brexpiprazole 2mg and ADT orally once daily for 6 weeks.
Placebo + ADT
n=202 Participants
Phase B: Placebo + Antidepressant therapy (ADT): Participants received matching placebo and ADT orally once daily for 6 weeks
Change in the Montgomery-Asberg Depression
-10.4 Units on a scale
Standard Error 0.63
-8.07 Units on a scale
Standard Error 0.61

SECONDARY outcome

Timeframe: From baseline (end of Phase A [Week 8]) to week 14

Population: The primary analysis was performed on Efficacy Sample which included all randomized participants who took at least one dose of trial medication in Phase B and who have both an end of Phase A (Week 8) and at least one post-randomization MADRS Total Score during Phase B.

To assess the change in the Sheehan Disability Scale (SDS) Score (the mean of 3 individual item scores) from Baseline (End of Phase A \[Week 8\]) to Week 14 (End of Phase B). SDS was a 3-item clinician-rated questionnaire used to evaluate impairments in the domains of work, social life, and family life/home responsibility. Each item was scored using a scale of 0 to 10 (a higher score indicates symptoms have disrupted work, social life, and family life/home responsibility extremely). The maximum total score was 30; 0 = not at all, to 30 = extremely.

Outcome measures

Outcome measures
Measure
Brexpiprazole + ADT
n=187 Participants
Phase B: Brexpiprazole +Antidepressant therapy (ADT): Participants received brexpiprazole 2mg and ADT orally once daily for 6 weeks.
Placebo + ADT
n=200 Participants
Phase B: Placebo + Antidepressant therapy (ADT): Participants received matching placebo and ADT orally once daily for 6 weeks
Change in the Sheehan Disability Scale (SDS) From Baseline to End of Treatment
-1.63 Units on a scale
Standard Error 0.18
-1.41 Units on a scale
Standard Error 0.17

SECONDARY outcome

Timeframe: From baseline (end of Phase A [Week 8]) to week 14

Population: Subpopulation of \<25% Improvement Sample: Comprised of all participants in the Efficacy Sample who had \<25% improvement at the end of Phase A in MADRS Total Score.

To assess the change from end of Phase A (Week 8 visit) to end of Phase B (Week 14 visit) in MADRS Total Score for the subpopulation with \< 25% improvement from baseline of Phase A (Week 0) to end of Phase A (Week 8) in MADRS Total Score. The MADRS was utilized as the primary efficacy assessment of the participant's level of depression and was administered utilizing the Structured Interview Guide for the MADRS (SIGMA). The MADRS consisted of 10 items each with 7 defined grades of severity. The 10 items were apparent sadness, reported sadness, inner tension, reduced sleep, reduced appetite, concentration difficulties, lassitude, inability to feel, pessimistic thoughts, suicidal thoughts. Each item was scored using a scale of 0 to 6 (a higher score indicates increased severity). The maximum total score was 60; 0, no symptom; 60, severely affected.

Outcome measures

Outcome measures
Measure
Brexpiprazole + ADT
n=161 Participants
Phase B: Brexpiprazole +Antidepressant therapy (ADT): Participants received brexpiprazole 2mg and ADT orally once daily for 6 weeks.
Placebo + ADT
n=158 Participants
Phase B: Placebo + Antidepressant therapy (ADT): Participants received matching placebo and ADT orally once daily for 6 weeks
Change From End of Phase A to End of Phase B in MADRS Total Score for the Subpopulation With <25% Improvement From Baseline of Phase A to End of Phase A in MADRS Total Score
-11.1 Units on a scale
Standard Error 0.71
-8.87 Units on a scale
Standard Error 0.71

SECONDARY outcome

Timeframe: From baseline (end of Phase A [Week 8]) to week 14

Population: Subpopulation of Anxious Distress Sample: Comprised of all participants in the Efficacy Sample who had anxious distress as specified in DSM-V.

To assess the change from end of Phase A (Week 8) to end of Phase B (Week 14) in MADRS Total Score for the subpopulations with anxious distress as specified in DSM-V. The MADRS was utilized as the primary efficacy assessment of the participant's level of depression and was administered utilizing the Structured Interview Guide for the MADRS (SIGMA). The MADRS consisted of 10 items each with 7 defined grades of severity. The 10 items were apparent sadness, reported sadness, inner tension, reduced sleep, reduced appetite, concentration difficulties, lassitude, inability to feel, pessimistic thoughts, suicidal thoughts. Each item was scored using a scale of 0 to 6 (a higher score indicates increased severity). The maximum total score was 60; 0, no symptom; 60, severely affected.

Outcome measures

Outcome measures
Measure
Brexpiprazole + ADT
n=124 Participants
Phase B: Brexpiprazole +Antidepressant therapy (ADT): Participants received brexpiprazole 2mg and ADT orally once daily for 6 weeks.
Placebo + ADT
n=124 Participants
Phase B: Placebo + Antidepressant therapy (ADT): Participants received matching placebo and ADT orally once daily for 6 weeks
Change From End of Phase A to End of Phase B in MADRS Total Score for the Subpopulations With Anxious Distress as Specified in Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V).
-11.8 Units on a scale
Standard Error 0.81
-8.87 Units on a scale
Standard Error 0.81

Adverse Events

Brexpiprazole + ADT

Serious events: 1 serious events
Other events: 49 other events
Deaths: 0 deaths

Placebo + ADT

Serious events: 0 serious events
Other events: 35 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Brexpiprazole + ADT
n=192 participants at risk
Phase B: Brexpiprazole +Antidepressant therapy (ADT): Participants received brexpiprazole 2mg and ADT orally once daily for 6 weeks.
Placebo + ADT
n=202 participants at risk
Phase B: Placebo + Antidepressant therapy (ADT): Participants received matching placebo and ADT orally once daily for 6 weeks
Ear and labyrinth disorders
Vertigo
0.52%
1/192 • Number of events 1 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.
0.00%
0/202 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.

Other adverse events

Other adverse events
Measure
Brexpiprazole + ADT
n=192 participants at risk
Phase B: Brexpiprazole +Antidepressant therapy (ADT): Participants received brexpiprazole 2mg and ADT orally once daily for 6 weeks.
Placebo + ADT
n=202 participants at risk
Phase B: Placebo + Antidepressant therapy (ADT): Participants received matching placebo and ADT orally once daily for 6 weeks
Infections and infestations
Upper Respiratory Tract Infection
5.2%
10/192 • Number of events 11 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.
5.0%
10/202 • Number of events 11 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.
Investigations
Weight Increased
5.2%
10/192 • Number of events 10 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.
0.50%
1/202 • Number of events 1 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.
Nervous system disorders
Akathisia
8.3%
16/192 • Number of events 17 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.
5.0%
10/202 • Number of events 12 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.
Nervous system disorders
Headache
3.6%
7/192 • Number of events 7 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.
7.4%
15/202 • Number of events 24 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.
Psychiatric disorders
Restlessness
8.3%
16/192 • Number of events 16 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.
2.0%
4/202 • Number of events 4 • Participants were assessed for the occurrence of adverse events (AEs) once the participant signed informed consent form through completion of the trial; up to 14-weeks after the participant entered the trial and actively monitored up to 30 (+2) days after their last dose of investigational medicinal product (this could be up to 22 weeks depending on duration of trial participation).
Safety sample comprised of randomized participants in Phase B who received at least one dose of double-blind trial medication. No adverse events were assessed or collected from Phase A or A+ participants as those phases are single-blind, non-randomized, monotherapy treatment periods of the study and are outside of the pre-specified definition of the safety sample per the trial design.

Additional Information

Global Medical Affairs

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Phone: 800 562-3974

Results disclosure agreements

  • Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
  • Publication restrictions are in place

Restriction type: OTHER