Trial Outcomes & Findings for Safety and Efficacy of NYX-783 in Subjects With Post-Traumatic Stress Disorder (NCT NCT04044664)

NCT ID: NCT04044664

Last Updated: 2022-05-17

Results Overview

CAPS-5 \[Clinician Administered PTSD Scale for DSM-5 (Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition)\] is a structured interview for diagnosis and assessment of PTSD. The assessor combines information about frequency and intensity of an item into a severity rating (0-4). CAPS-5 total symptom severity score is calculated by summing severity scores for the 20 DSM-5 PTSD symptoms. CAPS-5 symptom cluster severity scores are calculated by summing the individual item severity scores for symptoms corresponding to a given DSM-5 cluster: intrusions (items 1-5, minimum score 0, maximum score 20), avoidance (items 6-7, minimum score 0, maximum score 8), negative alterations in cognitions and mood (items 8-14, minimum score 0, maximum score 28), and alterations in arousal and reactivity (items 15-20, minimum score 0, maximum score 24). CAPS-5 Total Scores range from 0 to 80. A higher score corresponds to more severe PTSD.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

160 participants

Primary outcome timeframe

Change from baseline to week 4 (Stage 1)

Results posted on

2022-05-17

Participant Flow

Participants were randomized to Placebo, NYX-783 50mg QD, or NYX-783 10mg QD during Stage 1. Participants were re-randomized to Placebo, NYX-783 50mg QD, or NYX-783 10mg QD in Stage 2. Participants who received Placebo in Stage 1 were classified as Responders or Non-responders in Stage 2. 160 subjects were randomized. 7 subjects received 50mg QW in a prior protocol version; they were not assigned to the Arms/Groups above. 1 subject was randomized in Stage 1 but did not receive study drug.

Participant milestones

Participant milestones
Measure
Placebo
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
Stage 1
STARTED
103
26
23
Stage 1
COMPLETED
86
19
20
Stage 1
NOT COMPLETED
17
7
3
Stage 2
STARTED
76
26
22
Stage 2
From Placebo, Responder
20
13
11
Stage 2
From Placebo, Non-Responder
18
13
11
Stage 2
From NYX-783 (10 mg or 50 mg)
38
0
0
Stage 2
COMPLETED
67
24
21
Stage 2
NOT COMPLETED
9
2
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Placebo
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
Stage 1
Adverse Event
3
3
1
Stage 1
Lost to Follow-up
3
2
2
Stage 1
Withdrawal by Subject
9
1
0
Stage 1
noncompliance, personal reason
2
0
0
Stage 1
randomization error
0
1
0
Stage 2
Adverse Event
2
0
0
Stage 2
Lost to Follow-up
3
2
1
Stage 2
Withdrawal by Subject
3
0
0
Stage 2
protocol deviation
1
0
0

Baseline Characteristics

Safety and Efficacy of NYX-783 in Subjects With Post-Traumatic Stress Disorder

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Placebo
n=103 Participants
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
n=26 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
n=23 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
Total
n=152 Participants
Total of all reporting groups
Age, Continuous
40.8 years
STANDARD_DEVIATION 11.42 • n=5 Participants
43.8 years
STANDARD_DEVIATION 12.54 • n=7 Participants
43.7 years
STANDARD_DEVIATION 11.96 • n=5 Participants
41.8 years
STANDARD_DEVIATION 11.7 • n=4 Participants
Sex: Female, Male
Female
61 Participants
n=5 Participants
15 Participants
n=7 Participants
16 Participants
n=5 Participants
92 Participants
n=4 Participants
Sex: Female, Male
Male
42 Participants
n=5 Participants
11 Participants
n=7 Participants
7 Participants
n=5 Participants
60 Participants
n=4 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
2 Participants
n=7 Participants
0 Participants
n=5 Participants
2 Participants
n=4 Participants
Race (NIH/OMB)
Asian
4 Participants
n=5 Participants
0 Participants
n=7 Participants
2 Participants
n=5 Participants
6 Participants
n=4 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
Race (NIH/OMB)
Black or African American
23 Participants
n=5 Participants
6 Participants
n=7 Participants
6 Participants
n=5 Participants
35 Participants
n=4 Participants
Race (NIH/OMB)
White
75 Participants
n=5 Participants
17 Participants
n=7 Participants
15 Participants
n=5 Participants
107 Participants
n=4 Participants
Race (NIH/OMB)
More than one race
1 Participants
n=5 Participants
1 Participants
n=7 Participants
0 Participants
n=5 Participants
2 Participants
n=4 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
Region of Enrollment
United States
103 participants
n=5 Participants
26 participants
n=7 Participants
23 participants
n=5 Participants
152 participants
n=4 Participants
Baseline CAPS-5 Total Score
40.6 units on a scale
STANDARD_DEVIATION 7.58 • n=5 Participants
36.5 units on a scale
STANDARD_DEVIATION 7.37 • n=7 Participants
38.3 units on a scale
STANDARD_DEVIATION 8.26 • n=5 Participants
39.6 units on a scale
STANDARD_DEVIATION 7.76 • n=4 Participants

PRIMARY outcome

Timeframe: Change from baseline to week 4 (Stage 1)

Population: Intent to Treat, all randomized subjects

CAPS-5 \[Clinician Administered PTSD Scale for DSM-5 (Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition)\] is a structured interview for diagnosis and assessment of PTSD. The assessor combines information about frequency and intensity of an item into a severity rating (0-4). CAPS-5 total symptom severity score is calculated by summing severity scores for the 20 DSM-5 PTSD symptoms. CAPS-5 symptom cluster severity scores are calculated by summing the individual item severity scores for symptoms corresponding to a given DSM-5 cluster: intrusions (items 1-5, minimum score 0, maximum score 20), avoidance (items 6-7, minimum score 0, maximum score 8), negative alterations in cognitions and mood (items 8-14, minimum score 0, maximum score 28), and alterations in arousal and reactivity (items 15-20, minimum score 0, maximum score 24). CAPS-5 Total Scores range from 0 to 80. A higher score corresponds to more severe PTSD.

Outcome measures

Outcome measures
Measure
Placebo
n=103 Participants
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
n=26 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
n=23 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
CAPS-5 [Clinician-Administered PTSD Scale for DSM-5 (Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition)] Total Score and Subscores
Cognition and Mood sub-score
-4.2 units on a scale
Standard Error 0.5
-5.7 units on a scale
Standard Error 1.05
-3.4 units on a scale
Standard Error 1.04
CAPS-5 [Clinician-Administered PTSD Scale for DSM-5 (Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition)] Total Score and Subscores
CAPS-5 Total Score
-13.4 units on a scale
Standard Error 1.06
-15.7 units on a scale
Standard Error 2.24
-11.6 units on a scale
Standard Error 2.19
CAPS-5 [Clinician-Administered PTSD Scale for DSM-5 (Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition)] Total Score and Subscores
Intrusion sub-score
-4.4 units on a scale
Standard Error 0.35
-4.3 units on a scale
Standard Error 0.75
-3.7 units on a scale
Standard Error 0.72
CAPS-5 [Clinician-Administered PTSD Scale for DSM-5 (Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition)] Total Score and Subscores
Avoidance sub-score
-1.8 units on a scale
Standard Error 0.21
-2.2 units on a scale
Standard Error 0.44
-1.4 units on a scale
Standard Error 0.44
CAPS-5 [Clinician-Administered PTSD Scale for DSM-5 (Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition)] Total Score and Subscores
Arousal and Reactivity sub-score
-2.7 units on a scale
Standard Error .28
-4.1 units on a scale
Standard Error 0.61
-3.5 units on a scale
Standard Error 0.59

SECONDARY outcome

Timeframe: Change from baseline to week 4 (Stage 1)

Population: Intent to Treat, all randomized subjects

Assess the effect of NYX-783 compared to placebo in changes of symptoms as measured by PCL-5. The PCL-5 ranges from 0 to 80; a higher score corresponds to more severe PTSD.

Outcome measures

Outcome measures
Measure
Placebo
n=103 Participants
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
n=26 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
n=23 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
PCL-5 (PTSD-Checklist for DSM-5)
-16.6 units on a scale
Standard Deviation 14.75
-19.9 units on a scale
Standard Deviation 13.58
-17.0 units on a scale
Standard Deviation 16.45

SECONDARY outcome

Timeframe: Change from baseline to week 4 (Stage 1)

Population: Intent to Treat, all randomized subjects

Assess the effects of NYX-783 compared to placebo in changes in sleep quality as measure by the PSQI Global Score. The PSQI is a questionnaire to assess sleep quality and disturbances. In scoring the PSQI, seven component scores are derived, each scored 0 (no difficulty) to 3 (severe difficulty). The component scores are summed to produce a global score (range 0 to 21). Higher scores indicate worse sleep quality. A score \>5 indicates significant sleep disturbance.

Outcome measures

Outcome measures
Measure
Placebo
n=103 Participants
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
n=26 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
n=23 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
PSQI (Pittsburgh Sleep Quality Index) Global Score
-1.5 units on a scale
Standard Deviation 3.37
-1.2 units on a scale
Standard Deviation 2.89
-1.8 units on a scale
Standard Deviation 4.04

SECONDARY outcome

Timeframe: Change from baseline to week 4 (Stage 1)

Population: Intent to Treat, all randomized subjects

Assess the effects of NYX-783 compared to placebo in changes in sleep quality as measure by the PSQI-A Global Score. The PSQI-A score ranges from 0-21; lower scores represent less disruptive behavior.

Outcome measures

Outcome measures
Measure
Placebo
n=86 Participants
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
n=19 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
n=20 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
PSQI-A (Pittsburgh Sleep Quality Index-Addendum) Global Score
-2.9 units on a scale
Standard Deviation 3.76
-1.9 units on a scale
Standard Deviation 2.66
-3.1 units on a scale
Standard Deviation 4.84

SECONDARY outcome

Timeframe: Change from baseline to week 4 (Stage 1)

Population: Intent to Treat, all randomized subjects

Assess the effect of NYX-783 compared to placebo in changes in cognitive function as measured by the BAC Symbol Coding. BAC symbol coding score is a count which ranges from 0 to 110; higher scores represent higher function.

Outcome measures

Outcome measures
Measure
Placebo
n=103 Participants
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
n=26 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
n=23 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
BAC (Brief Assessment of Cognition) Symbol Coding
3.8 units on a scale
Standard Deviation 8.07
2.9 units on a scale
Standard Deviation 9.00
1.3 units on a scale
Standard Deviation 5.37

SECONDARY outcome

Timeframe: Change from baseline to week 4 (Stage 1)

Population: Intent to Treat, all randomized subjects

Assess the effect of NYX-783 compared to placebo in the change in global clinical severity of PTSD symptoms as measured by the CGI-S. The Clinical Global Impressions-Severity (CGI-S) score ranges from 1 (normal, not at all ill) to 7 (among the most extremely ill subjects).

Outcome measures

Outcome measures
Measure
Placebo
n=103 Participants
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
n=26 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
n=23 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
CGI-S (Clinical Global Impression - Severity)
-1.1 units on a scale
Standard Deviation 1.09
-1.2 units on a scale
Standard Deviation 1.03
-1.3 units on a scale
Standard Deviation 0.99

SECONDARY outcome

Timeframe: From baseline to week 4 (Stage 1)

Population: Intent to Treat, all randomized subjects

The HADS is a self-assessment tool consisting of two subscales, one for anxiety (HADS-A) and one for depression (HADS-D). Scores for items in each subscale of the HADS are summed to produce an anxiety score (HADS-A) or a Depression score (HADS-D), or can be added to produce a total score (HADS-T). Each item is rated on a 4-point scale (ranging from 0 = no not at all, to 3 = yes definitely), for a total score ranging from 0-42 and sub scores for anxiety or depression ranging from 0-21 for each subscale. Higher scores represent more severe anxiety or depression. HADS-A is presented here.

Outcome measures

Outcome measures
Measure
Placebo
n=103 Participants
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
n=26 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
n=23 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
HADS-A (Hospital Anxiety and Depression Scale)
-3.0 units on a scale
Standard Error .34
-4.4 units on a scale
Standard Error 0.72
-4.2 units on a scale
Standard Error 0.7

POST_HOC outcome

Timeframe: Baseline to Week 4 (Stage 1)

Population: Intent to Treat, subjects who completed Week 4

Clinician Administered PTSD Scale for DSM-5 (CAPS-5) is a 30-item structured interview for diagnosis and assessment of PTSD. A higher score corresponds to more severe PTSD.

Outcome measures

Outcome measures
Measure
Placebo
n=103 Participants
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
n=26 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
n=23 Participants
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
Responders (30% and 50% Improvement From Baseline) in CAPS-5 Total Score
30% decrease from baseline
38 participants
14 participants
9 participants
Responders (30% and 50% Improvement From Baseline) in CAPS-5 Total Score
50% decrease from baseline
23 participants
9 participants
6 participants

Adverse Events

Placebo

Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths

NYX-783 High Dose (50 mg QD)

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

NYX-783 Low Dose (10 mg QD)

Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Placebo
n=141 participants at risk
Placebo oral capsule: Matching placebo capsules.
NYX-783 High Dose (50 mg QD)
n=52 participants at risk
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
NYX-783 Low Dose (10 mg QD)
n=46 participants at risk
NYX-783: NYX-783 is a small molecule that modulates the N-methyl-D-aspartate receptor (NMDAR).
Nervous system disorders
Headache
5.7%
8/141 • Number of events 8 • Stage 1: 4 weeks, Stage 2: 4 weeks
Adverse Events were assessed at protocol specified study visits.
3.8%
2/52 • Number of events 2 • Stage 1: 4 weeks, Stage 2: 4 weeks
Adverse Events were assessed at protocol specified study visits.
6.5%
3/46 • Number of events 3 • Stage 1: 4 weeks, Stage 2: 4 weeks
Adverse Events were assessed at protocol specified study visits.
Infections and infestations
Urinary Tract Infection
0.71%
1/141 • Number of events 1 • Stage 1: 4 weeks, Stage 2: 4 weeks
Adverse Events were assessed at protocol specified study visits.
3.8%
2/52 • Number of events 2 • Stage 1: 4 weeks, Stage 2: 4 weeks
Adverse Events were assessed at protocol specified study visits.
0.00%
0/46 • Stage 1: 4 weeks, Stage 2: 4 weeks
Adverse Events were assessed at protocol specified study visits.

Additional Information

Aptinyx Clinical Development

Aptinyx

Phone: 847-871-0377

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place