Trial Outcomes & Findings for A Study to Evaluate the Efficacy and Safety of Adjunctive Troriluzole in Obsessive-Compulsive Disorder (NCT NCT04693351)
NCT ID: NCT04693351
Last Updated: 2026-05-22
Results Overview
Y-BOCS was a clinician-administered instrument used to assess the severity of obsessive compulsive disorder (OCD) symptoms and to monitor treatment response. The scale included 10 items: 5 items assessed obsessions and 5 items assessed compulsions. Each item was rated from 0 to 4, generating an obsessions subscale score (0-20), a compulsions subscale score (0-20), and a total score ranging from 0 to 40. Higher scores indicated greater OCD symptom severity. Negative change (or reduction in score) indicates improvement.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
589 participants
Primary outcome timeframe
Baseline and Week 8
Results posted on
2026-05-22
Participant Flow
A total 589 participants were randomized, and 581 participants received at least one dose of study treatment. The study was conducted at multiple sites in the United States, United Kingdom, China and Europe.
Participants who were stable on standard of care (SOC) medication and having an inadequate response along with Yale-Brown Obsessive Compulsive Scale (YBOCS) baseline score of (a) 22 or 23 or (b) 24 and above were randomized.
Participant milestones
| Measure |
Troriluzole
Participants received troriluzole 200 milligrams (mg) (100 mg\*2) capsules orally once daily for the first two weeks and up-titrated to 280 mg (140 mg\*2) capsules orally once daily for the next eight weeks, in the double-blind randomization phase.
|
Placebo
Participants received placebo-matched to troriluzole capsules orally once daily for 10 weeks of the double-blind randomization phase.
|
|---|---|---|
|
Overall Study
STARTED
|
294
|
295
|
|
Overall Study
Treated
|
292
|
289
|
|
Overall Study
Y-BOCS Score >=24 Cohort
|
259
|
257
|
|
Overall Study
Y-BOCS Score 22-23 Cohort
|
35
|
38
|
|
Overall Study
COMPLETED
|
256
|
258
|
|
Overall Study
NOT COMPLETED
|
38
|
37
|
Reasons for withdrawal
| Measure |
Troriluzole
Participants received troriluzole 200 milligrams (mg) (100 mg\*2) capsules orally once daily for the first two weeks and up-titrated to 280 mg (140 mg\*2) capsules orally once daily for the next eight weeks, in the double-blind randomization phase.
|
Placebo
Participants received placebo-matched to troriluzole capsules orally once daily for 10 weeks of the double-blind randomization phase.
|
|---|---|---|
|
Overall Study
Adverse Event
|
12
|
9
|
|
Overall Study
Lack of Efficacy
|
1
|
1
|
|
Overall Study
Non-compliance
|
0
|
2
|
|
Overall Study
Protocol Violation
|
0
|
2
|
|
Overall Study
Withdrawal by Subject
|
17
|
14
|
|
Overall Study
Lost to Follow-up
|
6
|
7
|
|
Overall Study
Miscellaneous
|
2
|
2
|
Baseline Characteristics
A Study to Evaluate the Efficacy and Safety of Adjunctive Troriluzole in Obsessive-Compulsive Disorder
Baseline characteristics by cohort
| Measure |
Troriluzole
n=292 Participants
Participants received troriluzole 200 mg capsules orally once daily for the first two weeks and up-titrated to 280 mg capsules orally once daily for the next eight weeks, in the double-blind randomization phase.
|
Placebo
n=289 Participants
Participants received placebo-matched to troriluzole capsules orally once daily for 10 weeks of the double-blind randomization phase.
|
Total
n=581 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
37.0 years
STANDARD_DEVIATION 12.69 • n=2 Participants
|
36.8 years
STANDARD_DEVIATION 12.94 • n=4 Participants
|
36.9 years
STANDARD_DEVIATION 12.81 • n=6 Participants
|
|
Sex: Female, Male
Female
|
184 Participants
n=2 Participants
|
187 Participants
n=4 Participants
|
371 Participants
n=6 Participants
|
|
Sex: Female, Male
Male
|
108 Participants
n=2 Participants
|
102 Participants
n=4 Participants
|
210 Participants
n=6 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
36 Participants
n=2 Participants
|
30 Participants
n=4 Participants
|
66 Participants
n=6 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
237 Participants
n=2 Participants
|
245 Participants
n=4 Participants
|
482 Participants
n=6 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
19 Participants
n=2 Participants
|
14 Participants
n=4 Participants
|
33 Participants
n=6 Participants
|
|
Race/Ethnicity, Customized
Asian
|
58 Participants
n=2 Participants
|
51 Participants
n=4 Participants
|
109 Participants
n=6 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
12 Participants
n=2 Participants
|
14 Participants
n=4 Participants
|
26 Participants
n=6 Participants
|
|
Race/Ethnicity, Customized
White
|
215 Participants
n=2 Participants
|
211 Participants
n=4 Participants
|
426 Participants
n=6 Participants
|
|
Race/Ethnicity, Customized
Other
|
5 Participants
n=2 Participants
|
11 Participants
n=4 Participants
|
16 Participants
n=6 Participants
|
|
Race/Ethnicity, Customized
Not Reported
|
2 Participants
n=2 Participants
|
2 Participants
n=4 Participants
|
4 Participants
n=6 Participants
|
|
Baseline (YBOCS) total score
|
27.5 units on a scale
STANDARD_DEVIATION 3.62 • n=2 Participants
|
27.6 units on a scale
STANDARD_DEVIATION 3.52 • n=4 Participants
|
27.5 units on a scale
STANDARD_DEVIATION 3.57 • n=6 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 8Population: Modified Intent to Treat (mITT) population included randomized participants who received at least 1 dose of study therapy and had a non-missing baseline assessment and at least one non-missing post-baseline on-treatment efficacy assessment (based on YBOCS in person only) during the randomization phase. Participants with available data were analyzed. As planned, the primary analysis was conducted on the baseline Y-BOCS ≥24 stratification cohort.
Y-BOCS was a clinician-administered instrument used to assess the severity of obsessive compulsive disorder (OCD) symptoms and to monitor treatment response. The scale included 10 items: 5 items assessed obsessions and 5 items assessed compulsions. Each item was rated from 0 to 4, generating an obsessions subscale score (0-20), a compulsions subscale score (0-20), and a total score ranging from 0 to 40. Higher scores indicated greater OCD symptom severity. Negative change (or reduction in score) indicates improvement.
Outcome measures
| Measure |
Placebo
n=217 Participants
Participants received placebo-matched to troriluzole capsules orally once daily for 10 weeks of the double-blind randomization phase.
|
Troriluzole
n=229 Participants
Participants received troriluzole 200 mg capsules orally once daily for the first two weeks and up-titrated to 280 mg capsules orally once daily for the next eight weeks, in the double-blind randomization phase.
|
|---|---|---|
|
Change From Baseline in the Y-BOCS Total Score in the Baseline Y-BOCS ≥24 Stratification Cohort at Week 8 (Negative Change Indicates Symptom Improvement)
|
-4.59 score on a scale
Standard Error 0.454
|
-3.62 score on a scale
Standard Error 0.446
|
SECONDARY outcome
Timeframe: From first dose up to approximately 12 weeksPopulation: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo).
An Adverse event (AE) was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in a participant, that did not necessarily have a causal relationship with treatment. An AE could be any unfavourable/unintended sign including an abnormal laboratory finding, symptom/disease temporally associated with the use of the study drug, whether or not considered related to it. A TEAE was defined as any AE that developed, worsened, or became serious after first dose of test treatment
Outcome measures
| Measure |
Placebo
n=289 Participants
Participants received placebo-matched to troriluzole capsules orally once daily for 10 weeks of the double-blind randomization phase.
|
Troriluzole
n=292 Participants
Participants received troriluzole 200 mg capsules orally once daily for the first two weeks and up-titrated to 280 mg capsules orally once daily for the next eight weeks, in the double-blind randomization phase.
|
|---|---|---|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
|
172 Participants
|
199 Participants
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: mITT population. Participants with available data were analyzed. As planned, the primary analysis was conducted on the baseline Y-BOCS ≥24 stratification cohort.
The SDS was a participant-rated measure of functional disability. SDS was assessed in 3 domains: work/school (0-10), social life (0-10), and family life (0-10). The score from each domain was summed into a single dimensional measure of global functional impairment that ranges from 0 (unimpaired) to 30 (highly impaired). Higher scores indicated a more severe impairment.
Outcome measures
| Measure |
Placebo
n=185 Participants
Participants received placebo-matched to troriluzole capsules orally once daily for 10 weeks of the double-blind randomization phase.
|
Troriluzole
n=195 Participants
Participants received troriluzole 200 mg capsules orally once daily for the first two weeks and up-titrated to 280 mg capsules orally once daily for the next eight weeks, in the double-blind randomization phase.
|
|---|---|---|
|
Change From Baseline in Sheehan Disability Scale (SDS) Total Score in the Baseline Y-BOCS ≥24 Stratification Cohort at Week 8
|
-3.57 score on a scale
Standard Error 0.538
|
-2.47 score on a scale
Standard Error 0.530
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: mITT population. Participants with available data were analyzed. As planned, the primary analysis was conducted on the baseline Y-BOCS ≥24 stratification cohort.
The CGI-S was a clinician rated assessment of the participants current illness state on a 7-point scale. Scores ranged from 1 (normal, not at all ill) to 7 (among the most extremely ill patients). Higher scores indicated a more severe illness.
Outcome measures
| Measure |
Placebo
n=217 Participants
Participants received placebo-matched to troriluzole capsules orally once daily for 10 weeks of the double-blind randomization phase.
|
Troriluzole
n=228 Participants
Participants received troriluzole 200 mg capsules orally once daily for the first two weeks and up-titrated to 280 mg capsules orally once daily for the next eight weeks, in the double-blind randomization phase.
|
|---|---|---|
|
Change From Baseline in Clinical Global Impression of Severity (CGI-S) Total Score in the Baseline Y-BOCS ≥24 Stratification Cohort at Week 8
|
-0.49 score on a scale
Standard Error 0.069
|
-0.38 score on a scale
Standard Error 0.068
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline and Week 8Population: mITT population included randomized participants who received at least 1 dose of study therapy and had non-missing baseline assessment and at least one non-missing post-baseline on-treatment efficacy assessment (based on YBOCS in person only) during randomization phase. Participants with available data were analyzed. Additional pre-specified analysis on baseline Y-BOCS = 22 or 23 stratification cohort also conducted.
Y-BOCS was a clinician-administered instrument used to assess the severity of obsessive compulsive disorder (OCD) symptoms and to monitor treatment response. The scale included 10 items: 5 items assessed obsessions and 5 items assessed compulsions. Each item was rated from 0 to 4, generating an obsessions subscale score (0-20), a compulsions subscale score (0-20), and a total score ranging from 0 to 40. Higher scores indicated greater OCD symptom severity. Negative change (or reduction in score) indicates improvement.
Outcome measures
| Measure |
Placebo
n=35 Participants
Participants received placebo-matched to troriluzole capsules orally once daily for 10 weeks of the double-blind randomization phase.
|
Troriluzole
n=30 Participants
Participants received troriluzole 200 mg capsules orally once daily for the first two weeks and up-titrated to 280 mg capsules orally once daily for the next eight weeks, in the double-blind randomization phase.
|
|---|---|---|
|
Change From Baseline in the Y-BOCS Total Score in the Baseline Y-BOCS = 22 or 23 Stratification Cohort at Week 8 (Negative Change Indicates Symptom Improvement)
|
-4.20 score on a scale
Standard Error 1.339
|
-3.81 score on a scale
Standard Error 1.441
|
Adverse Events
Troriluzole
Serious events: 3 serious events
Other events: 116 other events
Deaths: 0 deaths
Placebo
Serious events: 2 serious events
Other events: 85 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Troriluzole
n=292 participants at risk
Participants received troriluzole 200 mg capsules orally once daily for the first two weeks and up-titrated to 280 mg capsules orally once daily for the next eight weeks, in the double-blind randomization phase.
|
Placebo
n=289 participants at risk
Participants received placebo-matched to troriluzole capsules orally once daily for 10 weeks of the double-blind randomization phase.
|
|---|---|---|
|
Psychiatric disorders
Obsessive-compulsive disorder
|
0.34%
1/292 • Number of events 1 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
0.00%
0/289 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
|
Psychiatric disorders
Panic attack
|
0.34%
1/292 • Number of events 1 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
0.00%
0/289 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
|
Gastrointestinal disorders
Haemorrhoids
|
0.34%
1/292 • Number of events 1 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
0.00%
0/289 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/292 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
0.35%
1/289 • Number of events 1 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
|
Injury, poisoning and procedural complications
Lower limb fracture
|
0.00%
0/292 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
0.35%
1/289 • Number of events 1 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
Other adverse events
| Measure |
Troriluzole
n=292 participants at risk
Participants received troriluzole 200 mg capsules orally once daily for the first two weeks and up-titrated to 280 mg capsules orally once daily for the next eight weeks, in the double-blind randomization phase.
|
Placebo
n=289 participants at risk
Participants received placebo-matched to troriluzole capsules orally once daily for 10 weeks of the double-blind randomization phase.
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
14.4%
42/292 • Number of events 50 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
8.3%
24/289 • Number of events 29 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
|
Infections and infestations
Nasopharyngitis
|
5.1%
15/292 • Number of events 17 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
3.5%
10/289 • Number of events 10 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
|
Nervous system disorders
Headache
|
14.0%
41/292 • Number of events 53 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
13.1%
38/289 • Number of events 52 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
|
Nervous system disorders
Dizziness
|
8.6%
25/292 • Number of events 25 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
3.8%
11/289 • Number of events 12 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
|
Infections and infestations
Upper respiratory tract infection
|
5.1%
15/292 • Number of events 18 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
3.1%
9/289 • Number of events 9 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
|
General disorders
Fatigue
|
9.2%
27/292 • Number of events 30 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
9.0%
26/289 • Number of events 28 • For Adverse Events: From first dose up to approximately 12 weeks For All-cause Mortality: From Day 1 up to approximately 12 weeks
For AEs: Safety Population included enrolled participants who received at least one dose of blinded study therapy (troriluzole or placebo). For All-Cause Mortality: Randomized population included enrolled subjects who received a treatment assignment from the Interactive Response Technology (IRT) system.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER