Trial Outcomes & Findings for Troriluzole (BHV-4157) in Adult Participants With Obsessive Compulsive Disorder (NCT NCT03299166)

NCT ID: NCT03299166

Last Updated: 2026-01-22

Results Overview

The Y-BOCS is a clinician-administered scale used extensively in research and clinical practice to both rate severity of obsessive compulsive disorder (OCD) and to monitor improvement during treatment. It is designed to rate the severity of obsessions and compulsions as well as the type of symptoms in patients with OCD. The scale consists of 10 items; the first 5 items assess obsessions, and the last 5 items assess compulsions. Subscale scores can be calculated for obsessions and compulsions, each on a scale of 0 to 20. A total score ranging from 0 to 40 can then be correlated to overall severity. The higher the number on the Y-BOCS, the more severe the symptoms.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

426 participants

Primary outcome timeframe

Baseline, Week 12

Results posted on

2026-01-22

Participant Flow

The study was conducted at 56 sites in the United States.

A total of 426 participants were enrolled, of which 248 participants were randomized in a 1:1: ratio to receive troriluzole (BHV-4157) or placebo. A total of 178 participants were not randomized due to withdrawal of consent, lost to follow-up, failed inclusion/exclusion criteria, or other reasons.

Participant milestones

Participant milestones
Measure
Troriluzole - Randomization Phase
Randomization phase (Weeks 1 through 12): Participants received troriluzole 140 mg capsules once daily (QD) orally for the first 4 weeks and 200 mg (140 mg+ 60 mg capsules QD) for an additional 8 weeks in the double-blind (DB) randomization phase.
Placebo - Randomization Phase
Randomization phase (Weeks 1 through 12): Participants received troriluzole matching placebo capsules QD orally for up to 12 weeks in the DB randomization phase.
Overall Study
STARTED
124
124
Overall Study
Treated
122
122
Overall Study
Modified Intent-to- Treat (mITT) Participants
115
117
Overall Study
COMPLETED
101
109
Overall Study
NOT COMPLETED
23
15

Reasons for withdrawal

Reasons for withdrawal
Measure
Troriluzole - Randomization Phase
Randomization phase (Weeks 1 through 12): Participants received troriluzole 140 mg capsules once daily (QD) orally for the first 4 weeks and 200 mg (140 mg+ 60 mg capsules QD) for an additional 8 weeks in the double-blind (DB) randomization phase.
Placebo - Randomization Phase
Randomization phase (Weeks 1 through 12): Participants received troriluzole matching placebo capsules QD orally for up to 12 weeks in the DB randomization phase.
Overall Study
Adverse Event
9
4
Overall Study
Withdrawal by Subject
6
5
Overall Study
Physician Decision
1
1
Overall Study
Sponsor Decision
3
0
Overall Study
Lost to Follow-up
2
4
Overall Study
Randomization Error
0
1
Overall Study
Subject unable to return due to COVID
1
0
Overall Study
Unable to complete Week 12 visit at site
1
0

Baseline Characteristics

Troriluzole (BHV-4157) in Adult Participants With Obsessive Compulsive Disorder

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Troriluzole - Randomization Phase
n=122 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole 140 mg capsules once daily (QD) orally for the first 4 weeks and 200 mg (140 mg+ 60 mg capsules QD) for an additional 8 weeks in the double-blind (DB) randomization phase.
Placebo - Randomization Phase
n=122 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole matching placebo capsules QD orally for up to 12 weeks in the DB randomization phase.
Total
n=244 Participants
Total of all reporting groups
Age, Continuous
37.9 Years
STANDARD_DEVIATION 13.09 • n=270 Participants
35.5 Years
STANDARD_DEVIATION 13.26 • n=4 Participants
36.7 Years
STANDARD_DEVIATION 13.20 • n=9 Participants
Sex: Female, Male
Female
75 Participants
n=270 Participants
74 Participants
n=4 Participants
149 Participants
n=9 Participants
Sex: Female, Male
Male
47 Participants
n=270 Participants
48 Participants
n=4 Participants
95 Participants
n=9 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
10 Participants
n=270 Participants
17 Participants
n=4 Participants
27 Participants
n=9 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
112 Participants
n=270 Participants
105 Participants
n=4 Participants
217 Participants
n=9 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=270 Participants
0 Participants
n=4 Participants
0 Participants
n=9 Participants
Race (NIH/OMB)
American Indian or Alaska Native
1 Participants
n=270 Participants
2 Participants
n=4 Participants
3 Participants
n=9 Participants
Race (NIH/OMB)
Asian
8 Participants
n=270 Participants
7 Participants
n=4 Participants
15 Participants
n=9 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=270 Participants
0 Participants
n=4 Participants
0 Participants
n=9 Participants
Race (NIH/OMB)
Black or African American
12 Participants
n=270 Participants
7 Participants
n=4 Participants
19 Participants
n=9 Participants
Race (NIH/OMB)
White
100 Participants
n=270 Participants
103 Participants
n=4 Participants
203 Participants
n=9 Participants
Race (NIH/OMB)
More than one race
1 Participants
n=270 Participants
3 Participants
n=4 Participants
4 Participants
n=9 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=270 Participants
0 Participants
n=4 Participants
0 Participants
n=9 Participants
Total Y-BOCS score at Randomization
25.9 Units on a scale
STANDARD_DEVIATION 3.91 • n=270 Participants
26.0 Units on a scale
STANDARD_DEVIATION 4.32 • n=4 Participants
26.0 Units on a scale
STANDARD_DEVIATION 4.11 • n=9 Participants

PRIMARY outcome

Timeframe: Baseline, Week 12

Population: mITT: Randomized participants who received at least 1 dose of study therapy and provided a non-missing baseline assessment and at least 1 non-missing post-baseline efficacy assessment in the randomization phase.

The Y-BOCS is a clinician-administered scale used extensively in research and clinical practice to both rate severity of obsessive compulsive disorder (OCD) and to monitor improvement during treatment. It is designed to rate the severity of obsessions and compulsions as well as the type of symptoms in patients with OCD. The scale consists of 10 items; the first 5 items assess obsessions, and the last 5 items assess compulsions. Subscale scores can be calculated for obsessions and compulsions, each on a scale of 0 to 20. A total score ranging from 0 to 40 can then be correlated to overall severity. The higher the number on the Y-BOCS, the more severe the symptoms.

Outcome measures

Outcome measures
Measure
Troriluzole - Randomization Phase
n=115 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole 140 mg capsules once daily (QD) orally for the first 4 weeks and 200 mg (140 mg+ 60 mg capsules QD) for an additional 8 weeks in the double-blind (DB) randomization phase.
Placebo - Randomization Phase
n=117 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole matching placebo capsules QD orally for up to 12 weeks in the DB randomization phase.
Change From Baseline in the Yale-Brown Obsessive Compulsive Scale (Y-BOCS) Total Score
-5.91 Scores on a scale
Interval -7.05 to -4.77
-4.91 Scores on a scale
Interval -6.03 to -3.79

SECONDARY outcome

Timeframe: Up to 12 weeks

Population: Treated participants in the randomization phase

An AE is defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in participants or clinical investigation participants administered an investigational (medicinal) product and that does not necessarily have a causal relationship with this treatment. An SAE was defined as any event that met any of the following criteria at any dose: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect in the offspring of a participant who received study drug; other important medical events that may not have resulted in death, been life-threatening, or required hospitalization, or, based upon appropriate medical judgment, may have jeopardized the participant and may have required medical or surgical intervention to prevent other serious outcomes.

Outcome measures

Outcome measures
Measure
Troriluzole - Randomization Phase
n=122 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole 140 mg capsules once daily (QD) orally for the first 4 weeks and 200 mg (140 mg+ 60 mg capsules QD) for an additional 8 weeks in the double-blind (DB) randomization phase.
Placebo - Randomization Phase
n=122 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole matching placebo capsules QD orally for up to 12 weeks in the DB randomization phase.
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs) and AEs Leading to Study Drug Discontinuation in the DB Randomization Phase
Participants with at least 1 AE
61 participants
61 participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs) and AEs Leading to Study Drug Discontinuation in the DB Randomization Phase
Participants with at least 1 SAE
0 participants
0 participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs) and AEs Leading to Study Drug Discontinuation in the DB Randomization Phase
Participants with at least 1 AEs Leading to Study Drug Discontinuation
13 participants
5 participants

SECONDARY outcome

Timeframe: Up to 192 weeks

An AE is defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in participants or clinical investigation participants administered an investigational (medicinal) product and that does not necessarily have a causal relationship with this treatment. An SAE was defined as any event that met any of the following criteria at any dose: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect in the offspring of a participant who received study drug; other important medical events that may not have resulted in death, been life-threatening, or required hospitalization, or, based upon appropriate medical judgment may, have jeopardized the participant and may have required medical or surgical intervention to prevent other serious outcomes.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 12 weeks

Population: Treated participants in the randomization phase

Clinically significant laboratory abnormalities were defined as Grade 3 or 4 laboratory test results according to numeric laboratory test criteria found in Common Technical Criteria for Adverse Events Version 5.0 (2017) if available; otherwise, according to Division of Acquired Immune Deficiency Syndrome Table for Grading the Severity of Adult and Pediatric Adverse Events Corrected Version 2.1 (2017). Laboratory tests of clinical interest for troriluzole included absolute neutrophil count \< 500 per mm\^3, alanine aminotransferase or aspartate aminotransferase \> 3x upper limit of normal (ULN), and total bilirubin ≥ 2x ULN (Laboratory results were presented in US units).

Outcome measures

Outcome measures
Measure
Troriluzole - Randomization Phase
n=122 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole 140 mg capsules once daily (QD) orally for the first 4 weeks and 200 mg (140 mg+ 60 mg capsules QD) for an additional 8 weeks in the double-blind (DB) randomization phase.
Placebo - Randomization Phase
n=122 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole matching placebo capsules QD orally for up to 12 weeks in the DB randomization phase.
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Hemoglobin, serum
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Lymphocytes, low
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Lymphocytes, high
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Neutrophils
0 participants
1 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Platelets
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
White blood cells
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Alanine Aminotransferase
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Albumin
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Alkaline Phosphatase
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Aspartate Aminotransferase
2 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Bicarbonate
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Bilirubin
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Calcium, low
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Calcium, high
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Creatine Kinase
6 participants
2 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Creatinine
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Glucose, Serum, low
0 participants
1 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Glucose, Serum, high
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Lactate Dehydrogenase
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Potassium, low
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Potassium, high
1 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Sodium, low
0 participants
1 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Sodium, high
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Urate
0 participants
0 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Glucose, Urine
1 participants
1 participants
Number of Participants With Clinically Significant Laboratory Abnormalities During the DB Randomization Phase
Protein, Urine
0 participants
0 participants

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: The mITT participants in the randomization phase with available data were analyzed

The 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). If the participant has not worked or studied for reasons unrelated to OCD, then the total score was considered as missing.

Outcome measures

Outcome measures
Measure
Troriluzole - Randomization Phase
n=102 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole 140 mg capsules once daily (QD) orally for the first 4 weeks and 200 mg (140 mg+ 60 mg capsules QD) for an additional 8 weeks in the double-blind (DB) randomization phase.
Placebo - Randomization Phase
n=105 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole matching placebo capsules QD orally for up to 12 weeks in the DB randomization phase.
Change From Baseline in Functional Disability Assessed Using the Sheehan Disability Scale (SDS) Total Score
-4.68 Scores on a scale
Interval -6.01 to -3.36
-3.68 Scores on a scale
Interval -4.96 to -2.39

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: mITT participants

Measures severity of illness at the time of assessment compared with start of treatment. Scores range from 1 (normal, not at all ill) to 7 (among the most extremely ill patients).

Outcome measures

Outcome measures
Measure
Troriluzole - Randomization Phase
n=115 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole 140 mg capsules once daily (QD) orally for the first 4 weeks and 200 mg (140 mg+ 60 mg capsules QD) for an additional 8 weeks in the double-blind (DB) randomization phase.
Placebo - Randomization Phase
n=117 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole matching placebo capsules QD orally for up to 12 weeks in the DB randomization phase.
Change From Baseline in Clinical Global Impression of Severity Scale (CGI-S) Score
-0.67 Scores on a scale
Interval -0.84 to -0.5
-0.59 Scores on a scale
Interval -0.76 to -0.42

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: mITT participants

The Y-BOCS Obsessions Sub-scale consists of the last 5 items on the Y-BOCS and is measured on a scale of 0 to 20. A higher score on the Y-BOCS corresponds to greater symptom severity.

Outcome measures

Outcome measures
Measure
Troriluzole - Randomization Phase
n=115 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole 140 mg capsules once daily (QD) orally for the first 4 weeks and 200 mg (140 mg+ 60 mg capsules QD) for an additional 8 weeks in the double-blind (DB) randomization phase.
Placebo - Randomization Phase
n=117 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole matching placebo capsules QD orally for up to 12 weeks in the DB randomization phase.
Change From Baseline in the Y-BOCS Obsessions Sub-Scale Score
-2.91 Scores on a scale
Interval -3.53 to -2.28
-2.55 Scores on a scale
Interval -3.16 to -1.93

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 4 and Week 8

Population: mITT participants

The Y-BOCS is a clinician-administered scale used extensively in research and clinical practice to both rate severity of obsessive compulsive disorder (OCD) and to monitor improvement during treatment. It is designed to rate the severity of obsessions and compulsions as well as the type of symptoms in patients with OCD. The scale consists of 10 items; the first 5 items assess obsessions, and the last 5 items assess compulsions. Subscale scores can be calculated for obsessions and compulsions, each on a scale of 0 to 20. A total score ranging from 0 to 40 can then be correlated to overall severity. The higher the number on the Y-BOCS, the more severe the symptoms.

Outcome measures

Outcome measures
Measure
Troriluzole - Randomization Phase
n=115 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole 140 mg capsules once daily (QD) orally for the first 4 weeks and 200 mg (140 mg+ 60 mg capsules QD) for an additional 8 weeks in the double-blind (DB) randomization phase.
Placebo - Randomization Phase
n=117 Participants
Randomization phase (Weeks 1 through 12): Participants received troriluzole matching placebo capsules QD orally for up to 12 weeks in the DB randomization phase.
Change From Baseline in the Yale-Brown Obsessive Compulsive Scale (Y-BOCS) Total Score at Weeks 4 and 8
Week 4
-3.40 Scores on a scale
Interval -4.26 to -2.53
-2.93 Scores on a scale
Interval -3.78 to -2.08
Change From Baseline in the Yale-Brown Obsessive Compulsive Scale (Y-BOCS) Total Score at Weeks 4 and 8
Week 8
-5.10 Scores on a scale
Interval -6.16 to -4.04
-3.56 Scores on a scale
Interval -4.59 to -2.53

Adverse Events

Troriluzole - Randomization Phase

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

Placebo - Randomization Phase

Serious events: 0 serious events
Other events: 24 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Troriluzole - Randomization Phase
n=122 participants at risk
Randomization phase (Weeks 1 through 12): Participants received troriluzole 140 mg capsules once daily (QD) orally for the first 4 weeks and 200 mg (140 mg+ 60 mg capsules QD) for an additional 8 weeks in the double-blind (DB) randomization phase.
Placebo - Randomization Phase
n=122 participants at risk
Randomization phase (Weeks 1 through 12): Participants received troriluzole matching placebo capsules QD orally for up to 12 weeks in the DB randomization phase.
Nervous system disorders
Headache
8.2%
10/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
6.6%
8/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
Nervous system disorders
Dizziness
7.4%
9/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
1.6%
2/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
Nervous system disorders
Somnolence
6.6%
8/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
1.6%
2/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
Gastrointestinal disorders
Diarrhoea
0.82%
1/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
7.4%
9/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
Gastrointestinal disorders
Nausea
6.6%
8/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
1.6%
2/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
General disorders
Fatigue
7.4%
9/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
4.1%
5/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
Infections and infestations
Nasopharyngitis
5.7%
7/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).
0.82%
1/122 • Randomization Phase: Maximum duration: 12 weeks
Randomization phase: Treated participants in the randomization phase - enrolled participants who received at least 1 dose of blinded study therapy (troriluzole or placebo).

Additional Information

Chief Medical Officer

Biohaven Pharmaceuticals, Inc.

Phone: 203-404-0410

Results disclosure agreements

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

Restriction type: LTE60