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Troriluzole

Phase 3

Generalized Anxiety Disorder | Small molecule | Psychiatry |Biohaven Ltd.|Last Updated: Jun 24, 2026

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment881

FDA Designations

PRIORITY_REVIEWFAST_TRACKORPHAN_DRUG

Clinical trial landscape

Troriluzole · 8 trials · 21 indications

Phase 3 4Phase 2 3Early Phase 1 1
NCT04693351A Study to Evaluate the Efficacy and Safety of Adjunctive Troriluzole in Obsessive-Compulsive DisorderObsessive-Compulsive Disorder
COMPLETED589 Analytics
NCT04641143Efficacy and Safety Study of Adjunctive Troriluzole in Obsessive Compulsive DisorderObsessive-Compulsive Disorder
COMPLETED456 Analytics
NCT03701399Troriluzole in Adult Participants With Spinocerebellar AtaxiaSpinocerebellar Ataxias
ACTIVE NOT_RECRUITING299 Analytics
NCT03829241Randomized Trial of Adult Participants With Generalized Anxiety DisorderGeneralized Anxiety Disorder
COMPLETED881 Analytics
PHASE3COMPLETED
A Study to Evaluate the Efficacy and Safety of Adjunctive Troriluzole in Obsessive-Compulsive Disorder
Obsessive-Compulsive DisorderUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety Study of Adjunctive Troriluzole in Obsessive Compulsive Disorder
Obsessive-Compulsive DisorderUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Troriluzole in Adult Participants With Spinocerebellar Ataxia
Spinocerebellar AtaxiasUnlock trial analytics
PHASE3COMPLETED
Randomized Trial of Adult Participants With Generalized Anxiety Disorder
Generalized Anxiety DisorderUnlock trial analytics

Study Endpoints

Primary Endpoints

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)
Baseline and Week 8

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.

The total score on the Yale-Brown Obsessive Compulsive Scale (YBOCS)
Change in total score from baseline, assessed at screening, baseline, week 4, 8 &10

Improvement is measured by a lower total score

Randomization Phase: Change From Baseline in the Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA) Total Score at Week 48 in SCA Participants
Randomization Baseline; Week 48

The f-SARA was a 4-item performance based scale: 1-gait, 2-stance, 3-sitting, 4-speech disturbance (0:normal (no impairment), 1: mildly impaired function, but no assistance required, 2: moderately impaired function, but needs assistance for certain parts of task, 3: severely impaired function to degree that assistance is needed for all parts of the task, 4: unable to perform function). Total score was derived as sum of individual items; ranged from 0 to 16. Higher score indicated worst outcome. A negative change in score showed improvement.

Change From Baseline in the HAM-A Total Score at Week 8
Baseline, Week 8

The HAM-A was an investigator-administered scale and consisted of 14 items: anxious mood, tension, fears, insomnia, concentration, depressed mood, behavior at interview, somatic muscular, somatic sensory, cardiovascular, respiratory, gastrointestinal, genitourinary, and autonomic symptoms. Each item was scored on a scale of 0 (not present) to 4 (severe) with a total score range of 0-56. A decreased score indicated a decrease in anxiety symptoms.

Change From Baseline in Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) Total Score at Week 48
Baseline (Day 1) and Week 48

The ADAS-Cog is a structured scale that evaluates memory (word recall, word recognition), reasoning (following commands), language (naming, comprehension), orientation, ideational praxis (placing a letter in an envelope) and constructional praxis (copying geometric designs). Ratings of spoken language, language comprehension, word finding difficulty, and ability to remember test instructions were also obtained. The test was scored in terms of errors on a scale ranging from 0 (best) to 70 (worse), with higher scores indicate poorer performance and greater impairment.

Change From Baseline in Clinical Dementia Rating-Sum of Boxes (CDR-Sum of Boxes) Total Score at Week 48
Baseline (Day 1) and Week 48

The CDR-sum of boxes is a validated composite rating of cognition and everyday functioning used in longitudinal AD research which incorporates both informant input and direct assessment of performance. It assesses through semi-structured interview 3 cognitive domains including memory, orientation, and judgement/problem solving and 3 everyday functional domains including community affairs, home and hobbies and personal care. The individual domain score ranging from 0 (none) to 3 (severe) but the scores in each of these were combined to obtain a composite score (sum of boxes) ranging from 0 (best) to 18 (worst), with higher scores indicate poorer performance and greater impairment. The individual domain scores are added to create a sum of the box scores.

Change From Baseline in the Yale-Brown Obsessive Compulsive Scale (Y-BOCS) Total Score
Baseline, Week 12

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.

Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8
Baseline, Randomization Phase Week 8

The severity of ataxia was assessed with the SARA, an 8-item clinical rating scale from 0 (no ataxia) to 40 (most severe ataxia). The total score was derived as the sum of the individual items, which included gait (0-8), stance (0-6), sitting (0-4), speech disturbance (0-6), finger chase (0-4), nose-finger test (0-4), fast alternating hand movements (0-4), and heel-shin slide (0-4). Since the finger chase, nose-finger test, fast alternating hand movements, and heel-shin slide were repeated on both the right and left side, the average of the right and left side assessments was used to derive the total score. A negative change in score shows improvement.

The effect of troriluzole on high-gamma band power (a measure of neuronal activity) via electrocorticography during surgical resection
At time of surgery

Data will be summarized using descriptive statistics to compare between participants who received presurgical troriluzole and who did not

Secondary Endpoints

Number of Participants With Treatment Emergent Adverse Events (TEAEs)
From first dose up to approximately 12 weeks
Change From Baseline in Sheehan Disability Scale (SDS) Total Score in the Baseline Y-BOCS ≥24 Stratification Cohort at Week 8
Baseline and Week 8
Change From Baseline in Clinical Global Impression of Severity (CGI-S) Total Score in the Baseline Y-BOCS ≥24 Stratification Cohort at Week 8
Baseline and Week 8
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
TroriluzoleEXPERIMENTALParticipants 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.
PlaceboPLACEBO_COMPARATORParticipants received placebo-matched to troriluzole capsules orally once daily for 10 weeks of the double-blind randomization phase.
BHV-4157EXPERIMENTALtroriluzole, 280 mg (2 x 140 mg) capsules, QD
Troriluzole - Randomization PhaseEXPERIMENTALTroriluzole - Randomization Phase: Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks.
Placebo - Randomization PhasePLACEBO_COMPARATORPlacebo - Randomization Phase: Participants received matching placebo capsules orally QD for 8 weeks.
Troriluzole/Troriluzole - OLE (Open Label Extension) PhaseEXPERIMENTALParticipants received Troriluzole 140 mg capsules orally QD for 48 weeks in the extension period and were allowed to participate in 288 weeks for expanded extension phase, for a total of 336 weeks of open-label treatment.
Placebo/Troriluzole - OLE PhaseEXPERIMENTALParticipants who received placebo during randomization phase, received Troriluzole 140 mg capsules orally QD for 48 weeks in the extension period and were allowed to participate in 288 weeks for expanded extension phase, for a total of 336 weeks of open-label treatment.
Group A: Presurgical TroriluzoleEXPERIMENTAL18 participants will be randomly assigned to this group and with complete: * Baseline visit with assessments and MRI. * Cycle 0: * Day -6 through Day 0: Predetermined dose of Troriluzole 2x daily. * Day 0: pre-op MRI * Day 0: standard of care surgical resection of tumor * Day 0: post-op MRI * Cycle 1 through Cycle 3: --Days 1 through 28 of 28 day cycle: Predetermined dose of Troriluzole 2x daily. * Cycle 3 through End of Treatment: --Days 1 through 28 of 28 day cycle: Predetermined dose of Troriluzole 2x daily. * MRIs every 8 weeks while on treatment. * End of study visit with MRI * Follow up every 3 months for 1 year, and then every 6 months for the next 3 years.
Group B: Surgery + TroriluzoleEXPERIMENTAL9 participants will be randomly assigned to this group and with complete: * Baseline visit with assessments and MRI * Day 0: pre-op MRI * Day 0: standard of care surgical resection of tumor * Day 0: post-op MRI * Cycle 1 through Cycle 3: --Days 1 through 28 of 28 day cycle: Predetermined dose of Troriluzole 2x daily. * Cycle 3 through End of Treatment: --Days 1 through 28 of 28 day cycle: Predetermined dose of Troriluzole 2x daily. * MRIs every 8 weeks while on treatment. * End of study visit with MRI * Follow up every 3 months for 1 year, and then every 6 months for the next 3 years.

Interventions

NameTypeDescription
TroriluzoleDRUGCapsules for oral administration.
PlaceboDRUGDrug- matching capsules for oral administration.
Placebo oral capsuleDRUGOral matching placebo will be given daily for up to 48 weeks
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites89

Key Inclusion Criteria: 1. Primary diagnosis of OCD as per Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition as confirmed by the Mini International Neuropsychiatric Interview (MINI) at screening; the duration of the participants illness must be ≥ 1year 2. An inadequate response t...

Countries:United StatesCanadaChinaItalyNetherlandsSpainUnited Kingdom
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Recent Changes (Last 90 Days)

LOWJun 24, 2026NCT03701399Completion: 2026-06 → 2026-08
LOWJun 24, 2026NCT03701399Completion: 2026-06 → 2026-08
MEDIUMJun 22, 2026NCT04693351TRIAL_REMOVED: changed
MEDIUMJun 22, 2026NCT04693351TRIAL_REMOVED: changed
MEDIUMJun 22, 2026NCT04693351TRIAL_REMOVED: changed

Frequently asked questions about Troriluzole

What is Troriluzole used for?

Troriluzole is an investigational small molecule being developed for spinocerebellar ataxias, generalized anxiety disorder, obsessive-compulsive disorder, glioblastoma, and Alzheimer disease. It is in Phase 3 clinical development for spinocerebellar ataxia and obsessive-compulsive disorder.

What does Troriluzole target?

Troriluzole is a small molecule being studied for its effects on glutamate signaling in the central nervous system. It is being investigated for conditions including spinocerebellar ataxias, obsessive-compulsive disorder, generalized anxiety disorder, glioblastoma, and Alzheimer disease.

Who makes Troriluzole?

Troriluzole is being developed by Biohaven Ltd., a biopharmaceutical company. Biohaven is conducting clinical trials of Troriluzole in spinocerebellar ataxia and obsessive-compulsive disorder.

What phase is Troriluzole in?

Troriluzole is in Phase 3 clinical development. It has completed Phase 2 and Phase 3 trials in obsessive-compulsive disorder and spinocerebellar ataxia, and an additional Phase 3 trial in spinocerebellar ataxia is active but not recruiting.

What clinical trials is Troriluzole in?

Troriluzole has been studied in several clinical trials. NCT02960893 was a Phase 2 trial in spinocerebellar ataxia, NCT03299166 was a Phase 2 trial in obsessive-compulsive disorder, NCT03701399 is an active Phase 3 trial in spinocerebellar ataxia, and NCT04641143 was a Phase 3 trial in obsessive-compulsive disorder.

Is Troriluzole the same as BHV-4157?

Yes, Troriluzole is also known as BHV-4157. Clinical trials have used the name BHV-4157 to refer to this investigational drug, which is being developed by Biohaven Ltd.