Trial · NCT03701399

Troriluzole in Adult Participants With Spinocerebellar Ataxia

NCT03701399, Phase 3, Completed, Biohaven Pharmaceuticals, Inc.; results first posted 2025-02-10

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Troriluzole in Adult Participants With Spinocerebellar Ataxia. Trials, record NCT03701399. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03701399

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Troriluzole in Adult Participants With Spinocerebellar Ataxia
NCT number: NCT03701399
Status: Completed
Phases: Phase 3
Enrollment: 299 participants, actual
Lead sponsor: Biohaven Pharmaceuticals, Inc.
Conditions: Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 10
Interventions: troriluzole, Placebo
First posted: 2018-10-10
Last updated: 2026-11-12
Results first posted: 2025-02-10

Source ClinicalTrials.gov

Registered outcomes11

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	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.
Secondary	Randomization Phase: Change From Baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) Total Score at Week 48 in SCA Participants	Randomization Baseline, Week 48	PIFAS was a 17-item instrument designed to assess the level of functional disability. The scale was rater administered and the items were selected to encompass domains of relevance to SCA (i.e., mobility, speech/swallowing, and fatigue), and to capture function and activities of daily living within these domains. Statements were rated on a 5-point Likert scale ranging from "0" reflecting "not at all" to "4" reflecting "very much." The total score was derived as the sum of the individual items, ranged between 0 to 68. Higher score indicated a worst outcome. A negative change in score showed improvement.
Secondary	Randomization Phase: Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Total Score at Week 48 in SCA Participants	Randomization Baseline, Week 48	FARS was a scale consisting of combined timed measures of performance, FUNC (0-6, higher score indicated worst outcome), ADL with paramount neurologic examination (0-36, higher score indicated worst outcome), FARSn (0-125, higher score indicated worst outcome). Total score was sum of these 3 subscales: 0-167, higher score indicated worst outcome. For this outcome measure, only FARS-ADL sub-scale was evaluated. It was multicomponent scale designed to assess neurological domains affected in Friedreich's Ataxia, another hereditary cerebellar ataxia disorder. This subscale had been validated in Friedreich's ataxia, was a measure of functional disability, and correlated with SARA total scores. It assessed 9 areas of ADL with response categories rated on 5-point scale with "0" reflecting "normal" and "4" reflecting inability to perform specific function. Total score was derived as sum of individual items, ranging 0-36, higher score indicated worst outcome. FARS-ADL was rater administered.
Secondary	Randomization Phase: Change From Baseline in Functional Staging for Ataxia Scale From the Friedreich's Ataxia Rating Scale (FARS-FUNC) Total Score at Week 48 in SCA Participants	Randomization Baseline, Week 48	FARS was a scale consisting of combined timed measures of performance, FUNC (0-6, higher score indicated worst outcome), ADL with paramount neurologic examination (0-36, higher score indicated worst outcome), FARSn (0-125, higher score indicated worst outcome). The total score was a sum of these 3 subscales; 0-167, higher score indicated worst outcome. For this outcome measure, only the FARS-FUNC sub-scale was evaluated. FARS-FUNC was a subscale of the FARS designed to provide functional staging for ataxia in which clinicians were asked to assess function on a 6-stage staging system, with "0" reflecting "normal" and "6" reflecting a stage in participant was "total disabled".
Secondary	Randomization Phase: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (TESAEs); and AEs Leading to Treatment Discontinuation	From first dose of study drug to Week 48 plus 30 days (maximum duration: 52 weeks)	An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in a participant that did not necessarily had a causal relationship with this treatment. An SAE was defined as any AE that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect in the offspring of the participant or was considered another important medical event. Treatment-emergent AEs (TEAEs) in the randomization phase included any AE with an onset date on or after the first day of double-blind study drug and up to the first day of open-label study drug in the extension phase (for participants entering the extension phase) or last day of the randomization phase study drug + 30 days.
Other	Randomization Phase: Change From Baseline in the f-SARA Total Score at Week 48 in SCA3 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 shows improvement.
Other	Randomization Phase: Change From Baseline in PIFAS Total Score at Week 48 in SCA3 Participants	Randomization Baseline, Week 48	PIFAS was a 17-item instrument designed to assess the level of functional disability. The scale is rater administered and the items were selected to encompass domains of relevance to SCA (i.e., mobility, speech/swallowing, and fatigue), and to capture function and activities of daily living within these domains. Statements are rated on a 5-point Likert scale ranging from "0" reflecting "not at all" to "4" reflecting "very much." The total score is derived as the sum of the individual items, ranged between 0 to 68. Higher score indicated a worst outcome. A negative change in score showed improvement.
Other	Randomization Phase: Change From Baseline in FARS-ADL Total Score at Week 48 in SCA3 Participants	Randomization Baseline, Week 48	FARS was a scale consisting of combined timed measures of performance, FUNC (0-6, higher score indicated worst outcome), ADL with paramount neurologic examination (0-36, higher score indicated worst outcome), FARSn (0-125, higher score indicated worst outcome). Total score was sum of these 3 subscales: 0-167, higher score indicated worst outcome. For this outcome measure, only FARS-ADL sub-scale was evaluated. It was multicomponent scale designed to assess neurological domains affected in Friedreich's Ataxia, another hereditary cerebellar ataxia disorder. This subscale had been validated in Friedreich's ataxia, was a measure of functional disability, and correlated with SARA total scores. It assessed 9 areas of ADL with response categories rated on 5-point scale with "0" reflecting "normal" and "4" reflecting inability to perform specific function. Total score was derived as sum of individual items, ranging 0-36, higher score indicated worst outcome. FARS-ADL was rater administered.
Other	Randomization Phase: Change From Baseline in FARS-FUNC Total Score at Week 48 in SCA3 Participants	Randomization Baseline, Week 48	FARS was a scale consisting of combined timed measures of performance, FUNC (0-6, higher score indicated worst outcome), ADL with paramount neurologic examination (0-36, higher score indicated worst outcome), FARSn (0-125, higher score indicated worst outcome). The total score was a sum of these 3 subscales; 0-167, higher score indicated worst outcome. For this outcome measure, only the FARS-FUNC sub-scale was evaluated. FARS-FUNC was a subscale of the FARS designed to provide functional staging for ataxia in which clinicians were asked to assess function on a 6-stage staging system, with "0" reflecting "normal" and "6" reflecting a stage in participant was "total disabled".
Other	Change From Baseline in Clinical Global Impression - Global Improvement Scale (CGI-I) in SCA3 Participants	Randomization Baseline; Week 48	The CGI-I was a 7-point scale that required the clinician to assess how much the participant's illness had improved or worsened relative to the baseline visit and it was rated on a 7 point scale: 1= "Very much improved", 2= "Much improved", 3= "Minimally improved", 4= "No change", 5= "Minimally worse", 6= "Much worse" and 7= "Very much worse". Higher scores indicated greater impairment.

1 to 10 of 11Source ClinicalTrials.gov

See all 11 registered outcomes

Outcomes11

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	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 modified intent-to-treat (mITT) analysis set in randomization phase included randomized participants who received at least one dose of double-blind study medication (troriluzole or placebo) during the randomization phase and provided a non-missing baseline measurement and at least one non-missing post-baseline efficacy measurement during the randomization phase. Participants with available data were included. Pre-specified randomization strata by SCA genotype.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Randomization Phase: Change From Baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) Total Score at Week 48 in SCA Participants	Randomization Baseline, Week 48	Participants in mITT analysis set with available data were analyzed.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Randomization Phase: Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Total Score at Week 48 in SCA Participants	Randomization Baseline, Week 48	Participants in mITT analysis set with available data were analyzed.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Randomization Phase: Change From Baseline in Functional Staging for Ataxia Scale From the Friedreich's Ataxia Rating Scale (FARS-FUNC) Total Score at Week 48 in SCA Participants	Randomization Baseline, Week 48	Participants in mITT analysis set with available data were analyzed.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Randomization Phase: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (TESAEs); and AEs Leading to Treatment Discontinuation	From first dose of study drug to Week 48 plus 30 days (maximum duration: 52 weeks)	The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo).	participants	NUMBER
OTHER_PRE_SPECIFIED	Randomization Phase: Change From Baseline in the f-SARA Total Score at Week 48 in SCA3 Participants	Randomization Baseline; Week 48	Participants in mITT analysis set with available data were analyzed.	score on a scale	LEAST_SQUARES_MEAN
OTHER_PRE_SPECIFIED	Randomization Phase: Change From Baseline in PIFAS Total Score at Week 48 in SCA3 Participants	Randomization Baseline, Week 48	Participants in mITT analysis set with available data were analyzed.	score on a scale	LEAST_SQUARES_MEAN
OTHER_PRE_SPECIFIED	Randomization Phase: Change From Baseline in FARS-ADL Total Score at Week 48 in SCA3 Participants	Randomization Baseline, Week 48	Participants in mITT analysis set with available data were analyzed.	score on a scale	LEAST_SQUARES_MEAN
OTHER_PRE_SPECIFIED	Randomization Phase: Change From Baseline in FARS-FUNC Total Score at Week 48 in SCA3 Participants	Randomization Baseline, Week 48	Participants in mITT analysis set with available data were analyzed.	score on a scale	LEAST_SQUARES_MEAN
OTHER_PRE_SPECIFIED	Change From Baseline in Clinical Global Impression - Global Improvement Scale (CGI-I) in SCA3 Participants	Randomization Baseline; Week 48	Participants in mITT analysis set with available data were analyzed.	score on a scale	LEAST_SQUARES_MEAN

1 to 10 of 11Source ClinicalTrials.gov

See all 11 outcomes

Results by group34

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
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	Troriluzole	All SCA	0.20	Standard Error 0.19	score on a scale
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	Placebo	All SCA	0.27	Standard Error 0.18	score on a scale
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	Troriluzole	SCA3	-0.03	Standard Error 0.20	score on a scale
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	Placebo	SCA3	0.53	Standard Error 0.19	score on a scale
Randomization Phase: Change From Baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) Total Score at Week 48 in SCA Participants	Troriluzole	All SCA	-0.93	Standard Error 1.49	score on a scale
Randomization Phase: Change From Baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) Total Score at Week 48 in SCA Participants	Placebo	All SCA	1.03	Standard Error 1.43	score on a scale
Randomization Phase: Change From Baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) Total Score at Week 48 in SCA Participants	Troriluzole	SCA3	0.56	Standard Error 1.65	score on a scale
Randomization Phase: Change From Baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) Total Score at Week 48 in SCA Participants	Placebo	SCA3	2.96	Standard Error 1.67	score on a scale
Randomization Phase: Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Total Score at Week 48 in SCA Participants	Troriluzole	All SCA	0.453	Standard Error 0.502	score on a scale
Randomization Phase: Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Total Score at Week 48 in SCA Participants	Placebo	All SCA	-0.017	Standard Error 0.480	score on a scale

1 to 10 of 34Source ClinicalTrials.gov

See all 34 results by group

Participant flow8

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Troriluzole	109
Overall Study	STARTED	Placebo	109
Overall Study	Treated	Troriluzole	108
Overall Study	Treated	Placebo	109
Overall Study	COMPLETED	Troriluzole	96
Overall Study	COMPLETED	Placebo	96
Overall Study	NOT COMPLETED	Troriluzole	13
Overall Study	NOT COMPLETED	Placebo	13

Source ClinicalTrials.gov

Adverse events64

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Headache	Nervous system disorders	Troriluzole	13	108
other	Headache	Nervous system disorders	Placebo	8	109
other	Dizziness	Nervous system disorders	Troriluzole	9	108
other	Dizziness	Nervous system disorders	Placebo	11	109
other	Fall	Injury, poisoning and procedural complications	Troriluzole	13	108
other	Fall	Injury, poisoning and procedural complications	Placebo	24	109
other	Contusion	Injury, poisoning and procedural complications	Troriluzole	7	108
other	Contusion	Injury, poisoning and procedural complications	Placebo	7	109
other	Covid-19	Infections and infestations	Troriluzole	6	108
other	Covid-19	Infections and infestations	Placebo	3	109

1 to 10 of 64Source ClinicalTrials.gov

See all 64 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Troriluzole	1	109
Total, all-cause mortality	Placebo	1	109
Total, other adverse events	Troriluzole	54	108
Total, other adverse events	Placebo	58	109
Total, serious adverse events	Troriluzole	6	108
Total, serious adverse events	Placebo	8	109

Source ClinicalTrials.gov

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
34115419	DERIVED	PubMed
38814532	DERIVED	PubMed
39806095	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Troriluzole vs Placebo: deaths from any cause	Trial comparisons
Troriluzole vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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