Trial Outcomes & Findings for Trial in Adult Participants With Spinocerebellar Ataxia (SCA) (NCT NCT02960893)

Last Updated: 2025-10-08

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

141 participants

Primary outcome timeframe

Baseline, Randomization Phase Week 8

Results posted on

2025-10-08

Participant Flow

181 participants were screened, and 141 of these participants were randomized to treatment at 18 sites in the United States.

Participant milestones

Participant milestones
Measure	Troriluzole - Randomization Phase Participants received Troriluzole 140 milligrams (mg) capsules orally once daily (QD) for 8 weeks.	Placebo - Randomization Phase Participants received matching placebo capsules orally QD for 8 weeks.	Troriluzole/Troriluzole - Open Label Extension (OLE) Phase Participants 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 Phase Participants 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.
Randomization Phase (8 Weeks) STARTED	71	70	0	0
Randomization Phase (8 Weeks) COMPLETED	64	68	0	0
Randomization Phase (8 Weeks) NOT COMPLETED	7	2	0	0
Open Label Extension Phase (336 Weeks) STARTED	0	0	64	67
Open Label Extension Phase (336 Weeks) COMPLETED	0	0	28	36
Open Label Extension Phase (336 Weeks) NOT COMPLETED	0	0	36	31

Reasons for withdrawal

Reasons for withdrawal
Measure	Troriluzole - Randomization Phase Participants received Troriluzole 140 milligrams (mg) capsules orally once daily (QD) for 8 weeks.	Placebo - Randomization Phase Participants received matching placebo capsules orally QD for 8 weeks.	Troriluzole/Troriluzole - Open Label Extension (OLE) Phase Participants 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 Phase Participants 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.
Randomization Phase (8 Weeks) Adverse Event	5	0	0	0
Randomization Phase (8 Weeks) Withdrawal by Participant	2	1	0	0
Randomization Phase (8 Weeks) Reason Not Specified	0	1	0	0
Open Label Extension Phase (336 Weeks) Adverse Event	0	0	4	4
Open Label Extension Phase (336 Weeks) Death	0	0	0	1
Open Label Extension Phase (336 Weeks) Lost to Follow-up	0	0	3	3
Open Label Extension Phase (336 Weeks) Physician Decision	0	0	3	2
Open Label Extension Phase (336 Weeks) Withdrawal by Subject	0	0	19	18
Open Label Extension Phase (336 Weeks) Sponsor Request	0	0	4	2
Open Label Extension Phase (336 Weeks) Miscellaneous	0	0	3	1

Baseline Characteristics

Trial in Adult Participants With Spinocerebellar Ataxia (SCA)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Troriluzole - Randomization Phase n=71 Participants Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks.	Placebo - Randomization Phase n=70 Participants Placebo - Participants received matching placebo capsules orally QD for 8 weeks.	Total n=141 Participants Total of all reporting groups
Age, Continuous	52.5 years STANDARD_DEVIATION 15.08 • n=5 Participants	51.6 years STANDARD_DEVIATION 13.74 • n=7 Participants	52.1 years STANDARD_DEVIATION 14.39 • n=5 Participants
Sex: Female, Male Female	35 Participants n=5 Participants	37 Participants n=7 Participants	72 Participants n=5 Participants
Sex: Female, Male Male	36 Participants n=5 Participants	33 Participants n=7 Participants	69 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=5 Participants	6 Participants n=7 Participants	12 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	65 Participants n=5 Participants	64 Participants n=7 Participants	129 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	5 Participants n=5 Participants	11 Participants n=7 Participants	16 Participants n=5 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
Race (NIH/OMB) Black or African American	6 Participants n=5 Participants	4 Participants n=7 Participants	10 Participants n=5 Participants
Race (NIH/OMB) White	56 Participants n=5 Participants	53 Participants n=7 Participants	109 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Spinocerebellar Ataxia (SCA) Genotype SCA1	17 Participants n=5 Participants	18 Participants n=7 Participants	35 Participants n=5 Participants
Spinocerebellar Ataxia (SCA) Genotype SCA2	19 Participants n=5 Participants	18 Participants n=7 Participants	37 Participants n=5 Participants
Spinocerebellar Ataxia (SCA) Genotype SCA3	8 Participants n=5 Participants	6 Participants n=7 Participants	14 Participants n=5 Participants
Spinocerebellar Ataxia (SCA) Genotype SCA6	16 Participants n=5 Participants	15 Participants n=7 Participants	31 Participants n=5 Participants
Spinocerebellar Ataxia (SCA) Genotype SCA7	2 Participants n=5 Participants	5 Participants n=7 Participants	7 Participants n=5 Participants
Spinocerebellar Ataxia (SCA) Genotype SCA8	6 Participants n=5 Participants	6 Participants n=7 Participants	12 Participants n=5 Participants
Spinocerebellar Ataxia (SCA) Genotype SCA10	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Randomization Phase Week 8

Population: Full Analysis Set (FAS): All randomized participants with at least one dose of study drug and a baseline and post-baseline Total SARA score during the Randomization Phase. Here, number of participants analyzed signifies the number of evaluable participants with change from baseline in Total SARA score at Week 8.

Outcome measures

Outcome measures
Measure	Troriluzole - Randomization Phase n=63 Participants Participants received Troriluzole 140 mg capsules orally QD for 8 weeks.	Placebo - Randomization Phase n=68 Participants Participants received matching placebo capsules orally QD for 8 weeks.
Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8	-0.810 score on a scale Standard Deviation 1.7653	-1.059 score on a scale Standard Deviation 2.3221

SECONDARY outcome

Timeframe: From first dose of study drug to 30 days post the last dose in the Randomization Phase (Up to 12 weeks)

Population: Safety Analysis Set (Randomization Phase): All participants who received at least one dose of study drug during the Randomization Phase.

An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition, unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. An SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or suspected transmission of an infectious agent, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes. TEAEs were defined as those AEs that developed, worsened, or became serious after the first dose of study drug.

Outcome measures

Outcome measures
Measure	Troriluzole - Randomization Phase n=71 Participants Participants received Troriluzole 140 mg capsules orally QD for 8 weeks.	Placebo - Randomization Phase n=70 Participants Participants received matching placebo capsules orally QD for 8 weeks.
Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) During the Randomization Phase Deaths	0 Participants	0 Participants
Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) During the Randomization Phase SAEs	4 Participants	1 Participants
Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) During the Randomization Phase Discontinued due to AEs	3 Participants	0 Participants
Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) During the Randomization Phase TEAEs	40 Participants	35 Participants

SECONDARY outcome

Timeframe: From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 340 weeks)

Population: Safety Analysis Set (OLE Phase): All participants who received at least one dose of study drug during the OLE Phase.

Outcome measures

Outcome measures
Measure	Troriluzole - Randomization Phase n=64 Participants Participants received Troriluzole 140 mg capsules orally QD for 8 weeks.	Placebo - Randomization Phase n=67 Participants Participants received matching placebo capsules orally QD for 8 weeks.
Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase Deaths	2 Participants	1 Participants
Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase SAEs	11 Participants	9 Participants
Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase Discontinued due to AEs	9 Participants	4 Participants
Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase TEAEs	60 Participants	59 Participants

SECONDARY outcome

Timeframe: From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 348 weeks after last randomization participant was enrolled)

Population: All participants who received at least one dose of Troriluzole during the Randomization Phase or OLE Phase.

Outcome measures

Outcome measures
Measure	Troriluzole - Randomization Phase n=138 Participants Participants received Troriluzole 140 mg capsules orally QD for 8 weeks.	Placebo - Randomization Phase Participants received matching placebo capsules orally QD for 8 weeks.
Number of Participants Who Received At Least One Dose of Troriluzole in the Randomization Phase or OLE Phase With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs Deaths	3 Participants	—
Number of Participants Who Received At Least One Dose of Troriluzole in the Randomization Phase or OLE Phase With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs SAEs	23 Participants	—
Number of Participants Who Received At Least One Dose of Troriluzole in the Randomization Phase or OLE Phase With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs Discontinued due to AEs	19 Participants	—
Number of Participants Who Received At Least One Dose of Troriluzole in the Randomization Phase or OLE Phase With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs TEAEs	126 Participants	—

SECONDARY outcome

Timeframe: Randomization Phase Week 8

PGI-C is a patient self-reported global index scale that was used to rate the response of a condition to therapy. Participants rated their impression of benefit based on the following 7 categories: No change (or condition has gotten worse); Almost the same, hardly any change at all; A little better, but no noticeable change; Somewhat better, but the change has not made any real difference; Moderately better, and a slight but noticeable change; Better and a definitive improvement that has made a real and worthwhile difference; A great deal better and a considerable improvement that has made all the difference.

Outcome measures

Outcome measures
Measure	Troriluzole - Randomization Phase n=63 Participants Participants received Troriluzole 140 mg capsules orally QD for 8 weeks.	Placebo - Randomization Phase n=66 Participants Participants received matching placebo capsules orally QD for 8 weeks.
Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase Somewhat better	3 Participants	2 Participants
Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase Better	4 Participants	4 Participants
Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase Almost the same	15 Participants	15 Participants
Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase A little better	7 Participants	11 Participants
Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase Moderately better	3 Participants	7 Participants
Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase A great deal better	0 Participants	0 Participants
Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase No Change	31 Participants	27 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Randomization Baseline, Extension Phase Week 48

Outcome measures

Outcome measures
Measure	Troriluzole - Randomization Phase n=96 Participants Participants received Troriluzole 140 mg capsules orally QD for 8 weeks.	Placebo - Randomization Phase Participants received matching placebo capsules orally QD for 8 weeks.
Change From Randomization Baseline in Total Score on the SARA at Extension Phase Week 48	-0.42 score on a scale Standard Deviation 2.522	—

Adverse Events

Troriluzole - Randomization Phase

Serious events: 4 serious events

Other events: 22 other events

Deaths: 0 deaths

Placebo - Randomization Phase

Serious events: 1 serious events

Other events: 18 other events

Deaths: 0 deaths

Troriluzole/Troriluzole - OLE Phase

Serious events: 11 serious events

Other events: 48 other events

Deaths: 2 deaths

Placebo/Troriluzole - OLE Phase

Serious events: 9 serious events

Other events: 50 other events

Deaths: 1 deaths

Serious adverse events

Other adverse events

Additional Information

Chief Medical Officer

Biohaven Pharmaceuticals

Phone: 203-404-0410

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60