Spinocerebellar AtaxiasPhase 2/3NCT02960893
Trial in Adult Participants With Spinocerebellar Ataxia (SCA)
A Phase 2/3 study testing Troriluzole in people with Spinocerebellar Ataxias and Spinocerebellar Ataxia Genotype Type 1, run by Biohaven Pharmaceuticals, Inc. at 18 sites in United States.
Trial summary
The primary purpose of this study was to compare the efficacy of BHV-4157 (Troriluzole) 140 milligrams (mg) once daily versus placebo after 8 weeks of treatment in participants with spinocerebellar ataxia (SCA). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Genotype Type 1, Spinocerebellar Ataxia Genotype Type 2
- What is being tested?
- Troriluzole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 141 participants
- Where
- 18 sitesUnited States
- Study dates
- 15 Dec 2016to 20 Sep 2024
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10
- Ability to ambulate 8 meters without assistance (canes and other devices allowed)
- Screening total Scale for the Assessment and Rating of Ataxia (SARA) score ≥8
- Score of ≥ 2 on the gait subsection of the SARA
- Determined by the investigator to be medically stable at baseline/randomization and must be physically able and expected to complete the trial as designed
Who cannot join
- Any medical condition other than one of the hereditary ataxias specified in the inclusion criteria that could predominantly explain or contribute significantly to the participants symptoms of ataxia
- Mini Mental State Exam (MMSE) score < 24
- SARA total score of > 30 points at screening
- Clinical history of stroke
- Active liver disease or a history of hepatic intolerance to medications that in the investigator's judgment, is medically significant
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
United States18 sites
University of Michigan
Ann Arbor, Michigan, 48105
Emory University
Atlanta, Georgia, 30329
Johns Hopkins University
Baltimore, Maryland, 21287
Harvard University (Massachusetts General Hospital)
Boston, Massachusetts, 02114
Harvard University (Beth Israel Deaconess Medical Center)
Boston, Massachusetts, 02215
Northwestern University
Chicago, Illinois, 60611
University of Chicago
Chicago, Illinois, 60637
University of Texas Southwestern
Dallas, Texas, 75390
University of Colorado Denver
Denver, Colorado, 80045
University of Florida
Gainesville, Florida, 32611
Houston Methodist Research Center
Houston, Texas, 77030
CNS Trial
Long Beach, California, 90806
University of California, Los Angeles
Los Angeles, California, 90095
Columbia University
New York, New York, 10032
St. Joseph's Hospital and Medical Center
Phoenix, Arizona, 85013
University of Rochester Medical Center
Rochester, New York, 14642
University of California, San Francisco
San Francisco, California, 94158
University of South Florida
Tampa, Florida, 33612
About this study
Official title: A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Participants With Spinocerebellar Ataxia
The primary purpose of this study was to compare the efficacy of BHV-4157 (Troriluzole) 140 milligrams (mg) once daily versus placebo after 8 weeks of treatment in participants with spinocerebellar ataxia (SCA).
Read the detailed description
The study was conducted in 2 phases: Randomization Phase (8 weeks) followed by an open-label Extension Phase (336 weeks). During the Randomization Phase, participants received either Troriluzole 140 mg or matching placebo up to 8 weeks. Participants who agreed to enter the Open-label Extension Phase continued dosing of Troriluzole 140 mg for 336 weeks. The study was subsequently amended to follow participants for a total of 336 weeks in the Open-label Extension Phase.
- Conditions
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Genotype Type 1, Spinocerebellar Ataxia Genotype Type 2, Spinocerebellar Ataxia Genotype Type 3, Spinocerebellar Ataxia Genotype Type 6, Spinocerebellar Ataxia Genotype Type 7
- Being tested
- Troriluzole, Placebo
- Keywords
- Spinocerebellar Ataxia, SCA
- Medical subject headings
- Spinocerebellar Ataxias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TroriluzoleDrug
Randomization Phase: Neat (i.e., drug substance without excipients); loose filled capsule.
PlaceboDrug
Drug: Placebo Randomization Phase: Matching placebo loose filled capsule.
TroriluzoleDrug
OLE phase: Neat capsule or formulated capsule (i.e., drug substance with excipients).
| Study group | Role | What they receive |
|---|---|---|
| Troriluzole - Randomization Phase | Experimental | Troriluzole - Randomization Phase: Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks. Receives: Drug: Troriluzole |
| Placebo - Randomization Phase | Placebo comparator | Placebo - Randomization Phase: Participants received matching placebo capsules orally QD for 8 weeks. Receives: Drug: Placebo |
| Troriluzole/Troriluzole - OLE (Open Label Extension) Phase | Experimental | 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. Receives: Drug: Troriluzole |
| Placebo/Troriluzole - OLE Phase | Experimental | 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. Receives: Drug: Placebo; Drug: Troriluzole |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at 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.
Measured: Baseline, Randomization Phase Week 8
Secondary outcomes (4)
Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) 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.
Measured: From first dose of study drug to 30 days post the last dose in the Randomization Phase (Up to 12 weeks)
Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE 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.
Measured: From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 340 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
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.
Measured: 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)
Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase
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.
Measured: Randomization Phase Week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2016 (estimated)
- Study start15 Dec 2016
- Primary completion18 Aug 2017
- Study completion20 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Spinocerebellar AtaxiasStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 141 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biohaven Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02960893
- Sponsor's protocol ID
- BHV4157-201
- Status last verified
- Sep 2025
- First submitted
- 4 Nov 2016
- Last updated
- 8 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
- Expanded access
- Available
Publications
Schmahmann JD, Pierce S, MacMore J, L'Italien GJ. Development and Validation of a Patient-Reported Outcome Measure of Ataxia. Mov Disord. 2021 Oct;36(10):2367-2377. doi: 10.1002/mds.28670. Epub 2021 Jun 11.
PubMed 34115419
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Participants With Spinocerebellar Ataxia
- ClinicalTrials.gov ID
- NCT02960893
- Acronym / sponsor ID
- BHV4157-201
- Conditions
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Genotype Type 1, Spinocerebellar Ataxia Genotype Type 2, Spinocerebellar Ataxia Genotype Type 3, Spinocerebellar Ataxia Genotype Type 6, Spinocerebellar Ataxia Genotype Type 7
- Keywords
- Spinocerebellar Ataxia, SCA
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 141 participants (actual)
- Lead sponsor
- Biohaven Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Dec 2016 (actual)
- Primary completion
- 18 Aug 2017 (actual)
- Study completion
- 20 Sep 2024 (actual)
- First posted
- 10 Nov 2016 (estimated)
- Results
- Posted 14 Aug 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary purpose of this study was to compare the efficacy of BHV-4157 (Troriluzole) 140 milligrams (mg) once daily versus placebo after 8 weeks of treatment in participants with spinocerebellar ataxia (SCA).
Detailed description
The study was conducted in 2 phases: Randomization Phase (8 weeks) followed by an open-label Extension Phase (336 weeks). During the Randomization Phase, participants received either Troriluzole 140 mg or matching placebo up to 8 weeks. Participants who agreed to enter the Open-label Extension Phase continued dosing of Troriluzole 140 mg for 336 weeks. The study was subsequently amended to follow participants for a total of 336 weeks in the Open-label Extension Phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Troriluzole - Randomization Phase | Experimental | Drug: TroriluzoleTroriluzole - Randomization Phase: Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks. |
| Placebo - Randomization Phase | Placebo comparator | Drug: PlaceboPlacebo - Randomization Phase: Participants received matching placebo capsules orally QD for 8 weeks. |
| Troriluzole/Troriluzole - OLE (Open Label Extension) Phase | Experimental | Drug: TroriluzoleParticipants 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 | Experimental | Drug: Placebo, Drug: TroriluzoleParticipants 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. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Participants with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10
- Ability to ambulate 8 meters without assistance (canes and other devices allowed)
- Screening total Scale for the Assessment and Rating of Ataxia (SARA) score ≥8
- Score of ≥ 2 on the gait subsection of the SARA
- Determined by the investigator to be medically stable at baseline/randomization and must be physically able and expected to complete the trial as designed
Key exclusion criteria (5 listed)
- Any medical condition other than one of the hereditary ataxias specified in the inclusion criteria that could predominantly explain or contribute significantly to the participants symptoms of ataxia
- Mini Mental State Exam (MMSE) score < 24
- SARA total score of > 30 points at screening
- Clinical history of stroke
- Active liver disease or a history of hepatic intolerance to medications that in the investigator's judgment, is medically significant
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TroriluzoleRandomization Phase: Neat (i.e., drug substance without excipients); loose filled capsule. | Drug | Troriluzole - Randomization Phase, Troriluzole/Troriluzole - OLE (Open Label Extension) Phase |
| PlaceboDrug: Placebo Randomization Phase: Matching placebo loose filled capsule. | Drug | Placebo - Randomization Phase, Placebo/Troriluzole - OLE Phase |
| TroriluzoleOLE phase: Neat capsule or formulated capsule (i.e., drug substance with excipients). | Drug | Placebo/Troriluzole - OLE Phase, Troriluzole/Troriluzole - OLE (Open Label Extension) Phase |
Primary outcome
Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8
Time frame: 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.
4 secondary outcomes
Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) During the Randomization Phase
Time frame: From first dose of study drug to 30 days post the last dose in the Randomization Phase (Up to 12 weeks)
Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase
Time frame: From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 340 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
Time frame: 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)
Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase
Time frame: Randomization Phase Week 8
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34115419Schmahmann JD, Pierce S, MacMore J, L'Italien GJ. Development and Validation of a Patient-Reported Outcome Measure of Ataxia. Mov Disord. 2021 Oct;36(10):2367-2377. doi: 10.1002/mds.28670. Epub 2021 Jun 11.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Troriluzole in people with Spinocerebellar Ataxias and Spinocerebellar Ataxia Genotype Type 1, run by Biohaven Pharmaceuticals, Inc. at 18 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Aug 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8 (time frame: Baseline, Randomization Phase Week 8). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02960893, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2025.