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, Spinocerebellar Ataxia Genotype Type 8, Spinocerebellar Ataxia Genotype Type 10
Conditions
Keywords
Spinocerebellar Ataxia, SCA
Brief 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).
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.
Interventions
DRUGTroriluzole
Randomization Phase: Neat (i.e., drug substance without excipients); loose filled capsule.
DRUGPlacebo
Drug: Placebo Randomization Phase: Matching placebo loose filled capsule.
Sponsors
Biohaven Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * 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
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) During the Randomization Phase | From first dose of study drug to 30 days post the last dose in the Randomization Phase (Up to 12 weeks) | 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. |
| Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase | From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 340 weeks) | 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. |
| 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 | 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) | 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. |
| Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase | 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. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Change From Randomization Baseline in Total Score on the SARA at Extension Phase Week 48 | Randomization Baseline, Extension Phase Week 48 | 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. |
Countries
United States
Participant flow
Recruitment details
181 participants were screened, and 141 of these participants were randomized to treatment at 18 sites in the United States.
Participants by arm
| Arm | Count |
|---|---|
| Troriluzole - Randomization Phase Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks. | 71 |
| Placebo - Randomization Phase Placebo - Participants received matching placebo capsules orally QD for 8 weeks. | 70 |
| Total | 141 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| 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) | Miscellaneous | 0 | 0 | 3 | 1 |
| Open Label Extension Phase (336 Weeks) | Physician Decision | 0 | 0 | 3 | 2 |
| Open Label Extension Phase (336 Weeks) | Sponsor Request | 0 | 0 | 4 | 2 |
| Open Label Extension Phase (336 Weeks) | Withdrawal by Subject | 0 | 0 | 19 | 18 |
| Randomization Phase (8 Weeks) | Adverse Event | 5 | 0 | 0 | 0 |
| Randomization Phase (8 Weeks) | Reason Not Specified | 0 | 1 | 0 | 0 |
| Randomization Phase (8 Weeks) | Withdrawal by Participant | 2 | 1 | 0 | 0 |
Baseline characteristics
| Characteristic | Troriluzole - Randomization Phase | Total | Placebo - Randomization Phase |
|---|---|---|---|
| Age, Continuous | 52.5 years STANDARD_DEVIATION 15.08 | 52.1 years STANDARD_DEVIATION 14.39 | 51.6 years STANDARD_DEVIATION 13.74 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 6 Participants | 12 Participants | 6 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 65 Participants | 129 Participants | 64 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 5 Participants | 16 Participants | 11 Participants |
| Race (NIH/OMB) Black or African American | 6 Participants | 10 Participants | 4 Participants |
| Race (NIH/OMB) More than one race | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 3 Participants | 5 Participants | 2 Participants |
| Race (NIH/OMB) White | 56 Participants | 109 Participants | 53 Participants |
| Sex: Female, Male Female | 35 Participants | 72 Participants | 37 Participants |
| Sex: Female, Male Male | 36 Participants | 69 Participants | 33 Participants |
| Spinocerebellar Ataxia (SCA) Genotype SCA1 | 17 Participants | 35 Participants | 18 Participants |
| Spinocerebellar Ataxia (SCA) Genotype SCA10 | 3 Participants | 5 Participants | 2 Participants |
| Spinocerebellar Ataxia (SCA) Genotype SCA2 | 19 Participants | 37 Participants | 18 Participants |
| Spinocerebellar Ataxia (SCA) Genotype SCA3 | 8 Participants | 14 Participants | 6 Participants |
| Spinocerebellar Ataxia (SCA) Genotype SCA6 | 16 Participants | 31 Participants | 15 Participants |
| Spinocerebellar Ataxia (SCA) Genotype SCA7 | 2 Participants | 7 Participants | 5 Participants |
| Spinocerebellar Ataxia (SCA) Genotype SCA8 | 6 Participants | 12 Participants | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 71 | 0 / 70 | 2 / 64 | 1 / 67 |
| other Total, other adverse events | 22 / 71 | 18 / 70 | 48 / 64 | 50 / 67 |
| serious Total, serious adverse events | 4 / 71 | 1 / 70 | 11 / 64 | 9 / 67 |
Outcome results
Primary
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.
Time frame: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Troriluzole - Randomization Phase | 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 |
| Placebo - Randomization Phase | Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8 | -1.059 score on a scale | Standard Deviation 2.3221 |
p-value: 0.51995% CI: [-0.5, 0.9]Mixed Model with Repeated Measures
Secondary
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.
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)
Population: All participants who received at least one dose of Troriluzole during the Randomization Phase or OLE Phase.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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
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.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Troriluzole - Randomization Phase | Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase | Deaths | 2 Participants |
| Troriluzole - Randomization Phase | Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase | Discontinued due to AEs | 9 Participants |
| Troriluzole - Randomization Phase | Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase | SAEs | 11 Participants |
| Troriluzole - Randomization Phase | Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase | TEAEs | 60 Participants |
| Placebo - Randomization Phase | Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase | TEAEs | 59 Participants |
| Placebo - Randomization Phase | Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase | Deaths | 1 Participants |
| Placebo - Randomization Phase | Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase | SAEs | 9 Participants |
| Placebo - Randomization Phase | Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase | Discontinued due to AEs | 4 Participants |
Secondary
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.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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 |
| Placebo - Randomization Phase | 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 | 35 Participants |
| Placebo - Randomization Phase | 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 |
| Placebo - Randomization Phase | 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 | 0 Participants |
| Placebo - Randomization Phase | 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 | 1 Participants |
Secondary
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.
Time frame: 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 for PGI-C index at Week 8.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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 |
| Troriluzole - Randomization Phase | 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 |
| Placebo - Randomization Phase | 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 |
| Placebo - Randomization Phase | 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 | 27 Participants |
| Placebo - Randomization Phase | 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 |
| Placebo - Randomization Phase | 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 | 11 Participants |
| Placebo - Randomization Phase | 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 | 2 Participants |
| Placebo - Randomization Phase | 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 |
| Placebo - Randomization Phase | 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 | 7 Participants |
Other Pre-specified
Change From Randomization Baseline in Total Score on the SARA at Extension Phase Week 48
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.
Time frame: Randomization Baseline, Extension Phase Week 48
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 Randomization baseline in Total SARA score at Week 48.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Troriluzole - Randomization Phase | 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 |