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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Troriluzole - Randomization Phase | Experimental | Troriluzole - Randomization Phase: Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks. |
|
| Placebo - Randomization Phase | Placebo Comparator | Placebo - Randomization Phase: Participants received matching placebo capsules orally QD for 8 weeks. |
|
| 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. |
|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Troriluzole | Drug | Randomization Phase: Neat (i.e., drug substance without excipients); loose filled capsule. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline, Randomization Phase Week 8 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
Key Inclusion Criteria:
Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| St. Joseph's Hospital and Medical Center | Phoenix | Arizona | 85013 | United States | ||
| CNS Trial |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34115419 | Derived | 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. |
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181 participants were screened, and 141 of these participants were randomized to treatment at 18 sites in the United States.
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| ID | Title | Description |
|---|---|---|
| FG000 | Troriluzole - Randomization Phase | Participants received Troriluzole 140 milligrams (mg) capsules orally once daily (QD) for 8 weeks. |
| FG001 | Placebo - Randomization Phase | Participants received matching placebo capsules orally QD for 8 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Randomization Phase (8 Weeks) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 17, 2020 | Jul 24, 2020 |
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| Placebo | Drug | Drug: Placebo Randomization Phase: Matching placebo loose filled capsule. |
|
| Troriluzole | Drug | OLE phase: Neat capsule or formulated capsule (i.e., drug substance with excipients). |
|
| 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. | 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. | 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. | Randomization Phase Week 8 |
| Randomization Baseline, Extension Phase Week 48 |
| Long Beach |
| California |
| 90806 |
| United States |
| University of California, Los Angeles | Los Angeles | California | 90095 | United States |
| University of California, San Francisco | San Francisco | California | 94158 | United States |
| University of Colorado Denver | Denver | Colorado | 80045 | United States |
| University of Florida | Gainesville | Florida | 32611 | United States |
| University of South Florida | Tampa | Florida | 33612 | United States |
| Emory University | Atlanta | Georgia | 30329 | United States |
| Northwestern University | Chicago | Illinois | 60611 | United States |
| University of Chicago | Chicago | Illinois | 60637 | United States |
| Johns Hopkins University | Baltimore | Maryland | 21287 | United States |
| Harvard University (Massachusetts General Hospital) | Boston | Massachusetts | 02114 | United States |
| Harvard University (Beth Israel Deaconess Medical Center) | Boston | Massachusetts | 02215 | United States |
| University of Michigan | Ann Arbor | Michigan | 48105 | United States |
| Columbia University | New York | New York | 10032 | United States |
| University of Rochester Medical Center | Rochester | New York | 14642 | United States |
| University of Texas Southwestern | Dallas | Texas | 75390 | United States |
| Houston Methodist Research Center | Houston | Texas | 77030 | United States |
| FG002 | 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. |
| FG003 | 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. |
| COMPLETED | Completed the Randomization Phase |
|
| NOT COMPLETED |
|
|
| Open Label Extension Phase (336 Weeks) |
|
|
Safety Analysis Set (Randomization Phase): All participants who received at least one dose of study drug during the Randomization Phase.
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| ID | Title | Description |
|---|---|---|
| BG000 | Troriluzole - Randomization Phase | Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks. |
| BG001 | Placebo - Randomization Phase | Placebo - Participants received matching placebo capsules orally QD for 8 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Spinocerebellar Ataxia (SCA) Genotype | The Spinocerebellar Ataxia (SCA) data displayed are not grades or stages of disease, but rather the SCA genotypes, which do not correlate with severity of disease. | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | 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. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Randomization Phase Week 8 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | Safety Analysis Set (Randomization Phase): All participants who received at least one dose of study drug during the Randomization Phase. | Posted | Count of Participants | Participants | From first dose of study drug to 30 days post the last dose in the Randomization Phase (Up to 12 weeks) |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | Safety Analysis Set (OLE Phase): All participants who received at least one dose of study drug during the OLE Phase. | Posted | Count of Participants | Participants | From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 340 weeks) |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | All participants who received at least one dose of Troriluzole during the Randomization Phase or OLE Phase. | Posted | Count of Participants | Participants | 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) |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Count of Participants | Participants | Randomization Phase Week 8 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Mean | Standard Deviation | score on a scale | Randomization Baseline, Extension Phase Week 48 |
|
|
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)
Safety Analysis Set: All participants who received at least one dose of study drug during the Randomization Phase/OLE Phase
MedDRA versions used: Randomization phase: v19.0, OLE Phase: v20.0 and v27.0
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Troriluzole - Randomization Phase | Participants received Troriluzole 140 mg capsules orally QD for 8 weeks. | 0 | 71 | 4 | 71 | 22 | 71 |
| EG001 | Placebo - Randomization Phase | Participants received matching placebo capsules orally QD for 8 weeks. | 0 | 70 | 1 | 70 | 18 | 70 |
| EG002 | Troriluzole/Troriluzole - 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. | 2 | 64 | 11 | 64 | 48 | 64 |
| EG003 | 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. | 1 | 67 | 9 | 67 | 50 | 67 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Inflammation | General disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Cerebral infarction | Nervous system disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Hospitalisation | Surgical and medical procedures | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Fibula fracture | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Concussion | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Hip fracture | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Joint dislocation | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Diarrhoea haemorrhagic | Gastrointestinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Lower gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Acute myocardial infarction | Cardiac disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Adult failure to thrive | Metabolism and nutrition disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Adenocarcinoma pancreas | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Pancreatic carcinoma metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Transient global amnesia | Nervous system disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Ankle operation | Surgical and medical procedures | MedDRA (19, 20, 27) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Balance disorder | Nervous system disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (19, 20, 27) | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (19, 20, 27) | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | Biohaven Pharmaceuticals | 203-404-0410 | clinicaltrials@biohavenpharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 27, 2020 | Jul 24, 2020 | SAP_001.pdf |
| ID | Term |
|---|---|
| D020754 | Spinocerebellar Ataxias |
| ID | Term |
|---|---|
| D002524 | Cerebellar Ataxia |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D013132 | Spinocerebellar Degenerations |
| D013118 | Spinal Cord Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D001259 | Ataxia |
| D020820 | Dyskinesias |
| D009461 | Neurologic Manifestations |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| Death |
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| Lost to Follow-up |
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| Physician Decision |
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| Withdrawal by Subject |
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| Sponsor Request |
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| Miscellaneous |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| SCA2 |
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| SCA3 |
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| SCA6 |
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| SCA7 |
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| SCA8 |
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| SCA10 |
|
Participants received matching placebo capsules orally QD for 8 weeks.
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