Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2016-002629-13 | EudraCT Number |
Not provided
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The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS who had completed the double-blind placebo-controlled study of tirasemtiv in ALS (CY 4031).
Enrolled participants will begin dosing of tirasemtiv 125 mg twice daily (250 mg/day) for a period of 4 weeks and will titrate to their tolerated dose, the maximum dose being 250 mg twice daily (500 mg/day). This study will also compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 with those who completed treatment with placebo in CY 4031 during continued treatment of both groups with tirasemtiv during CY 4033, compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 during that study with their clinical course during continued treatment with tirasemtiv during CY 4033, and compare the clinical course of patients who completed treatment with placebo in CY 4031 during that study with their clinical course during treatment with tirasemtiv during CY 4033.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Delayed Start Treatment | Experimental | The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033. |
|
| Early Start Treatment | Experimental | The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| tirasemtiv | Drug | Oral |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events | The number of participants with adverse events was used as the measure for the long-term safety and tolerability of tirasemtiv. | From the first dose of tirasemtiv through 28 days after the last dose |
| Measure | Description | Time Frame |
|---|---|---|
| Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 24 in CY 4033 | Slow vital capacity (SVC) was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to percent predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics [eg, height, age, sex]). |
Not provided
Inclusion Criteria:
Able to comprehend and willing to sign an Informed Consent Form (ICF). If verbal consent is given, a Legal Designee of the patient must sign the ICF form
Completed participation on study drug and the Follow-Up Visit in the CY 4031 study
Male patients, who have not had a vasectomy AND confirmed zero sperm count, must agree for the duration of their participation in the study to either:
Female patients who are not post-menopausal (≥ 1 year) or sterilized, must:
Exclusion Criteria:
Not provided
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| Name | Affiliation | Role |
|---|---|---|
| MD, Cytokinetics | Cytokinetics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics | Phoenix | Arizona | 85013 | United States | ||
Eligible patients completed participation in Study CY 4031 on study drug and had completed the scheduled follow-up visit for that study.
Patients with ALS were enrolled at 69 sites in Belgium, Canada, France, Germany, Ireland, Italy, Netherlands, Portugal, Spain, the United Kingdom, and the United States. The study was conducted from 17 October 2016 (date first patient enrolled) through 26 October 2018 (date of last patient contact).
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Delayed Start Treatment | The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033. |
| FG001 | Early Start Treatment | The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 13, 2016 | Apr 20, 2021 |
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| baseline and 24 weeks |
| Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 48 in CY 4033 | Slow vital capacity was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to percent predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics [eg, height, age, sex]). | baseline and 48 weeks |
| Change From CY 4031 Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score at Week 24 | The ALSFRS-R is used to measure the progression and severity of disability in patients with ALS. The ALSFRS-R consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in the following 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function. Comparing postbaseline to baseline assessments, a negative value indicates a worsening in function. | baseline and 24 weeks |
| Change From CY 4031 Baseline in ALSFRS-R Total Score at Week 48 | The ALSFRS-R is used to measure the progression and severity of disability in patients with ALS. The ALSFRS-R consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in the following 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function. Comparing postbaseline to baseline assessments, a negative value indicates a worsening in function. | baseline and 48 weeks |
| Cedars-Sinai Medical Center |
| Los Angeles |
| California |
| 90048 |
| United States |
| University of California, Irvine | Orange | California | 92868 | United States |
| UC Davis Medical Center | Sacramento | California | 95817 | United States |
| Forbes Norris MDA/ALS Research Center | San Francisco | California | 94115 | United States |
| Stanford Hospital and Clinics | Stanford | California | 94305 | United States |
| University of Colorado Hospital Anschutz Outpatient Pavilion | Aurora | Colorado | 80045 | United States |
| Hospital for Special Care | New Britain | Connecticut | 06053 | United States |
| George Washington University Medical Center | Washington D.C. | District of Columbia | 20037 | United States |
| Mayo Clinic | Jacksonville | Florida | 32224 | United States |
| University of Miami, Miller School of Medicine | Miami | Florida | 33136 | United States |
| Carol & Frank Morsani Center for Advanced Health Care - University of South Florida | Tampa | Florida | 33512 | United States |
| The Emory Clinic | Atlanta | Georgia | 30322 | United States |
| Indiana University | Indianapolis | Indiana | 46202 | United States |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Johns Hopkins University | Baltimore | Maryland | 21287 | United States |
| University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School | Worcester | Massachusetts | 01655 | United States |
| University of Michigan Health System | Ann Arbor | Michigan | 48109 | United States |
| Henry Ford Health System | Detroit | Michigan | 48202 | United States |
| Hennepin County Medical Center | Minneapolis | Minnesota | 55415 | United States |
| St. Louis University, Department of Neurology & Psychiatry | St Louis | Missouri | 63104 | United States |
| Barnes-Jewish Hospital | St Louis | Missouri | 63110 | United States |
| Dartmouth Hitchcock Medical Center | Lebanon | New Hampshire | 03756 | United States |
| Hospital for Special Surgery | New York | New York | 10021 | United States |
| Neurological Institute | New York | New York | 10032 | United States |
| SUNY Upstate Medical University | Syracuse | New York | 13210 | United States |
| Neurosciences Institute, Neurology - Charlotte | Charlotte | North Carolina | 28207 | United States |
| Duke Neurological Disorders Clinic | Durham | North Carolina | 27705 | United States |
| Department of Neurology, Wake Forest School of Medicine | Winston-Salem | North Carolina | 27157 | United States |
| The Ohio State University Wexner Medical Center | Columbus | Ohio | 43210 | United States |
| Providence Brain and Spine Inst. ALS Center | Portland | Oregon | 97213 | United States |
| Penn State Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Temple University School of Medicine | Philadelphia | Pennsylvania | 19140 | United States |
| Vanderbilt University Medical Center - Clinical Research Center | Nashville | Tennessee | 37232 | United States |
| Texas Neurology, PA | Dallas | Texas | 75214 | United States |
| UTHSCSA - First Outpatient Research Unit | San Antonio | Texas | 78229 | United States |
| University of Virginia Health System | Charlottesville | Virginia | 22908 | United States |
| University of Washington Medical Center | Seattle | Washington | 98195 | United States |
| West Virginia University Hospitals | Morgantown | West Virginia | 26506 | United States |
| Froedtert Memorial Lutheran Hospital | Milwaukee | Wisconsin | 53226 | United States |
| UZ Leuven | Leuven | Vlaams Brabant | 3000 | Belgium |
| University of Calgary | Calgary | Alberta | T2N 1N4 | Canada |
| University of Alberta | Edmonton | Alberta | T6G 1Z1 | Canada |
| Stan Cassidy Centre for Rehabilitation | Fredericton | New Brunswick | E3B 0C7 | Canada |
| QE II Health Sciences, Nova Scotia Health Authority | Halifax | Nova Scotia | B3H 3A7 | Canada |
| McMaster University Medical Centre | Hamilton | Ontario | L8N 3Z5 | Canada |
| London Health Sciences Centre | London | Ontario | N6A 5A5 | Canada |
| Sunnybrook Health Sciences Center | Toronto | Ontario | M4N 3M5 | Canada |
| Hopital Notre-Dame/CHUM | Montreal | Quebec | H2L 4M1 | Canada |
| Montreal Neurological Institute and Hospital | Montreal | Quebec | H3A 2B4 | Canada |
| CHU de Quebec - Univerite' Laval | Québec | Quebec | G1J 1Z4 | Canada |
| Hopital Dupuytren, service de neurologie | Limoges | 87042 | France |
| Hopital Gui de chauliac | Montpellier | 34295 | France |
| CHU de Nice - Hopital Pasteur 2 | Nice | 06001 | France |
| Hopital Bretonneau | Tours | 37044 | France |
| University of Ulm, Department of Neurology | Ulm | Baden-Wurttemberg | 89081 | Germany |
| Hannover Medical School, Department of Neurology | Hanover | Lower Saxony | 30625 | Germany |
| Charite Campus Virchow-Klinikum, Department of Neurology | Berlin | 13353 | Germany |
| Clinical Research Centre Beaumont Hospital | Dublin | Dublin 9 | Ireland |
| IRCCS Istituto Auxologico Italiano - U.O. Neurologia | Milan | Lombardy | 20149 | Italy |
| Centro Clinico NEMO - Fondazione Serena Onlus, ASST Grande Ospedale Metropolitano Niguarda, Ospedale Niguarda | Milan | Lombardy | 20162 | Italy |
| Dipartimento di Neuroscienze "Rita Levi Moltalcini" A.O.U. Citta della Salute e della Scienza di Torino P.O. "Molinette" | Turin | Piedmont | 10126 | Italy |
| University Medical Center Utrecht | Utrecht | 3584 CX | Netherlands |
| Hospital Santa Maria - Centro Hospitalar Lisboa Norte | Lisbon | 1649-035 | Portugal |
| Hospital San Rafael | Madrid | 28016 | Spain |
| King's College Hospital NHS Foundation Trust | London | SE5 9RS | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
The data provided are for the Efficacy Analysis Set, which consisted of patients who completed CY 4031, were enrolled in CY 4033, received at least 1 dose of tirasemtiv, and had at least one post-baseline efficacy outcome assessment during CY 4033.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Delayed Start Treatment | The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033. |
| BG001 | Early Start Treatment | The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033. |
| 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 |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Time since ALS symptom onset at screening in parent study | Mean | Standard Deviation | months |
|
| 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 | Number of Participants With Adverse Events | The number of participants with adverse events was used as the measure for the long-term safety and tolerability of tirasemtiv. | The data are presented for the Safety Analysis Set, which consisted of all patients who enrolled and received at least 1 dose of tirasemtiv in Study CY 4033. | Posted | Count of Participants | Participants | From the first dose of tirasemtiv through 28 days after the last dose |
|
|
| |||||||||||||||||||||||||||||
| Secondary | Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 24 in CY 4033 | Slow vital capacity (SVC) was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to percent predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics [eg, height, age, sex]). | The analysis population includes patients who received at least 1 dose of tirasemtiv and had slow vital capacity results at Week 24. | Posted | Least Squares Mean | Standard Error | change in % predicted SVC | baseline and 24 weeks |
|
| |||||||||||||||||||||||||||||
| Secondary | Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 48 in CY 4033 | Slow vital capacity was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to percent predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics [eg, height, age, sex]). | The analysis population includes patients who received at least 1 dose of tirasemtiv and had slow vital capacity results at Week 48. | Posted | Least Squares Mean | Standard Error | percent predicted slow vital capacity | baseline and 48 weeks |
|
| |||||||||||||||||||||||||||||
| Secondary | Change From CY 4031 Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score at Week 24 | The ALSFRS-R is used to measure the progression and severity of disability in patients with ALS. The ALSFRS-R consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in the following 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function. Comparing postbaseline to baseline assessments, a negative value indicates a worsening in function. | The analysis population includes patients who received at least 1 dose of tirasemtiv and had an ALSFRS-R total score at Week 24. | Posted | Least Squares Mean | Standard Error | scores on a scale | baseline and 24 weeks |
| ||||||||||||||||||||||||||||||
| Secondary | Change From CY 4031 Baseline in ALSFRS-R Total Score at Week 48 | The ALSFRS-R is used to measure the progression and severity of disability in patients with ALS. The ALSFRS-R consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in the following 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function. Comparing postbaseline to baseline assessments, a negative value indicates a worsening in function. | The analysis population includes patients who received at least 1 dose of tirasemtiv and had an ALSFRS-R total score at Week 48. | Posted | Least Squares Mean | Standard Error | scores on a scale | baseline and 48 weeks |
|
Adverse events (AEs) were collected from the first administration of tirasemtiv through 28 days after the last dose of tirasemtiv.
An AE was treatment-emergent if it started after initiation of study drug dosing in CY 4033 or was present at initiation of study drug dosing in CY 4033 and subsequently worsened in severity.
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 | Delayed Start Treatment | The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033. | 21 | 115 | 32 | 115 | 111 | 115 |
| EG001 | Early Start Treatment | The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033. | 28 | 165 | 53 | 165 | 155 | 165 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Bronchial secretion retention | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Chronic respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Hypoventilation | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Increased bronchial secretion | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Lower respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Obstructive airways disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Lung infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Abscess | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Bronchitis viral | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Bronchopneumonia | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Lobar pneumonia | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Tracheobronchitis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Amyotrophic lateral sclerosis | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Ischaemic stroke | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Metabolic encephalopathy | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Pneumoperitoneum | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Traumatic fracture | Injury, poisoning and procedural complications | MedDRA (18.0) | Systematic Assessment |
| |
| Traumatic intracranial haemorrhage | Injury, poisoning and procedural complications | MedDRA (18.0) | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA (18.0) | Systematic Assessment |
| |
| Euthanasia | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Feeling abnormal | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Cardio-respiratory arrest | Cardiac disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Acute myocardial infarction | Cardiac disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Calculus urinary | Renal and urinary disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (18.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dizziness | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Feeling abnormal | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (18.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (18.0) | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA (18.0) | Systematic Assessment |
| |
| Post-traumatic pain | Injury, poisoning and procedural complications | MedDRA (18.0) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Increased upper airway secretion | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (18.0) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (18.0) | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| MD Cytokinetics | Cytokinetics, Inc. | 650-624-2929 | medicalaffairs@cytokinetics.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 24, 2017 | Apr 20, 2021 | SAP_001.pdf |
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C572767 | CK-2017357 |
Not provided
Not provided
Not provided
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| Units | Counts |
|---|
| Participants |
|
|
|