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This is an open-label, multi-center study designed to extend the evaluation of the safety, tolerability, and clinical effects of oral administration of KNS-760704 in patients with ALS.
Patients who complete the Part 2 Week 28 visit in study KNS-760704-CL201 and patients with ALS who were actively receiving RTPB [(6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazolediamine dihydro-chloride monohydrate] under Research IND #60,948 were eligible to participate in this study.
Eligible patients received 1 tablet of KNS-760704 (150 mg) every 12 hours (Q12H) (300 mg total daily dose) for up to 180 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| KNS-760704 300 mg/day | Experimental | Open-label KNS-760704 (150 mg Q12H) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| KNS-760704 | Drug | 150 mg Q12H KNS-760704 given orally (300 mg total daily dose) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Potentially Clinically Significant Hematology Results | Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test. | 180 weeks |
| Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities | Number of participants with potentially clinically significant liver enzyme abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter. | 180 weeks |
| Number of Participants With Potentially Clinically ECG Abnormalities | Number of participants with potentially clinically significant ECG abnormalities for the safety population are presented. Percentages are based on the number of patients in the safety population who had at least one non-missing, post-baseline value. | 180 weeks |
| Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Number of participants with potentially clinically significant vital sign abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter. | 180 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12 | The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Merit Cudkowicz, M.D., MSc | NeuroClinical Trials Unit (Massachusetts General Hospital) | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | 72205 | United States | ||
| UCLA, Dept. of Neurology - Neuromuscular/ALS Research Center |
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| ID | Title | Description |
|---|---|---|
| FG000 | 300 mg Per Day | KNS-760704 (dexpramipexole) - 150 mg BID |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| 12 weeks |
| Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24 | The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function. | 24 weeks |
| Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48 | The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function. | 48 weeks |
| Change in Upright Vital Capacity From Baseline to Week 12 | Change in Percent Predicted Upright Vital Capacity from Baseline to Week 12. A negative change indicates clinical worsening. | 12 weeks |
| Change in Upright Vital Capacity From Baseline to Week 24 | Change in Percent Predicted Upright Vital Capacity from Baseline to Week 24. A negative change indicates clinical worsening. | 24 weeks |
| Change in Upright Vital Capacity From Baseline to Week 48 | Change in Percent Predicted Upright Vital Capacity from Baseline to Week 48. A negative change indicates clinical worsening. | 48 weeks |
| Change in McGill Single-Item Scale (SIS) From Baseline to Week 12 | The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life. | 12 weeks |
| Change in McGill Single-Item Scale (SIS) From Baseline to Week 24 | The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life. | 24 weeks |
| Change in McGill Single-Item Scale (SIS) From Baseline to Week 48 | The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life. | 48 weeks |
| Number of Subjects With Feeding Tube Placed During the Study. | Number of participants who had a feeding tube placed during the study. | 144 weeks |
| Los Angeles |
| California |
| 90095 |
| United States |
| The Forbes Norris MDA/ALS Research Center | San Francisco | California | 94115 | United States |
| University of Miami Miller School of Medicine | Miami | Florida | 33136 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Johns Hopkins University School of Medicine | Baltimore | Maryland | 21228 | United States |
| Massachusettes General Hospital | Boston | Massachusetts | 02129 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Bryan LGH Medical Center East | Lincoln | Nebraska | 68506 | United States |
| Columbia University, Lou Gehrig MDA/ALS Research Center | New York | New York | 10032 | United States |
| SUNY Upstate Medical University | Syracuse | New York | 13210 | United States |
| Penn State Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Drexel University College Of Medicine | Philadelphia | Pennsylvania | 19102 | United States |
| University of Pittsburgh School of Medicine | Pittsburgh | Pennsylvania | 15213 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| University of Texas Health Sciences Center of San Antonio | San Antonio | Texas | 78229 | United States |
| University of Utah | Salt Lake City | Utah | 84132 | United States |
| University of Virginia Health System | Charlottesville | Virginia | 22908 | United States |
| University of Washington | Seattle | Washington | 98195 | United States |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Safety Population was defined as all subjects who received at least 1 dose of study treatment during the study.
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| ID | Title | Description |
|---|---|---|
| BG000 | 300 mg Per Day | KNS-760704 (dexpramipexole) - 150 mg BID |
| 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 |
| ||||||||||||||||||||||
| Age, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Number | 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 | Number of Participants With Potentially Clinically Significant Hematology Results | Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test. | Number of subjects evaluated is the number of subjects who had a baseline assessment and at least one post-baseline assessment. | Posted | Count of Participants | Participants | 180 weeks |
|
|
| ||||||||||||||||||||||||||||
| Primary | Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities | Number of participants with potentially clinically significant liver enzyme abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter. | Number of subjects evaluated is the number of subjects who had a baseline assessment and at least one post-baseline assessment. | Posted | Count of Participants | Participants | 180 weeks |
|
| |||||||||||||||||||||||||||||
| Primary | Number of Participants With Potentially Clinically ECG Abnormalities | Number of participants with potentially clinically significant ECG abnormalities for the safety population are presented. Percentages are based on the number of patients in the safety population who had at least one non-missing, post-baseline value. | Number of subjects evaluated is the number of subjects who had a baseline assessment and at least one post-baseline assessment. | Posted | Count of Participants | Participants | 180 weeks |
|
| |||||||||||||||||||||||||||||
| Primary | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Number of participants with potentially clinically significant vital sign abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter. | Number of subjects evaluated is the number of subjects in the Safety Population who had a baseline assessment and at least one post-baseline assessment. | Posted | Count of Participants | Participants | 180 weeks |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12 | The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function. | All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed. | Posted | Mean | Standard Deviation | units on a scale | 12 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24 | The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function. | All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed. | Posted | Mean | Standard Deviation | units on a scale | 24 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48 | The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function. | All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed. | Posted | Mean | Standard Deviation | units on a scale | 48 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Change in Upright Vital Capacity From Baseline to Week 12 | Change in Percent Predicted Upright Vital Capacity from Baseline to Week 12. A negative change indicates clinical worsening. | All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed. | Posted | Mean | Standard Deviation | percentage of predicted vital capacity | 12 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Change in Upright Vital Capacity From Baseline to Week 24 | Change in Percent Predicted Upright Vital Capacity from Baseline to Week 24. A negative change indicates clinical worsening. | All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed. | Posted | Mean | Standard Deviation | percentage of predicted vital capacity | 24 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Change in Upright Vital Capacity From Baseline to Week 48 | Change in Percent Predicted Upright Vital Capacity from Baseline to Week 48. A negative change indicates clinical worsening. | All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed. | Posted | Mean | Standard Deviation | percentage of predicted vital capacity | 48 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Change in McGill Single-Item Scale (SIS) From Baseline to Week 12 | The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life. | All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed. | Posted | Mean | Standard Deviation | units on a scale | 12 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Change in McGill Single-Item Scale (SIS) From Baseline to Week 24 | The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life. | All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed. | Posted | Mean | Standard Deviation | units on a scale | 24 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Change in McGill Single-Item Scale (SIS) From Baseline to Week 48 | The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life. | All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed. | Posted | Mean | Standard Deviation | units on a scale | 48 weeks |
|
| ||||||||||||||||||||||||||||
| Secondary | Number of Subjects With Feeding Tube Placed During the Study. | Number of participants who had a feeding tube placed during the study. | Randomized subjects who had at least 1 post-baseline clinical status evaluation. Subjects who had a feeding tube in place at the beginning of the study were not included in this analysis. | Posted | Number | participants | 144 weeks |
|
|
Adverse events were monitored and reported from the first dose through 30 days following the last dose.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 300 mg Per Day | KNS-760704 (dexpramipexole) - 150 mg BID | 38 | 74 | 48 | 74 | 74 | 74 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Colitis ischaemic | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Oedema | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Pyelonephritis acute | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Abscess limb | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Acinetobacter infection | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Bronchopneumonia | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Lung infection pseudomonal | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Pneumonia staphylococcal | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Tracheobronchitis | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| Face injury | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| Facial bones fracture | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| Failure to thrive | Metabolism and nutrition disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Amyotrophic lateral sclerosis | Nervous system disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Delirium | Psychiatric disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Delusion | Psychiatric disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Respiratory arrest | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Lung infiltration | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Pulmonary infarction | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Upper airway obstruction | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Wound drainage | Surgical and medical procedures | MedDRA 15.0 | Non-systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA 15.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Blood triglycerides increased | Investigations | MedDRA 15.0 | Non-systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA 15.0 | Non-systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Amyotrophic lateral sclerosis | Nervous system disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Respiratory arrest | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Decubitus ulcer | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Head of Regulatory | Knopp Biosciences | 4124881776 | greg@knoppbio.com |
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| D016472 | Motor Neuron Disease |
| D009422 | Nervous System Diseases |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| 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 |
|---|---|
| D000097662 | Dexpramipexole |
| ID | Term |
|---|---|
| D052160 | Benzothiazoles |
| D013844 | Thiazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
Not provided
Not provided
| >65 |
|
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
| Neutrophils (<1.5 x 10^9/L) |
|
|
| Neutrophils (>13.5 x 10^9/L) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|