Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| EUDRA CT #: 2011-006119-70 |
Not provided
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The Initial Phase 3 Study (NCTO1281189) did not meet its primary efficacy endpoint.
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| Name | Class |
|---|---|
| Biogen | INDUSTRY |
Not provided
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The purpose of the study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive, degenerative disease of motor neurons in the brain and spinal cord that leads to muscle atrophy and spasticity in limb and bulbar muscles resulting in weakness and loss of ambulation, oropharyngeal dysfunction, weight loss, and ultimately respiratory failure. The purpose of this study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dexpramipexole | Experimental | Dexpramipexole open-label |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Dexpramipexole | Drug | Oral tablet 150 mg given twice daily (BID) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Subjects Who Reported an Adverse Event | The number of subjects who reported an adverse event during the study | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
| Number of Subjects Who Experienced a Serious Adverse Event | The number of subjects enrolled who reported a serious adverse event during the study | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
| Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event | The number of subjects enrolled who discontinued the study treatment due to an adverse event during the study | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
| Number of Participants With Potentially Clinically Significant Vital Sign Results | Number of Participants with Potentially Clinically Significant Vital Sign Abnormalities. 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. | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
| Number of Participants With Potentially Clinically Significant Hematology Results | Number of Participants with Potentially Clinically Significant Hematology Results. 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. | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
| Number of Participants With Potentially Clinically Significant Blood Chemistry Results |
| Measure | Description | Time Frame |
|---|---|---|
| Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study | 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 between 0 to 48, with higher scores representing better function. Slope is calculated using a linear mixed effects model with x-axis time in months and y-axis ALSFRS-R score. Units for slope are change per month in units on the ALSFRS-R scale. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Michael Bozik, MD | Knopp Biosciences | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Barrow Neurological Institute - St. Joseph's Hospital | Phoenix | Arizona | 85013 | United States | ||
| University of California at San Francisco - Fresno |
Not provided
Not provided
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| ID | Title | Description |
|---|---|---|
| FG000 | Dexpramipexole 300 mg/Day | Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
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Number of Participants with Potentially Clinically Significant Blood Chemistry Results. 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. |
| Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
| Number of Participants With Potentially Clinically Significant ECG Results | Number of Participants with Potentially Clinically Significant ECG Results. 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. | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
| Up to maximum 226 days: approximately 32.2 weeks |
| Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study | SNIP is a test of inspiratory force (sternocleidomastoid and diaphragm) measured via a nasal cannula and is used to assess respiratory muscle weakness and to monitor changes in respiratory muscle strength over time. During the SNIP maneuver, the patient is asked to perform a strong, sharp, maximal sniff, whereby nasal pressure is measured via nasal cannula. The maximum recorded value after several attempts, with rest in between attempts, was use in the analysis. | Up to maximum 226 days: approximately 32.2 weeks |
| Death up to 6 Months | Kaplan-Meier estimate of percentage of subjects who died up to 6 months | 6 Months |
| Percentage of Participants With Death or Death Equivalent up to 6 Months | Kaplan-Meier estimate of percentage of subjects who died or had a death equivalent event (tracheostomy or permanent assisted ventilation [PAV], defined as use of noninvasive ventilation [NIV] for ≥22 hours per day for ≥10 days) up to 6 months | 6 months |
| Fresno |
| California |
| 03721 |
| United States |
| University of California, Irvine | Orange | California | 92868 | United States |
| University of California, Davis | Sacramento | California | 95817 | United States |
| California Pacific Medical Center | San Francisco | California | 94115 | United States |
| Hospital for Special Care | New Britain | Connecticut | 06053 | United States |
| Mayo Clinic - Jacksonville | Jacksonville | Florida | 32224 | United States |
| University of Miami Miller School of Medicine | Miami | Florida | 33136 | United States |
| University of South Florida Medical Center | Tampa | Florida | 33612 | United States |
| Emory University | Atlanta | Georgia | 30322 | United States |
| Northwestern University | Chicago | Illinois | 60611 | United States |
| Indiana University | Indianapolis | Indiana | 46202 | United States |
| University of Iowa | Iowa City | Iowa | 52242 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Johns Hopkins University School of Medicine | Baltimore | Maryland | 21287 | United States |
| Massachusetts General Hospital | Charlestown | Massachusetts | 02129 | United States |
| St. Mary's Health Care | Grand Rapids | Michigan | 49503 | United States |
| Hennepin County Medical Center | Minneapolis | Minnesota | 55404 | United States |
| Mayo Clinic - Rochester | Rochester | Minnesota | 55905 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Neurology Associates, P.C. | Lincoln | Nebraska | 68506 | United States |
| University of Nevada School of Medicine | Las Vegas | Nevada | 89102 | United States |
| Dartmouth-Hitchcock Medical Center | Lebanon | New Hampshire | 03756 | United States |
| Columbia University | New York | New York | 10032 | United States |
| Research Foundation of the State University of New York | Syracuse | New York | 12207 | United States |
| Carolinas Medical Center | Charlotte | North Carolina | 28207 | United States |
| Duke University Medical Center | Durham | North Carolina | 27705 | United States |
| Wake Forest University | Winston-Salem | North Carolina | 27157 | United States |
| The Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| Ohio State University | Columbus | Ohio | 43210 | United States |
| Providence ALS Center | Portland | Oregon | 97213 | United States |
| Penn State Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Drexel University College of Medicine | Philadelphia | Pennsylvania | 19102 | United States |
| ALS Center at Penn | Philadelphia | Pennsylvania | 19107 | United States |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15213 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| Texas Neurology | Dallas | Texas | 75214 | United States |
| Methodist Neurological Institute | Houston | Texas | 77030 | United States |
| University of Texas Health Sciences Center | 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 |
| Prince of Wales Hospital | Randwick | New South Wales | Australia |
| Westmead Hospital | Westmead | New South Wales | 2145 | Australia |
| Royal Brisbane and Women's Hospital | Herston | Queensland | 4029 | Australia |
| Calvary Health Care Bethlehem | Melbourne | Victoria | 3121 | Australia |
| AZ St-Lucas | Ghent | 9000 | Belgium |
| UZ Leuven | Leuven | 3000 | Belgium |
| Univ of Calgary / Foothills MC | Calgary | Alberta | T2V 1P9 | Canada |
| CHUM - Hopital Notre Dame | Montreal | Quebec | H2L 4M1 | Canada |
| Mcgill University | Montreal | Quebec | H3A 2B4 | Canada |
| London Health Sciences Centre | London | Canada |
| Sunnybrook and Women's College and Health Sciences Centre | Toronto | M4N 3M5 | Canada |
| University of British Columbia | Vancouver | Canada |
| CHRU de Lille - Hôpital Roger Salengro | Lille | 59037 | France |
| Centre Hospitalier La Timone | Marseille | France |
| CHU Gui de Chauliac | Montpellier | 34295 | France |
| CHU de Nice - Hôpital de l'Archet 1 | Nice | France |
| Hôpital La Pitié Salpétrière | Paris | 75013 | France |
| Charité - Universitätsmedizin Berlin | Berlin | Germany |
| Bergmannsheil Gmbh | Bochum | Germany |
| Medizinische Hochschule Hannover (MHH) | Hanover | Germany |
| Universitätsklinikum Jena | Jena | Germany |
| University of Ulm, RKU | Ulm | Germany |
| Beaumont Hospital | Dublin | Dublin 9 | Ireland |
| Academisch Medisch Centrum | Amsterdam | 1105 AZ | Netherlands |
| UMC St. Radboud | Nijmegen | 6525 GA | Netherlands |
| Universitair Medisch Centrum Utrecht | Utrecht | 3584 CX | Netherlands |
| Hospital Universitario de Bellvitge | Barcelona | 8907 | Spain |
| Hospital Vall d'Hebron | Barcelona | Spain |
| Hospital La Paz | Madrid | 28046 | Spain |
| Hospital Carlos III | Madrid | Spain |
| Sahlgrenska Universitetssjukhuset | Gothenburg | 41345 | Sweden |
| Karolinska Universitetssjukhuset, Solna | Stockholm | 17176 | Sweden |
| Queen Elizabeth Hospital | Birmingham | B15 2TH | United Kingdom |
| Walton Centre for Neurology & Neurosurgery | Liverpool | L9 7LJ | United Kingdom |
| Kings College Hospital NHS Foundation Trust | London | SE5 8AF | United Kingdom |
| Newcastle University Hospital - Clinical Ageing Research Unit | Newcastle | NE4 5PL | United Kingdom |
| John Radcliffe Hospital | Oxford | United Kingdom |
| Sheffield Institute for Transnational Neuroscience | Sheffield | S10 2HQ | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
This open-label extension study consisted of a Baseline Visit, a treatment period of up to 36 months, and a Safety Follow-Up Visit. Subjects who completed either Study CL211 or Study 223AS302 were eligible to participate in this study. If possible, the last visit of the lead-in study was used the Baseline. Otherwise, subjects were required to enroll within 14 days of their last visit in the lead-in study.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Dexpramipexole 150 mg BID | Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | One subject did not have age at Baseline reported. | Mean | Standard Deviation | years |
| ||||||||||||||||
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||||
| Weight (kg) | Weight was unable to be measured for many subjects due to ALS disease impairment (e.g., use of a mobility device). | Mean | Standard Deviation | kg |
| ||||||||||||||||
| Height (cm) | Mean | Standard Deviation | cm |
| |||||||||||||||||
| Body mass index (kg/m^2) | Weight, and therefore BMI, was unable to be measured for many subjects due to ALS disease impairment (e.g., use of a mobility device). | Mean | Standard Deviation | kg/m^2 |
|
| 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 Subjects Who Reported an Adverse Event | The number of subjects who reported an adverse event during the study | The safety population is defined as all subjects who received at least 1 dose of study treatment | Posted | Count of Participants | Participants | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
|
|
| ||||||||||||||||||||||||||
| Primary | Number of Subjects Who Experienced a Serious Adverse Event | The number of subjects enrolled who reported a serious adverse event during the study | The safety population is defined as all subjects who received at least 1 dose of study treatment | Posted | Count of Participants | Participants | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
|
| |||||||||||||||||||||||||||
| Primary | Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event | The number of subjects enrolled who discontinued the study treatment due to an adverse event during the study | The safety population is defined as all subjects who received at least 1 dose of study treatment | Posted | Count of Participants | Participants | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
|
| |||||||||||||||||||||||||||
| Primary | Number of Participants With Potentially Clinically Significant Vital Sign Results | Number of Participants with Potentially Clinically Significant Vital Sign Abnormalities. 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. | The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline vital sign measurement during the study. | Posted | Count of Participants | Participants | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
|
| |||||||||||||||||||||||||||
| Primary | Number of Participants With Potentially Clinically Significant Hematology Results | Number of Participants with Potentially Clinically Significant Hematology Results. 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. | The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline hematology assessment during the study. | Posted | Count of Participants | Participants | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
|
| |||||||||||||||||||||||||||
| Primary | Number of Participants With Potentially Clinically Significant Blood Chemistry Results | Number of Participants with Potentially Clinically Significant Blood Chemistry Results. 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. | The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline blood chemistry assessment during the study. | Posted | Count of Participants | Participants | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
|
| |||||||||||||||||||||||||||
| Primary | Number of Participants With Potentially Clinically Significant ECG Results | Number of Participants with Potentially Clinically Significant ECG Results. 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. | The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline ECG assessment during the study. | Posted | Count of Participants | Participants | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) |
|
| |||||||||||||||||||||||||||
| Secondary | Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study | 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 between 0 to 48, with higher scores representing better function. Slope is calculated using a linear mixed effects model with x-axis time in months and y-axis ALSFRS-R score. Units for slope are change per month in units on the ALSFRS-R scale. | Randomized subjects who had at least 1 post-baseline clinical evaluation | Posted | Least Squares Mean | Standard Error | Change per month in ALSFRS-R | Up to maximum 226 days: approximately 32.2 weeks |
|
| ||||||||||||||||||||||||||
| Secondary | Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study | SNIP is a test of inspiratory force (sternocleidomastoid and diaphragm) measured via a nasal cannula and is used to assess respiratory muscle weakness and to monitor changes in respiratory muscle strength over time. During the SNIP maneuver, the patient is asked to perform a strong, sharp, maximal sniff, whereby nasal pressure is measured via nasal cannula. The maximum recorded value after several attempts, with rest in between attempts, was use in the analysis. | Randomized subjects who had at least 1 post-baseline clinical evaluation | Posted | Least Squares Mean | Standard Error | Change per month in SNIP (in cm H20) | Up to maximum 226 days: approximately 32.2 weeks |
|
| ||||||||||||||||||||||||||
| Secondary | Death up to 6 Months | Kaplan-Meier estimate of percentage of subjects who died up to 6 months | Subjects who were randomized, received at least 1 dose of study treatment, had at least 1 available post-dosing efficacy evaluation (ALSFRS-R) or died during the study period. | Posted | Number | 95% Confidence Interval | percentage of participants | 6 Months |
|
| ||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Death or Death Equivalent up to 6 Months | Kaplan-Meier estimate of percentage of subjects who died or had a death equivalent event (tracheostomy or permanent assisted ventilation [PAV], defined as use of noninvasive ventilation [NIV] for ≥22 hours per day for ≥10 days) up to 6 months | Subjects who were randomized, received at least 1 dose of study treatment, had at least 1 available post-dosing efficacy evaluation (ALSFRS-R) or died during the study period. | Posted | Number | 95% Confidence Interval | percentage of participants | 6 months |
|
|
Adverse events were collected between the time of the first dose of study treatment and the last study visit for each enrolled subject. In this study, the maximum subject duration was a maximum 226 days: approximately 32.2 weeks.
Not provided
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 | Dexpramipexole 300 mg/Day | Dexpramipexole: Oral tablet 150 mg given twice daily up to 36 months | 73 | 616 | 152 | 616 | 215 | 616 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| LEUKOPENIA | Blood and lymphatic system disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| NEUTROPENIA | Blood and lymphatic system disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| CARDIAC ARREST | Cardiac disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| CARDIO-RESPIRATORY ARREST | Cardiac disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| STRESS CARDIOMYOPATHY | Cardiac disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| VENTRICULAR FIBRILLATION | Cardiac disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| COLITIS | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| CONSTIPATION | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| DYSPHAGIA | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| FAECALOMA | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| GASTROINTESTINAL HAEMORRHAGE | Gastrointestinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| DEATH | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| EUTHANASIA | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| GENERAL PHYSICAL HEALTH DETERIORATION | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| HAEMORRHOIDS | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| CHOLECYSTITIS | Hepatobiliary disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| BRONCHITIS | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| CATHETER SITE INFECTION | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| CLOSTRIDIUM DIFFICILE COLITIS | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| ENTEROCOCCAL INFECTION | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| ENTEROCOCCAL SEPSIS | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| ENTEROCOLITIS INFECTIOUS | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| GASTROENTERITIS | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| HERPES ZOSTER OTICUS | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| LOWER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| ORAL INFECTION | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| PNEUMONIA | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| PNEUMONIA PNEUMOCOCCAL | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| PSEUDOMONAL BACTERAEMIA | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| UROSEPSIS | Infections and infestations | MedDRA 15.0 | Non-systematic Assessment |
| |
| CAPNOTHORAX | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| CERVICAL VERTEBRAL FRACTURE | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| FALL | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| FEMUR FRACTURE | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| HIP FRACTURE | Injury, poisoning and procedural complications | MedDRA 15.0 | Non-systematic Assessment |
| |
| BLOOD GLUCOSE INCREASED | Investigations | MedDRA 15.0 | Non-systematic Assessment |
| |
| LIVER FUNCTION TEST ABNORMAL | Investigations | MedDRA 15.0 | Non-systematic Assessment |
| |
| SLEEP STUDY | Investigations | MedDRA 15.0 | Non-systematic Assessment |
| |
| WEIGHT DECREASED | Investigations | MedDRA 15.0 | Non-systematic Assessment |
| |
| HYPOKALAEMIA | Metabolism and nutrition disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| HYPONATRAEMIA | Metabolism and nutrition disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| JOINT SWELLING | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| BRONCHIAL CARCINOMA | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.0 | Non-systematic Assessment |
| |
| AMYOTROPHIC LATERAL SCLEROSIS | Nervous system disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| ANXIETY | Psychiatric disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| CONFUSIONAL STATE | Psychiatric disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| HALLUCINATION | Psychiatric disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| CALCULUS URETERIC | Renal and urinary disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| HYDRONEPHROSIS | Renal and urinary disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| URINARY RETENTION | Renal and urinary disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| ACUTE RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| ASPIRATION | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| CHRONIC RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| DYSPNOEA | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| FOREIGN BODY ASPIRATION | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| HYPOVENTILATION | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| LARYNGOSPASM | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| NASAL SEPTUM DEVIATION | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| PNEUMONIA ASPIRATION | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| PNEUMOTHORAX | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| PULMONARY EMBOLISM | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| RESPIRATORY ARREST | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| RESPIRATORY DISORDER | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| RESPIRATORY DISTRESS | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| RESPIRATORY MUSCLE WEAKNESS | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| ANGIOEDEMA | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| RASH GENERALISED | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| RESPITE CARE | Social circumstances | MedDRA 15.0 | Non-systematic Assessment |
| |
| GASTROSTOMY TUBE INSERTION | Surgical and medical procedures | MedDRA 15.0 | Non-systematic Assessment |
| |
| INTESTINAL RESECTION | Surgical and medical procedures | MedDRA 15.0 | Non-systematic Assessment |
| |
| MECHANICAL VENTILATION | Surgical and medical procedures | MedDRA 15.0 | Non-systematic Assessment |
| |
| TRACHEOSTOMY | Surgical and medical procedures | MedDRA 15.0 | Non-systematic Assessment |
| |
| DEEP VEIN THROMBOSIS | Vascular disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| SHOCK | Vascular disorders | MedDRA 15.0 | Non-systematic Assessment |
| |
| THROMBOSIS | Vascular disorders | MedDRA 15.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| URINARY TRACT INFECTION | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| CONSTIPATION | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| NAUSEA | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| OEDEMA PERIPHERAL | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| FALL | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
|
Sponsor must be consulted
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Head of Regulatory | Knopp Biosciences | 4124881776 | greg@knoppbio.com |
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| D016472 | Motor Neuron Disease |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | 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
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| >65 years |
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| missing |
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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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| Sweden |
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| Belgium |
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| United States |
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| Ireland |
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| United Kingdom |
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| Australia |
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| France |
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| Germany |
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| Spain |
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