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As a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. The planned duration was 152 weeks. After termination, the actual mean duration of treatment was approx. 28 weeks (range approx. 2 to 71 weeks).
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A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial.
The planned duration of the open-label trial was 152 weeks, but the trial was terminated early as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. Therefore, the actual mean duration of open-label treatment was approximately 28 weeks (range approximately 2 to 71 weeks).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arimoclomol | Experimental | 248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Arimoclomol | Drug | 2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) Over the Open-label Treatment Period | Adverse event (AE) data were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No participant was treated for 76 weeks. Participants with on-treatment TEAEs are reported. An on-treatment TEAE is any TEAE in the on-treatment period defined as the time from first dose of IMP until 14 days since the last preceding administration of IMP (either before a temporary IMP interruption with duration >14 days or the last dose at the end of trial). A participant may have several on-treatment periods separated by interruption intervals. | From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks |
| Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (1) | Standard hematology parameters. White blood cell differential count for basophils, eosinophils, leukocytes, lymphocytes, monocytes, and neutrophils, and platelet count. | Week 76 |
| Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (2) | Standard hematology parameters. Percentage of leukocytes were determined for basophils, eosinophils, lymphocytes, monocytes, and neutrophils | Week 76 |
| Mean and Change From Baseline in Clinical Safety Laboratory Tests - Erythrocytes | Standard hematology parameter. | Week 76 |
| Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematocrit |
| Measure | Description | Time Frame |
|---|---|---|
| Change in ALS Functional Rating Scale - Revised (ALSFRS-R) From Baseline to Week 76 | The ALSFRS-R is an ordinal rating scale used to determine subjects' subjective assessment of their capability and independence with 12 functional activities ('speech', 'salivation', 'swallowing', handwriting', 'cutting food and handling utensils', 'dressing and hygiene', 'turning in bed and adjusting bed clothes', 'walking', 'dyspnoea', 'orthopnoea' and 'respiratory insufficiency'). Each activity is rated on a 5-point scale (from 0 [no ability] to 4 [normal]), giving a maximal ALSFRS-R score of 48. A lower score corresponds to a lower capability and independence. |
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Inclusion Criteria:
Exclusion Criteria:
Known or suspected allergy or intolerance to the IMP (Arimoclomol or constituents)
Exposure to any other investigational treatment, advanced therapy medicinal product or use of any other prohibited concomitant medications
Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants until 3 months after last dose. Pre-menopausal women must have a negative pregnancy test prior to dosing with trial medication.
Any of the following medically significant conditions:
Any serious adverse event or moderate/severe adverse event from the ORARIALS-01 trial which is ongoing at the time of transitioning to ORARIALS-02 and assessed as probably related to IMP
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| Name | Affiliation | Role |
|---|---|---|
| Michael Benatar, MD PhD | University of Miami | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center | Phoenix | Arizona | 85013 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Arimoclomol (Open-label) | 248 mg arimoclomol base 3 times daily (planned duration 152 weeks; actual duration after early termination of the trial approx. 28 weeks [range 2-71 weeks]). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 8, 2020 | May 12, 2023 |
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Non-randomized open label
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Standard hematology parameter.
| Week 76 |
| Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hemoglobin | Standard hematology parameter. | Week 76 |
| Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (1) | Standard clinical chemistry parameters. Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, and Lactate Dehydrogenase. | Week 76 |
| Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (2) | Standard clinical chemistry parameters. Calcium, Calcium Corrected, Cholesterol, Glucose, HDL Cholesterol, LDL Cholesterol, Potassium, Sodium, Triglycerides, and Urea Nitrogen. | Week 76 |
| Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (3) | Standard clinical chemistry parameters. Bilirubin, Creatinine, Direct Bilirubin, and Indirect Bilirubin. | Week 76 |
| Mean and Change From Baseline in Clinical Safety Laboratory Tests - Albumin and Protein | Standard clinical chemistry parameters. | Week 76 |
| Mean and Change From Baseline in Clinical Safety Laboratory Tests - Cystatin C | Standard clinical chemistry parameter. | Week 76 |
| Mean and Change From Baseline in Vital Signs - Blood Pressure | Standard vital signs. Systolic and diastolic blood pressure. | Week 76 |
| Mean and Change From Baseline in Vital Signs - Pulse Rate | Standard vital signs measurement. | Week 76 |
| Mean and Change From Baseline in Vital Signs - Respiratory Rate | Standard vital signs measurement. | Week 76 |
| Mean and Change From Baseline in Vital Signs - Temperature | Standard vital signs measurement. | Week 76 |
| Number of Participants With Potentially Clinically Significant Abnormalities in Clinical Safety Laboratory Tests and Vital Signs Over the Open-label Treatment Period | Clinical safety laboratory data and vital signs were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No patient was treated for 76 weeks. | From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks |
| Columbia-Suicide Severity Rating Scale (C-SSRS) Over the Open-label Treatment Period | The C-SSRS is a detailed questionnaire assessing both suicidal behavior and suicidal ideation through a series of simple, plain-language questions administered as an interview by a qualified investigator or delegate. | From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks |
| Week 76 |
| Change in Percentage (%) Predicted Slow Vital Capacity (SVC) From Baseline to Week 76 (for Subjects Who Did Not Meet the Survival Endpoint in the ORARIALS-01 Trial) | Slow vital capacity (SVC) measures the volume that can be exhaled from a full inhalation after exhaling to a maximum as slowly as possible. Predicted SVC was derived per European Community of Coal and Steel (ECCS) reference equations:
| 76 weeks |
| UC Irvine Health ALS and Neuromuscular Center | Orange | California | 92868 | United States |
| University of Miami | Miami | Florida | 33136 | United States |
| Hospital for Special Surgery | New York | New York | 10021 | United States |
| Providence Brain & Spine Institute | Portland | Oregon | 97213 | United States |
| University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center | Philadelphia | Pennsylvania | 19107 | United States |
| University of Virginia Health System | Charlottesville | Virginia | 22908 | United States |
| Catholic University Leuven | Leuven | Belgium |
| London Health Sciences Centre | London | Ontario | N6A 5A5 | Canada |
| Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac | Montpellier | 34295 | France |
| Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422 | Paris | 75013 | France |
| Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen | Berlin | 13353 | Germany |
| Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie | Hanover | 30625 | Germany |
| Universitaetsklinikum Ulm - Klinik fuer Neurologie | Ulm | 89081 | Germany |
| Instituti Clinica Scientifici Maugeri | Milan | 20138 | Italy |
| Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino | Torino | 10126 | Italy |
| University Medical Center Utrecht | Utrecht | 3584CX | Netherlands |
| Centrum Medyczne NeuroProtect | Warsaw | 01-684 | Poland |
| Citi Clinic | Warsaw | 02-473 | Poland |
| Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11 | Barcelona | 08035 | Spain |
| Hospital Carlos III - Hospital Universitario La Paz, ALS Unit | Madrid | 28046 | Spain |
| Umeå University Hospital | Umeå | 90737 | Sweden |
| Leonard Wolfson Experimental Neurology Centre | London | WC1N 3BG | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Arimoclomol (Open-label) | 248 mg arimoclomol base 3 times daily |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Number | participants |
| |||||||||||||||||||||||
| ALS Functional Rating Scale - Revised (ALSFRS-R) | The ALSFRS-R is an ordinal rating scale used to determine subjects' subjective assessment of their capability and independence with 12 functional activities ('speech', 'salivation', 'swallowing', handwriting', 'cutting food and handling utensils', 'dressing and hygiene', 'turning in bed and adjusting bed clothes', 'walking', 'dyspnoea', 'orthopnoea' and 'respiratory insufficiency'). Each activity is rated on a 5-point scale (from 0 [no ability] to 4 [normal]), giving a maximal ALSFRS-R score of 48. A lower score corresponds to a lower capability and independence. | Mean | Standard Deviation | units on a scale |
|
| 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 Treatment-emergent Adverse Events (TEAEs) Over the Open-label Treatment Period | Adverse event (AE) data were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No participant was treated for 76 weeks. Participants with on-treatment TEAEs are reported. An on-treatment TEAE is any TEAE in the on-treatment period defined as the time from first dose of IMP until 14 days since the last preceding administration of IMP (either before a temporary IMP interruption with duration >14 days or the last dose at the end of trial). A participant may have several on-treatment periods separated by interruption intervals. | Safety analysis set: All enrolled patients who received at least one dose of arimoclomol. | Posted | Count of Participants | Participants | From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks |
|
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (1) | Standard hematology parameters. White blood cell differential count for basophils, eosinophils, leukocytes, lymphocytes, monocytes, and neutrophils, and platelet count. | Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | 10^9 cells/L | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (2) | Standard hematology parameters. Percentage of leukocytes were determined for basophils, eosinophils, lymphocytes, monocytes, and neutrophils | Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | percentage of leukocytes | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Clinical Safety Laboratory Tests - Erythrocytes | Standard hematology parameter. | Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | 10^12 cells/L | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematocrit | Standard hematology parameter. | Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | L of cells / L of blood | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hemoglobin | Standard hematology parameter. | Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | g/L | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (1) | Standard clinical chemistry parameters. Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, and Lactate Dehydrogenase. | Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints | Posted | Mean | Standard Deviation | U/L | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (2) | Standard clinical chemistry parameters. Calcium, Calcium Corrected, Cholesterol, Glucose, HDL Cholesterol, LDL Cholesterol, Potassium, Sodium, Triglycerides, and Urea Nitrogen. | Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | mmol/L | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (3) | Standard clinical chemistry parameters. Bilirubin, Creatinine, Direct Bilirubin, and Indirect Bilirubin. | Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | micromol/L | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Clinical Safety Laboratory Tests - Albumin and Protein | Standard clinical chemistry parameters. | Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | g/L | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Clinical Safety Laboratory Tests - Cystatin C | Standard clinical chemistry parameter. | Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | mg/L | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Vital Signs - Blood Pressure | Standard vital signs. Systolic and diastolic blood pressure. | Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | mmHg | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Vital Signs - Pulse Rate | Standard vital signs measurement. | Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | beats/min | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Vital Signs - Respiratory Rate | Standard vital signs measurement. | Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | breaths/min | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean and Change From Baseline in Vital Signs - Temperature | Standard vital signs measurement. | Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | Degrees Celsius | Week 76 |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With Potentially Clinically Significant Abnormalities in Clinical Safety Laboratory Tests and Vital Signs Over the Open-label Treatment Period | Clinical safety laboratory data and vital signs were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No patient was treated for 76 weeks. | Safety analysis set: All enrolled patients that received at least one dose of arimoclomol. | Posted | Count of Participants | Participants | From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks |
|
| |||||||||||||||||||||||||||||||||||||
| Primary | Columbia-Suicide Severity Rating Scale (C-SSRS) Over the Open-label Treatment Period | The C-SSRS is a detailed questionnaire assessing both suicidal behavior and suicidal ideation through a series of simple, plain-language questions administered as an interview by a qualified investigator or delegate. | Safety analysis set: All enrolled patients who received at least one dose of arimoclomol. | Posted | Count of Participants | Participants | From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change in ALS Functional Rating Scale - Revised (ALSFRS-R) From Baseline to Week 76 | The ALSFRS-R is an ordinal rating scale used to determine subjects' subjective assessment of their capability and independence with 12 functional activities ('speech', 'salivation', 'swallowing', handwriting', 'cutting food and handling utensils', 'dressing and hygiene', 'turning in bed and adjusting bed clothes', 'walking', 'dyspnoea', 'orthopnoea' and 'respiratory insufficiency'). Each activity is rated on a 5-point scale (from 0 [no ability] to 4 [normal]), giving a maximal ALSFRS-R score of 48. A lower score corresponds to a lower capability and independence. | Data were not collected at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Week 76 |
|
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Percentage (%) Predicted Slow Vital Capacity (SVC) From Baseline to Week 76 (for Subjects Who Did Not Meet the Survival Endpoint in the ORARIALS-01 Trial) | Slow vital capacity (SVC) measures the volume that can be exhaled from a full inhalation after exhaling to a maximum as slowly as possible. Predicted SVC was derived per European Community of Coal and Steel (ECCS) reference equations:
| Data only available for 1 patient at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. | Posted | Mean | Standard Deviation | percentage of predicted SVC | 76 weeks |
|
|
Adverse event (AE) data were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months.
A treatment-emergent AE (TEAE) is defined as an AE with onset, or a pre-existing AE that worsened in severity, on or after the first dose of IMP. An on-treatment TEAE is any TEAE in the on-treatment period defined as the time from first dose of IMP until 14 days since the last preceding administration of IMP (either before a temporary IMP interruption with duration >14 days or the last dose at the end of trial). A patient may have several on-treatment periods separated by interruption intervals.
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 | Arimoclomol (Open-label) | 248 mg arimoclomol base 3 times daily | 23 | 120 | 21 | 120 | 91 | 120 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | MedDRA (21.1) | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | MedDRA (21.1) | Systematic Assessment |
| |
| Corona virus infection | Infections and infestations | MedDRA (21.1) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (21.1) | Systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Coronary artery disease | Cardiac disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA (21.1) | Systematic Assessment |
| |
| False positive investigation result | Investigations | MedDRA (21.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Urinary tract infection | Infections and infestations | MedDRA (21.1) | Systematic Assessment |
| |
| Corona virus infection | Infections and infestations | MedDRA (21.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (21.1) | Systematic Assessment |
| |
| Cystatin C increased | Investigations | MedDRA (21.1) | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Fungal skin infection | Infections and infestations | MedDRA (21.1) | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA (21.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (21.1) | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA (21.1) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Type 2 diabetes mellitus | Metabolism and nutrition disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (21.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (21.1) | Systematic Assessment |
|
Early termination led to data for a limited number of patients. No conclusions can be drawn from the laboratory results due to data for only a few patients.
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Affairs | Zevra Denmark A/S | +1-888-289-5607 | medicalaffairs@zevra.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 30, 2021 | May 12, 2023 | SAP_001.pdf |
Not provided
| 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 |
|---|---|
| C486387 | arimoclomol |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Sweden |
|
| Belgium |
|
| United States |
|
| Poland |
|
| Italy |
|
| United Kingdom |
|
| France |
|
| Germany |
|
| Spain |
|
| Title | Measurements |
|---|---|
|
| Severe TEAEs |
|
| Treatment-related TEAEs |
|
| Probably related TEAEs |
|
| Possibly related TEAEs |
|
| Not related TEAEs |
|
| Serious TEAEs (SAEs) |
|
| Treatment-related serious TEAEs |
|
| TEAEs leading to IMP withdrawal |
|
| TEAEs leading to IMP interruption |
|
|
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Erythrocytes, Week 76 |
| |||||
| Erythrocytes, change from baseline to Week 76 |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Hematocrit, Week 76 |
| |||||
| Hematocrit, change from baseline to Week 76 |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Hemoglobin, Week 76 |
| |||||
| Hemoglobin, change from baseline to Week 76 |
|
|
|
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Albumin, Week 76 |
| |||||
| Albumin, change from baseline to Week 76 |
| |||||
| Protein, Week 76 |
| |||||
| Protein, change from baseline to Week 76 |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Cystatin C, Week 76 |
| |||||
| Cystatin C, change from baseline to Week 76 |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Systolic Blood Pressure, Week 76 |
| |||||
| Systolic Blood Pressure, change from baseline to Week 76 |
| |||||
| Diastolic Blood Pressure, Week 76 |
| |||||
| Diastolic Blood Pressure, change from baseline to Week 76 |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Pulse Rate, Week 76 |
| |||||
| Pulse Rate, change from baseline to Week 76 |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Respiratory Rate, Week 76 |
| |||||
| Respiratory Rate, change from baseline to Week 76 |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Temperature, Week 76 |
| |||||
| Temperature, change from baseline to Week 76 |
|
|
|
|