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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-000665-79 | EudraCT Number | ||
| U1111-1196-0653 | Other Identifier | UTN |
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| Name | Class |
|---|---|
| Achillion, a wholly owned subsidiary of Alexion | INDUSTRY |
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The purpose of this study is to evaluate the long-term safety and efficacy of ACH-0144471 in participants with paroxysmal nocturnal hemoglobinuria (PNH) who have demonstrated clinical benefit from ACH-0144471 in Study ACH471-100. This study is designed to include up to 12 participants.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ACH-0144471 | Experimental | All participants will receive ACH-0144471 during the treatment period. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ACH-0144471 | Drug | ACH-0144471 will be administered to all participants enrolled in the study. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in LDH Level at Week 25 | Change from Baseline = Serum LDH levels at Week 25 - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100. | Baseline, Week 25 |
| Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25 | Change from Baseline = Hgb levels at Week 25 - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100. | Baseline, Week 25 |
| Change From Baseline in Reticulocyte Counts at Week 25 | Change from Baseline = reticulocyte count at Week 25 - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100. | Baseline, Week 25 |
| Number of RBC Units Transfused | Baseline up to Week 169 | |
| Number of RBC Transfusion Instances | Baseline up to Week 169 | |
| Change From Baseline in PNH Clone Size at Week 25 | The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 25 - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100. | Baseline, Week 25 |
| Change From Baseline in AP Complement Functional Activity at Week 25 | Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at Week 25 - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in LDH Level at Weeks 49 and 169 | Change from Baseline = Serum LDH levels at specified postbaseline visit - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100. | Baseline, Weeks 49 and 169 |
| Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Avellino | Italy | ||||
| Clinical Trial Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41124654 | Derived | Kulasekararaj A, Browett P, Risitano AM, Patriquin CJ, Yenerel MN, Marceau D, Sahin F, Algarra JL, Ogawa M, Yu J, Cross NB, Notaro R, Lee JW, Brodsky RA. Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria. Blood Adv. 2026 Feb 10;10(3):540-554. doi: 10.1182/bloodadvances.2025017731. |
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Participants who demonstrated a clinical benefit from danicopan in the primary Study ACH471-100 (NCT03053102), were eligible for long-term treatment with danicopan in this extension study ACH471-103.
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| ID | Title | Description |
|---|---|---|
| FG000 | Danicopan | Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams [mg] 3 times daily [TID]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Full analysis set included all enrolled and treated participants.
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| ID | Title | Description |
|---|---|---|
| BG000 | Danicopan | Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams [mg] 3 times daily [TID]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in LDH Level at Week 25 | Change from Baseline = Serum LDH levels at Week 25 - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the post baseline assessments for LDH level. | Posted | Mean | Standard Deviation | U/L | Baseline, Week 25 |
|
Baseline up to 4.5 years
Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
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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 | Danicopan | Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams [mg] 3 times daily [TID]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cystitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dysphagia | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. | Alexion Pharmaceuticals, Inc. | 855-752-2356 | clinicaltrials@alexion.com |
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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 4, 2021 | Dec 21, 2022 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 23, 2021 | Dec 21, 2022 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| D006456 | Hemoglobinuria |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| C000718467 | danicopan |
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| Baseline, Week 25 |
| Change From Baseline in Free Hgb at Week 25 | Change from Baseline = free Hgb at Week 25 - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100. | Baseline, Week 25 |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation | An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Baseline up to 4.5 years |
Change from Baseline = Hgb levels at specified postbaseline visit - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100. |
| Baseline, Weeks 49 and 169 |
| Change From Baseline in Reticulocyte Counts at Weeks 49 and 169 | Change from Baseline = reticulocyte count at specified postbaseline visit - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100. | Baseline, Weeks 49 and 169 |
| Change From Baseline in PNH Clone Size at Weeks 49 and 73 | The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at specified postbaseline visit - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100. | Baseline, Weeks 49 and 73 |
| Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145 | Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at specified postbaseline visit - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100. | Baseline, Weeks 49 and 145 |
| Change From Baseline in Free Hgb at Weeks 49 and 169 | Change from Baseline = free Hgb at specified postbaseline visit - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100. | Baseline, Weeks 49 and 169 |
| Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153 | The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life. | Baseline, Weeks 21, 41, and 153 |
| Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153 | EORTC-QLQ-C30 is comprised of 30 questions. First 28 questions used 4-point scale (1=not at all,2=a little,3=quite a bit,4=very much) for evaluating 5 functional scales (physical, role, emotional, cognitive, social), 3 symptom scales (fatigue, nausea/vomiting, pain) & other single items. For each item, high score = high level of symptomatology/problem. Last 2 questions represented participant's assessment of overall health (global health status) and quality of life, coded on 7-point scale (1=very poor to 7=excellent). Answers were converted into grading scale, with total score between 0 and 100. A high score represented a favourable outcome with a best quality of life for participant. | Baseline, Weeks 21, 41, and 153 |
| Naples |
| Italy |
| Clinical Trial Site | Auckland | New Zealand |
| Clinical Trial Site | Seoul | South Korea |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Lactate Dehydrogenase (LDH) Level | Mean | Standard Deviation | units (U)/liter (L) |
|
| Hemoglobin (Hgb) Level in the Absence of Red Blood Cell (RBC) Transfusion | Mean | Standard Deviation | grams (g)/liter (L) |
|
| Reticulocyte Counts | Mean | Standard Deviation | 10^12 cells/L |
|
| Paroxysmal Nocturnal Hemoglobinuria (PNH) Clone Size | Here, 'number analyzed' signifies participants evaluable for this baseline measure. | Mean | Standard Deviation | percentage of the total cell population |
|
| Free Hgb | Mean | Standard Deviation | mg/deciliter (dL) |
|
| Alternative Pathway (AP) Complement Functional Activity | Serum AP functional activity was measured by the Wieslab functional immunoassay method. | Mean | Standard Deviation | percentage of activity |
|
|
|
| Primary | Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25 | Change from Baseline = Hgb levels at Week 25 - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Hgb level. | Posted | Mean | Standard Deviation | g/L | Baseline, Week 25 |
|
|
|
| Primary | Change From Baseline in Reticulocyte Counts at Week 25 | Change from Baseline = reticulocyte count at Week 25 - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for reticulocyte count. | Posted | Mean | Standard Deviation | 10^12 cells/L | Baseline, Week 25 |
|
|
|
| Primary | Number of RBC Units Transfused | Full analysis set included all enrolled and treated participants. | Posted | Mean | Standard Deviation | RBC units | Baseline up to Week 169 |
|
|
|
| Primary | Number of RBC Transfusion Instances | Full analysis set included all enrolled and treated participants. | Posted | Mean | Standard Deviation | RBC transfusion instances | Baseline up to Week 169 |
|
|
|
| Primary | Change From Baseline in PNH Clone Size at Week 25 | The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 25 - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for PNH clone size. | Posted | Mean | Standard Deviation | percentage of the total cell population | Baseline, Week 25 |
|
|
|
| Primary | Change From Baseline in AP Complement Functional Activity at Week 25 | Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at Week 25 - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Serum AP functional activity. | Posted | Mean | Standard Deviation | percentage of activity | Baseline, Week 25 |
|
|
|
| Primary | Change From Baseline in Free Hgb at Week 25 | Change from Baseline = free Hgb at Week 25 - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for free Hgb. | Posted | Mean | Standard Deviation | mg/dL | Baseline, Week 25 |
|
|
|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation | An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Safety analysis set included all enrolled participants who received at least 1 dose of danicopan. | Posted | Number | participants | Baseline up to 4.5 years |
|
|
|
| Secondary | Change From Baseline in LDH Level at Weeks 49 and 169 | Change from Baseline = Serum LDH levels at specified postbaseline visit - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for LDH level. 'Number analyzed' = participants evaluable at specified timepoint. | Posted | Mean | Standard Deviation | U/L | Baseline, Weeks 49 and 169 |
|
|
|
| Secondary | Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169 | Change from Baseline = Hgb levels at specified postbaseline visit - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Hgb level. 'Number analyzed' = participants evaluable at specified timepoint. | Posted | Mean | Standard Deviation | g/L | Baseline, Weeks 49 and 169 |
|
|
|
| Secondary | Change From Baseline in Reticulocyte Counts at Weeks 49 and 169 | Change from Baseline = reticulocyte count at specified postbaseline visit - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for reticulocyte count. 'Number analyzed' = participants evaluable at specified timepoint. | Posted | Mean | Standard Deviation | 10^12 cells/L | Baseline, Weeks 49 and 169 |
|
|
|
| Secondary | Change From Baseline in PNH Clone Size at Weeks 49 and 73 | The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at specified postbaseline visit - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for PNH clone size. 'Number analyzed' = participants evaluable at specified timepoint. | Posted | Mean | Standard Deviation | percentage of the total cell population | Baseline, Weeks 49 and 73 |
|
|
|
| Secondary | Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145 | Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at specified postbaseline visit - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Serum AP functional activity. 'Number analyzed' = participants evaluable at specified timepoint. | Posted | Mean | Standard Deviation | percentage of activity | Baseline, Weeks 49 and 145 |
|
|
|
| Secondary | Change From Baseline in Free Hgb at Weeks 49 and 169 | Change from Baseline = free Hgb at specified postbaseline visit - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for free Hgb. 'Number analyzed' = participants evaluable at specified timepoint. | Posted | Mean | Standard Deviation | mg/dL | Baseline, Weeks 49 and 169 |
|
|
|
| Secondary | Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153 | The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for FACIT-Fatigue scale score. 'Number analyzed' = participants evaluable at specified timepoint. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Weeks 21, 41, and 153 |
|
|
|
| Secondary | Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153 | EORTC-QLQ-C30 is comprised of 30 questions. First 28 questions used 4-point scale (1=not at all,2=a little,3=quite a bit,4=very much) for evaluating 5 functional scales (physical, role, emotional, cognitive, social), 3 symptom scales (fatigue, nausea/vomiting, pain) & other single items. For each item, high score = high level of symptomatology/problem. Last 2 questions represented participant's assessment of overall health (global health status) and quality of life, coded on 7-point scale (1=very poor to 7=excellent). Answers were converted into grading scale, with total score between 0 and 100. A high score represented a favourable outcome with a best quality of life for participant. | Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for EORTC-QLQ-C30 (Global Health Status/Qol) score. 'Number analyzed' = participants evaluable at specified timepoint. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Weeks 21, 41, and 153 |
|
|
|
| 0 |
| 8 |
| 3 |
| 8 |
| 8 |
| 8 |
| Gastroenteritis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Infectious mononucleosis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Haemolysis | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Non-cardiac chest pain | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Chest discomfort | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Influenza like illness | General disorders | MedDRA 23.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Cellulitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Herpes simplex | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Lethargy | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Haemoglobinuria | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
|
| Chromaturia | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
|
| Haematuria | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
|
| Paroxysmal nocturnal haemoglobinuria | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
|
| Accidental overdose | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
|
| Intentional overdose | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
|
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
|
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
|
| Haemolysis | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Limb discomfort | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
| Dry throat | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Hypothyroidism | Endocrine disorders | MedDRA 23.0 | Systematic Assessment |
|
| Conjunctival hyperaemia | Eye disorders | MedDRA 23.0 | Systematic Assessment |
|
| Uterine leiomyoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
|
| Hand dermatitis | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
Not provided
Not provided
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
| D011507 | Proteinuria |
| D014555 | Urination Disorders |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D020924 | Urological Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| Title | Measurements |
|---|
|
| TEAE Grade 4 |
|
| AE leading to discontinuation |
|
|
|
|
|
|
|
|
| Change at Week 153 |
|
|
|
| Change at Week 153 |
|
|