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| Name | Class |
|---|---|
| CMIC Co, Ltd. Japan | INDUSTRY |
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The objective of this study was to assess the long-term safety and efficacy of eculizumab in hemolytic PNH patients who completed the 4-week screening and 12-week treatment period of the C07-001 study. In addition, pharmacokinetic and pharmacodynamic assessments of eculizumab were conducted.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eculizumab | Experimental | Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eculizumab | Drug | Each vial contains 30 mL of 10 mg/mL eculizumab; dose of 900 mg intravenous every 14 days. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Lactate Dehydrogenase | 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in FACIT-Fatigue Scale Total Score | The FACIT-Fatigue scale, Version 4.0, 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. Patients 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23934275 | Result | Kanakura Y, Ohyashiki K, Shichishima T, Okamoto S, Ando K, Ninomiya H, Kawaguchi T, Nakao S, Nakakuma H, Nishimura J, Kinoshita T, Bedrosian CL, Ozawa K, Omine M. Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial. Int J Hematol. 2013 Oct;98(4):406-16. doi: 10.1007/s12185-013-1404-y. Epub 2013 Aug 11. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Eculizumab | Treatment with eculizumab for patients with paroxysmal nocturnal hemoglobinuria who have successfully completed the C07-001 protocol (NCT01192399) Eculizumab: 900 mg intravenous every 14 days. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Eculizumab | Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol 900 mg intravenous every 14 days. |
| 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 Lactate Dehydrogenase | Posted | Mean | Standard Error | Units/Liter | 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) |
|
|
From baseline (after first dose) to end of study (1 year).
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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 | Eculizumab | Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol 900 mg intravenous every 14 days. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. | Alexion Pharmaceuticals, Inc. | clinicaltrials@alexion.com |
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| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| C481642 | eculizumab |
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| 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) |
| Change From Baseline in PNH Red Blood Cell (RBC) Count | 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) |
| Change From Baseline in Number of Units of Packed RBCs Transfused | Baseline is defined as the number of units transfused in 3 months prior to baseline | 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) |
| Change From Baseline in Plasma Free Hemoglobin | 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
|
|
| Secondary | Change From Baseline in FACIT-Fatigue Scale Total Score | The FACIT-Fatigue scale, Version 4.0, 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. Patients 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. | Posted | Mean | Standard Error | units on a scale | 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) |
|
|
|
| Secondary | Change From Baseline in PNH Red Blood Cell (RBC) Count | Posted | Mean | Standard Error | cellsx10^12/L | 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) |
|
|
|
| Secondary | Change From Baseline in Number of Units of Packed RBCs Transfused | Baseline is defined as the number of units transfused in 3 months prior to baseline | Posted | Mean | Standard Error | units | 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) |
|
|
|
| Secondary | Change From Baseline in Plasma Free Hemoglobin | Posted | Mean | Standard Error | mg/dL | 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) |
|
|
|
| 0 |
| 27 |
| 12 |
| 27 |
| 26 |
| 27 |
| Haemolysis | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Colitis | Gastrointestinal disorders | Non-systematic Assessment |
|
| Pyrexia | General disorders | Non-systematic Assessment |
|
| Cholelithiasis | Hepatobiliary disorders | Non-systematic Assessment |
|
| Jaundice cholestatic | Hepatobiliary disorders | Non-systematic Assessment |
|
| Pneumonia | Infections and infestations | Non-systematic Assessment |
|
| Bronchitis | Infections and infestations | Non-systematic Assessment |
|
| Cellulitis | Infections and infestations | Non-systematic Assessment |
|
| Gastroenteritis viral | Infections and infestations | Non-systematic Assessment |
|
| Herpes virus infection | Infections and infestations | Non-systematic Assessment |
|
| Sepsis | Infections and infestations | Non-systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | Non-systematic Assessment |
|
| Animal bite | Injury, poisoning and procedural complications | Non-systematic Assessment |
|
| Subdural haematoma | Injury, poisoning and procedural complications | Non-systematic Assessment |
|
| Acute kidney injury | Renal and urinary disorders | Non-systematic Assessment |
|
| Skin disorder | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Conjunctival haemorrhage | Eye disorders | Non-systematic Assessment |
|
| Cataract | Eye disorders | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | Non-systematic Assessment |
|
| Abdominal discomfort | Gastrointestinal disorders | Non-systematic Assessment |
|
| Dental caries | Gastrointestinal disorders | Non-systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | Non-systematic Assessment |
|
| Enterocolitis | Gastrointestinal disorders | Non-systematic Assessment |
|
| Mouth ulceration | Gastrointestinal disorders | Non-systematic Assessment |
|
| Pyrexia | General disorders | Non-systematic Assessment |
|
| Hyperbilirubinaemia | Hepatobiliary disorders | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | Non-systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | Non-systematic Assessment |
|
| Gastroenteritis | Infections and infestations | Non-systematic Assessment |
|
| Pharyngitis | Infections and infestations | Non-systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | Non-systematic Assessment |
|
| Laceration | Injury, poisoning and procedural complications | Non-systematic Assessment |
|
| Blood alkaline phosphatase increased | Investigations | Non-systematic Assessment |
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| Blood bilirubin increased | Investigations | Non-systematic Assessment |
|
| Diabetes mellitus | Metabolism and nutrition disorders | Non-systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Headache | Nervous system disorders | Non-systematic Assessment |
|
| Upper respiratory tract inflammation | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
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| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |