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A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.
The objective of this study was to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH. In addition, the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in Japanese patients were assessed and compared to that of non-Japanese patients who had participated earlier studies of eculizumab treatment in hemolytic PNH.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eculizumab | Experimental | Eculizumab intravenous infusions every week x 4 doses, then 900 mg 1 week later for 1 dose, then 900 mg every 2 weeks for 4 doses |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eculizumab | Biological |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Lactate Dehydrogenase | Baseline, Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Functional Assessment of Chronic Illness Therapy (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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| Name | Affiliation | Role |
|---|---|---|
| Stephen Squinto, PhD | Alexion Pharmaceuticals, Inc. | Study Director |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21222185 | 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, Valentine ME, Khursigara G, Ozawa K, Omine M. Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: the AEGIS clinical trial. Int J Hematol. 2011 Jan;93(1):36-46. doi: 10.1007/s12185-010-0748-9. Epub 2011 Jan 12. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Eculizumab | Eculizumab intravenous infusions every week x 4 doses, then 900 mg 1 week later for 1 dose, then 900 mg every 2 weeks for 4 doses |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Eculizumab | Eculizumab intravenous infusions every week x 4 doses, then 900 mg 1 week later for 1 dose, then 900 mg every 2 weeks for 4 doses |
| 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 | Baseline, Week 12 |
|
|
Adverse events were collected over the full study period (12 weeks).
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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 | 600 mg of eculizumab as intravenous (IV) infusion once a week for 4 doses, followed by 900 mg eculizumab IV infusion 1 week later for 1 dose, then 900 mg eculizumab IV infusion every 2 weeks for 4 doses. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA (11.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (11.0) | 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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| Baseline, Week 12 |
| Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count | Baseline, Week 12 |
| Number of Units of Packed Red Blood Cells (pRBCs) Transfused | Comparison of number of units of pRBCs transfused in the 12 weeks prior to the first dose of eculizumab, and between baseline and 12 weeks after the first dose of eculizumab | 12 weeks pre-treatment, baseline, 12 weeks post-treatment |
| Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC) | Baseline to Week 12 |
| Change From Baseline in Plasma Free Hemoglobin | Baseline, Week 12 |
| Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 Score (Global Health Status) | The European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) incorporates 5 functional scales (physical, role, cognitive, emotional, and social), 3 symptom scales (fatigue, pain, and nausea and vomiting), a global health status scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnoea, loss of appetite, insomnia, constipation and diarrhea) and perceived financial impact of the disease. All of the scales and single-item measures range in score from 0 to 100. For global health status, a high score represents a high quality of life. | Baseline, Week 12 |
| 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 Functional Assessment of Chronic Illness Therapy (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 | Baseline, Week 12 |
|
|
|
| Secondary | Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count | Posted | Mean | Standard Error | cells x 10^12/L | Baseline, Week 12 |
|
|
|
| Secondary | Number of Units of Packed Red Blood Cells (pRBCs) Transfused | Comparison of number of units of pRBCs transfused in the 12 weeks prior to the first dose of eculizumab, and between baseline and 12 weeks after the first dose of eculizumab | Posted | Mean | Standard Error | Units | 12 weeks pre-treatment, baseline, 12 weeks post-treatment |
|
|
|
| Secondary | Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC) | Posted | Mean | Standard Error | U/L x Day | Baseline to Week 12 |
|
|
|
| Secondary | Change From Baseline in Plasma Free Hemoglobin | Posted | Mean | Standard Error | mg/dL | Baseline, Week 12 |
|
|
|
| Secondary | Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 Score (Global Health Status) | The European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) incorporates 5 functional scales (physical, role, cognitive, emotional, and social), 3 symptom scales (fatigue, pain, and nausea and vomiting), a global health status scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnoea, loss of appetite, insomnia, constipation and diarrhea) and perceived financial impact of the disease. All of the scales and single-item measures range in score from 0 to 100. For global health status, a high score represents a high quality of life. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline, Week 12 |
|
|
|
| 0 |
| 29 |
| 1 |
| 29 |
| 28 |
| 29 |
| C-Reactive Protein Increased | Investigations | MedDRA (11.0) | Systematic Assessment |
|
| Leukopenia | Blood and lymphatic system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Tinnitis | Ear and labyrinth disorders | MedDRA (11.0) | Systematic Assessment |
|
| Conjunctival haemorrhage | Eye disorders | MedDRA (11.0) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Chest discomfort | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Blood alkaline phosphatase increased | Investigations | MedDRA (11.0) | Systematic Assessment |
|
| Blood bilirubin increased | Investigations | MedDRA (11.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Upper respiratory tract inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
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| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
| Title | Measurements |
|---|
|