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The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.
An open-label extension study to evaluate long-term safety of eculizumab in PNH patients who had completed the TRIUMPH (C04-001), SHEPHERD (C04-002), and X03-001 studies.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eculizumab | Experimental | 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| eculizumab | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Treatment-emergent Adverse Events | From time of consent to a maximum of 2.5 years of study treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve | From time of first infusion through 24 months of study treatment | |
| Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline | 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. 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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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University Medical Center, Division of Hematology | Stanford | California | 94305 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 17702897 | Derived | Hillmen P, Muus P, Duhrsen U, Risitano AM, Schubert J, Luzzatto L, Schrezenmeier H, Szer J, Brodsky RA, Hill A, Socie G, Bessler M, Rollins SA, Bell L, Rother RP, Young NS. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007 Dec 1;110(12):4123-8. doi: 10.1182/blood-2007-06-095646. Epub 2007 Aug 16. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Eculizumab | eculizumab: 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| From time of first infusion through 24 months of study treatment |
| Incidence of Thrombosis After Eculizumab Infusion | Thrombosis was defined as occurrence of major adverse vascular events | From time of first ever dose through last dose (up to 24 months of study treatment) |
| Hartford Hospital, Cancer Clinical Research Office |
| Hartford |
| Connecticut |
| 06102 |
| United States |
| Cleveland Clinic Florida, Department of Clinical Research | Weston | Florida | 33331 | United States |
| Indianapolis University Cancer Center | Indianapolis | Indiana | 46202 | United States |
| Johns Hopkins University Medical Center | Baltimore | Maryland | 21205 | United States |
| National Heart, Lung, and Blood Institute, National Institutes of Health | Bethesda | Maryland | 20892 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Mayo Clinic, Divison of Hematology | Rochester | Minnesota | 55902 | United States |
| Washington University, Department of Internal Medicine/Division of Hematology | St Louis | Missouri | 63110 | United States |
| NYU Clinical Cancer Center | New York | New York | 10016 | United States |
| Memorial Sloan Kettering Cancer Center | New York | New York | 10017 | United States |
| Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program | Durham | North Carolina | 27710 | United States |
| Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thombosis Program | Philadelphia | Pennsylvania | 19104 | United States |
| Royal North Shore Hospital, Haematology Department | Saint Leonards | New South Wales | 2065 | Australia |
| Princess Alexandra Hospital, Oncology Haematology Radiation Department | Woolloongabba | Queensland | 4102 | Australia |
| The Queen Elizabeth Hospital, Haematology/Oncology Department | Woodville South | South Australia | 5011 | Australia |
| The Royal Perth Hospital, Department of Haematology/Level 2 | Perth | Western Australia | 6000 | Australia |
| Royal Melbourne Hospital, Department of Clinical Haematology & Medical Oncology | Parkville | 3050 | Australia |
| Ucl St. Luc, Hematology Department | Brussels | 1200 | Belgium |
| University of Alberta, Cross Cancer Institute | Edmonton | Alberta | T6G 2B7 | Canada |
| London Regional Cancer Centre, Clinical Research Unit Room C3080 | London | Ontario | N6A4L6 | Canada |
| Hopital Saint Louis, Centre d'investigation Clinique | Paris | Cedex | 10 75475 | France |
| Universitatsklinikum Essen, Zentrum fur Innere Medizin | Essen | D-45147 | Germany |
| Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin | Hanover | D-30625 | Germany |
| Universitatskliniken des Saarlandes, Innere Medizin 1 | Homburg/Saar | D-66421 | Germany |
| Institut fur Klinische Transfusionmedizin und Immunogenetik, Abtlg. Transfusionmedizin des Univ. Ulm | Ulm | D-89081 | Germany |
| St. James's Hospital, Cancer Clinical Trial Office | Dublin | 8 | Ireland |
| Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica | Florence | 50139 | Italy |
| Ospedale San Martino, Department of Hematology | Genova | I-16132 | Italy |
| Ospedale Maggiore di Milano, Divisione di Ematologia | Milan | 35-20122 | Italy |
| Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico | Naples | 5-80131 | Italy |
| Ospedale San Bortolo, Divisione di Ematologia | Vicenza | 37-36100 | Italy |
| UMC St. Radboud, Department of Hematology | Nijmegen | GA | 6525 | Netherlands |
| Hospital Clinic i Provincial, Servicio de Hematologia | Barcelona | 08036 | Spain |
| Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia | Barcelona | 08916 | Spain |
| Hospital De La Paz, Servicio de Hematologia | Madrid | 28046 | Spain |
| Stockholm South Hospital, Division of Hematology | Stockholm | 118-83 | Sweden |
| Leeds General Infirmary, D Floor Brotherton Wing | Leeds | LS1 3EX | United Kingdom |
| St. George's Hospital, Department of Haematology | London | SW17OQT | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Eculizumab | eculizumab: 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks |
| 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 |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| 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 | Incidence of Treatment-emergent Adverse Events | Posted | Count of Participants | Participants | From time of consent to a maximum of 2.5 years of study treatment |
|
|
| ||||||||||||||||||||||||||||
| Secondary | Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve | Posted | Mean | Standard Deviation | U/L x Day | From time of first infusion through 24 months of study treatment |
|
|
| |||||||||||||||||||||||||||
| Secondary | Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline | 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. 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 Deviation | units on a scale | From time of first infusion through 24 months of study treatment |
|
| |||||||||||||||||||||||||||
| Secondary | Incidence of Thrombosis After Eculizumab Infusion | Thrombosis was defined as occurrence of major adverse vascular events | Posted | Number | Number of events per 100 patient-years | From time of first ever dose through last dose (up to 24 months of study treatment) |
|
|
From time of consent to a maximum of 2.5 years of study treatment
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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 | eculizumab: 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks | 3 | 187 | 57 | 187 | 180 | 187 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Agranulocytosis | Blood and lymphatic system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Aplastic anaemia | Blood and lymphatic system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Paroxysmal Nocturnal Haemoglobinuria | Renal and urinary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Cardiac Failure | Cardiac disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Retinal vein thrombosis | Eye disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Ascites | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Gastrointestinal hemorrhage | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Sigmoiditis | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Bile duct stone | Hepatobiliary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Hepatic cirrhosis | Hepatobiliary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Hepatic vein thrombosis | Hepatobiliary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Portal vein thrombosis | Hepatobiliary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Bacterial sepsis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Cellulitis gangrenous | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Empyema | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Endocarditis | Immune system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Enterococcal sepsis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Gallbladder abscess | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Haemophilus infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Liver abscess | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Meningococcal sepsis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Necrotising fasciitis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Penile infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Staphylococcal Infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Viral Infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Viral Upper Respiratory Tract Infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Drug Toxicity | Injury, poisoning and procedural complications | MedDRA 6.1 | Systematic Assessment |
| |
| Wrist Fracture | Injury, poisoning and procedural complications | MedDRA 6.1 | Systematic Assessment |
| |
| Haemoglobin Decreased | Investigations | MedDRA 6.1 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Intervertebral Disc Protrusion | Musculoskeletal and connective tissue disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Tenosynovitis Stenosans | Musculoskeletal and connective tissue disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 6.1 | Systematic Assessment |
| |
| Chronic Myelomonocytic Leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 6.1 | Systematic Assessment |
| |
| Malignant Melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 6.1 | Systematic Assessment |
| |
| Metastases to Bone | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 6.1 | Systematic Assessment |
| |
| Cerebral Haemorrhage | Nervous system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Convulsion | Nervous system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Metabolic Encephalopathy | Nervous system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Suicidal Ideation | Psychiatric disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Calculus Urinary | Renal and urinary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Renal Failure Acute | Renal and urinary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Renal Impairment | Renal and urinary disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Erectile Dysfunction | Reproductive system and breast disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Ovarian Cyst Ruptured | Reproductive system and breast disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Pleurisy | Respiratory, thoracic and mediastinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Phlebothrombosis | Vascular disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Thrombophlebitis | Vascular disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Thrombosis | Vascular disorders | MedDRA 6.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Influenza like Illness | General disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Oedema Peripheral | General disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Chest Pain | General disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Viral Infection | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Herpes Simplex | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 6.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 6.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Pain in Extremity | Musculoskeletal and connective tissue disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Muscle Cramp | Musculoskeletal and connective tissue disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Pharyngolaryngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 6.1 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 6.1 | Systematic Assessment |
|
Participation in this multicenter study involved a commitment to publish the data from the study in a cooperative publication prior to release of study results on an individual basis.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. | Alexion Pharmaceuticals, Inc. | clinicaltrials@alexion.com |
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| D006461 | Hemolysis |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C481642 | eculizumab |
Not provided
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| Caucasian |
|
| Other |
|
|
|
| Title |
|---|
| Denominators |
|---|
| Categories |
|---|
| Prior to first eculizumab dose |
| |||||
| From first dose of eculizumab through last dose |
|