Ignite Creation Date:
2024-05-05 @ 11:48 AM
Last Modification Date:
2024-10-26 @ 9:13 AM
Study NCT ID:
NCT00130000
Status:
COMPLETED
Last Update Posted:
2017-07-02
First Post:
2005-08-11
Brief Title:
Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
SHEPHERD Safety in Hemolytic PNH Patients Treated With Eculizumab A Multi-Center Open-Label Research Design
Status:
COMPLETED
Status Verified Date:
2007-06-25
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will evaluate the safety and effectiveness of the experimental drug eculizumab for treating paroxysmal nocturnal hemoglobinuria PNH a disorder that can cause premature destruction of red blood cells PNH may result in anemia requiring blood transfusions Patients may be at high risk of life-threatening blood clots in their veins and may have urine discoloration stomach pain difficulty swallowing tiredness and poor quality of life Men may have problems getting or maintaining an erection Eculizumab is a monoclonal antibody that may improve the survival of red blood cells in patients with PNH
Patients 18 years of age and older who have been diagnosed with PNH for more than 6 months who have active disease and who require blood transfusions may be eligible for this study Each candidate is screened with a physical examination electrocardiogram blood and urine tests and a questionnaire for information on how PNH affects the patient physically socially emotionally and functionally
Participants receive infusions of eculizumab through a needle in a vein once a week for five doses and then every two weeks for another 24 doses All patients are vaccinated against Neisseria meningitides a bacteria that can cause symptoms possibly including life-threatening meningitis in susceptible people including people who take eculizumab
At every treatment visit patients update their health status transfusion record and medication use review their laboratory results from the preceding visit have vital signs measured and provide a blood sample for laboratory tests At selected visits they also provide a urine sample have a repeat electrocardiogram and complete a questionnaire At the final treatment visit participants have a complete physical examination in addition to the routine procedures
Patients 18 years of age and older who have been diagnosed with PNH for more than 6 months who have active disease and who require blood transfusions may be eligible for this study Each candidate is screened with a physical examination electrocardiogram blood and urine tests and a questionnaire for information on how PNH affects the patient physically socially emotionally and functionally
Participants receive infusions of eculizumab through a needle in a vein once a week for five doses and then every two weeks for another 24 doses All patients are vaccinated against Neisseria meningitides a bacteria that can cause symptoms possibly including life-threatening meningitis in susceptible people including people who take eculizumab
At every treatment visit patients update their health status transfusion record and medication use review their laboratory results from the preceding visit have vital signs measured and provide a blood sample for laboratory tests At selected visits they also provide a urine sample have a repeat electrocardiogram and complete a questionnaire At the final treatment visit participants have a complete physical examination in addition to the routine procedures
Detailed Description:
Paroxysmal nocturnal hemoglobinuria PNH is an acquired clonal disorder of the hematopoietic stem cell characterized by intravascular hemolysis hemoglobinuria anemia and thrombosis The clinical features of PNH result from the lack of one or more of GPI-linked proteins that serve to protect cells from autologous complement mediated attack Two such proteins CD55 decay accelerating factor and CD59 reactive lysis inhibitor have been shown to be absent from PNH erythrocytes and platelets as well as other cell types
Evidence strongly suggests that lack of the terminal complement inhibitor CD59 is responsible for the sensitivity of PNH erythrocytes and platelets to the effects of autologous complement Since the pathogenesis of PNH is due to the inability of PNH red cells and platelets to inhibit the activation of terminal complement it is logical to hypothesize that a terminal complement inhibitor could effectively stop the intravascular hemolysis obviate or lessen the need for transfusions and possibly decrease the propensity of life threatening thrombosis Eculizumab h5G11-mAb is a humanized monoclonal antibody that like CD59 inhibits terminal complement
This study is an open label multi-center study of eculizumab administered intravenously for 52 weeks to approximately 85 PNH patients The study is designed to evaluate the safety of eculizumab in transfusion dependent patients with paroxysmal nocturnal hemoglobinuria PNH and to determine if the administration of this terminal complement inhibitor could provide a safe and effective substitute for CD59 function
Evidence strongly suggests that lack of the terminal complement inhibitor CD59 is responsible for the sensitivity of PNH erythrocytes and platelets to the effects of autologous complement Since the pathogenesis of PNH is due to the inability of PNH red cells and platelets to inhibit the activation of terminal complement it is logical to hypothesize that a terminal complement inhibitor could effectively stop the intravascular hemolysis obviate or lessen the need for transfusions and possibly decrease the propensity of life threatening thrombosis Eculizumab h5G11-mAb is a humanized monoclonal antibody that like CD59 inhibits terminal complement
This study is an open label multi-center study of eculizumab administered intravenously for 52 weeks to approximately 85 PNH patients The study is designed to evaluate the safety of eculizumab in transfusion dependent patients with paroxysmal nocturnal hemoglobinuria PNH and to determine if the administration of this terminal complement inhibitor could provide a safe and effective substitute for CD59 function
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 05-H-0217 | None | None | None |