Ignite Creation Date:
2024-05-05 @ 11:37 AM
Last Modification Date:
2024-10-26 @ 9:11 AM
Study NCT ID:
NCT00098280
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2004-12-03
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:
A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation Randomized Multi-Center Double-Blind Placebo-Controlled Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
Status:
COMPLETED
Status Verified Date:
2006-02
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 examine the safety and effectiveness of the experimental drug eculizumab in treating patients with paroxysmal nocturnal hemoglobinuria PNH a rare disorder of red blood cells that leads to premature destruction of the cells and resulting anemia Patients may be at high risk of blood clots and may develop bone marrow failure or aplastic anemia with low white blood cell and platelet counts Eculizumab is a monoclonal antibody that may help improve the survival of red blood cells
Patients 18 years of age and older with PNH who require blood transfusions for anemia and have received at least four transfusions in the 12 months preceding evaluation for this study may be eligible to enroll Candidates are screened with a medical history physical examination and check of vital signs
Participants have an electrocardiogram EKG and blood and urine tests and are vaccinated against Neisseria meningitides a common bacteria that can cause a disabling or fatal type of meningitis They then enter an observation phase of the study with monthly visits during which they complete a questionnaire update their health status transfusion record and medication use have their vital signs checked and PNH symptoms evaluated have blood and urine tests and receive a transfusion if necessary These visits continue for up to 3 months until patients receive a qualifying transfusion that is a transfusion given as a consequence of a certain hemoglobin level with symptoms or a different level without symptoms
Patients are then randomly assigned to receive either eculizumab or a placebo salt solution with no active ingredient Both study medications are given intravenously through a vein over 30 minutes once a week for five doses and then once every 2 weeks for another 11 doses At each treatment visit study weeks 0-24 patients update their health status transfusion records and medication use have their vital signs checked and provide a blood sample At various visits they also complete a questionnaire provide a urine sample and have an EKG At the last treatment visit week 26 or the final visit for patients who end their participation before visit 18 patients have a complete physical examination in addition to the procedures listed above
Patients 18 years of age and older with PNH who require blood transfusions for anemia and have received at least four transfusions in the 12 months preceding evaluation for this study may be eligible to enroll Candidates are screened with a medical history physical examination and check of vital signs
Participants have an electrocardiogram EKG and blood and urine tests and are vaccinated against Neisseria meningitides a common bacteria that can cause a disabling or fatal type of meningitis They then enter an observation phase of the study with monthly visits during which they complete a questionnaire update their health status transfusion record and medication use have their vital signs checked and PNH symptoms evaluated have blood and urine tests and receive a transfusion if necessary These visits continue for up to 3 months until patients receive a qualifying transfusion that is a transfusion given as a consequence of a certain hemoglobin level with symptoms or a different level without symptoms
Patients are then randomly assigned to receive either eculizumab or a placebo salt solution with no active ingredient Both study medications are given intravenously through a vein over 30 minutes once a week for five doses and then once every 2 weeks for another 11 doses At each treatment visit study weeks 0-24 patients update their health status transfusion records and medication use have their vital signs checked and provide a blood sample At various visits they also complete a questionnaire provide a urine sample and have an EKG At the last treatment visit week 26 or the final visit for patients who end their participation before visit 18 patients have a complete physical examination in addition to the procedures listed above
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 the 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 is a humanized monoclonal antibody that like CD59 inhibits terminal complement
This study is a randomized double-blind placebo controlled multi-center study of eculizumab or placebo administered intravenously to approximately 75 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 is a humanized monoclonal antibody that like CD59 inhibits terminal complement
This study is a randomized double-blind placebo controlled multi-center study of eculizumab or placebo administered intravenously to approximately 75 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-0048 | None | None | None |