Ignite Creation Date:
2024-05-05 @ 11:25 AM
Last Modification Date:
2024-10-26 @ 9:07 AM
Study NCT ID:
NCT00039923
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2002-06-14
Brief Title:
Transfer of GPI-Linked Proteins to Transfused Patients With Paroxysmal Nocturnal Hemoglobinuria
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Pilot Study to Determine if Transfer of Gpi-Linked Proteins Occurs Following Transfusion of Red Cells to Patients With Paroxysmal Nocturnal Hemoglobinuria
Status:
COMPLETED
Status Verified Date:
2005-06
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 blood cells of patients with paroxysmal nocturnal hemoglobinuria PNH after they receive a blood transfusion to determine if certain proteins GPI-linked proteins in the transfused blood transfer to the patients blood cells GPI-linked proteins which are normally present on red cells and regulate red cell survival are absent in patients with PNH Their lack is believed to account for the premature destruction of red blood cells in these patients resulting in a low hemoglobin and hematocrit Patients may experience fatigue flank pain and other symptoms requiring treatment with blood transfusion
Patients with PNH 18 years of age or older with group A1 blood who require at least three units of red cells and who have not been transfused with group O blood within the last 3 months may be eligible for this study
Participants will come to the NIH Clinical Center for the following procedures
Interview about the severity of their anemia-related symptoms
Blood test
Blood transfusion if required Patients will be transfused with compatible group O blood The donor blood will be washed rinsed with a salt solution until it is 99 free of donor plasma Group O blood is given instead of group A1 in order to be able to distinguish the patients cells from the transfused cells
Blood samples of 3 teaspoons each will be drawn 1 day 1 week and 3 weeks after the transfusion These samples may be collected by the patients doctor locally and sent to NIH by mail
If it is found that GPI-linked proteins transfer to the patients cells the study will also examine how long the proteins remain attached and will assess whether the proteins are functional and prevent cell destruction
Patients with PNH 18 years of age or older with group A1 blood who require at least three units of red cells and who have not been transfused with group O blood within the last 3 months may be eligible for this study
Participants will come to the NIH Clinical Center for the following procedures
Interview about the severity of their anemia-related symptoms
Blood test
Blood transfusion if required Patients will be transfused with compatible group O blood The donor blood will be washed rinsed with a salt solution until it is 99 free of donor plasma Group O blood is given instead of group A1 in order to be able to distinguish the patients cells from the transfused cells
Blood samples of 3 teaspoons each will be drawn 1 day 1 week and 3 weeks after the transfusion These samples may be collected by the patients doctor locally and sent to NIH by mail
If it is found that GPI-linked proteins transfer to the patients cells the study will also examine how long the proteins remain attached and will assess whether the proteins are functional and prevent cell destruction
Detailed Description:
Paroxysmal nocturnal hemoglobinuria PNH is a clonal bone marrow disorder resulting from an acquired somatic X-linked mutation of the PIG-A gene in an hematopoietic stem cell Absence of PIG-A function in a cell prevents synthesis of the glycosylphosphatidylinositol GPI moiety which anchors many different types of proteins to the cell membrane Intravascular red cell destruction the hallmark of the disorder is caused by susceptibility of the abnormal erythrocyte to complement-mediated lysis this sensitivity is due to lack of CD59 a potent inhibitor of the late components of complement and reactive lysis In vitro studies from this laboratory have demonstrated transfer of GPI-linked proteins CD55 and CD59 from normal to deficient cells and transfer is associated with resistance to hemolysis Patients with PNH frequently require transfusion as their standard care In addition patients with all blood groups requiring transfusion will often receive compatible group O blood Group O blood is prevalent in blood bank inventories and red cell survival after transfusion is equal to that after transfusion of in group blood The purpose of this study is to examine protein transfer of GPI-linked proteins from transfused cells to deficient cells obtained from patients with PNH Patients with group A1 blood will receive compatible group O blood so that donor and recipient blood cells can be discriminated Flow cytometric studies will be performed subsequently to determine if transfer of GPI-linked protein to patients cells has occurred
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 |
|---|---|---|---|
| 02-H-0227 | None | None | None |