Ignite Creation Date:
2025-12-25 @ 2:44 AM
Ignite Modification Date:
2026-02-22 @ 6:45 PM
Study NCT ID:
NCT02298933
Status:
COMPLETED
Last Update Posted:
2019-01-03
First Post:
2014-11-21
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Complement Inhibition Using Eculizumab to Overcome Platelet Transfusion Refractoriness in Patients With Severe Thrombocytopenia
Sponsor:
National Heart, Lung, and Blood Institute (NHLBI)
Organization:
Study Overview
Official Title:
Complement Inhibition Using Eculizumab to Overcome Platelet Transfusion Refractoriness in Patients With Severe Thrombocytopenia
Status:
COMPLETED
Status Verified Date:
2018-01-18
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:
Background:
\- Platelets are tiny cells in the blood that help stop bleeding. Thrombocytopenia happens when people do not have enough normal platelets. Getting a transfusion of another person s platelets can help stop too much bleeding. But because these cells are from other people, the body may reject them,putting them at risk for serious bleeding complications. This conditions is called alloimmune platelet refractoriness . There are evidence that in many patients, platelet counts fail to increase after a platelet transfusion because the transfused platelets are destroyed by the body s defence soldier, called complement . Researchers want to see if a drug, that inhibits complement, can help increase platelet levels and reduce bleeding
Objectives:
\- To see if eculizumab increases platelet levels more after a transfusion. To see if it reduces the chance of bleeding too much.
Eligibility:
\- Adults 18-75 years old who have thrombocytopenia and alloimmune platelet refractoriness.
Design:
* Participants will be screened with medical history, physical exam, and blood tests.
* Participants will have the procedures listed below. They can have them while they are in the hospital. Or they can go to the outpatient clinic for them. Each visit may take up to 3 hours.
* Participants will get a meningitis vaccine if needed. Then they will get the study drug as an infusion.
* Participants will have a platelet transfusion. Their blood will be drawn every 24 hours until the platelet count is less than 10,000 per 1 microliter of blood.
* They will take antibiotics for 14 days.
* Participants will have a checkup and blood drawn twice a week for 2 weeks. They will get more transfusions if needed.
\- Platelets are tiny cells in the blood that help stop bleeding. Thrombocytopenia happens when people do not have enough normal platelets. Getting a transfusion of another person s platelets can help stop too much bleeding. But because these cells are from other people, the body may reject them,putting them at risk for serious bleeding complications. This conditions is called alloimmune platelet refractoriness . There are evidence that in many patients, platelet counts fail to increase after a platelet transfusion because the transfused platelets are destroyed by the body s defence soldier, called complement . Researchers want to see if a drug, that inhibits complement, can help increase platelet levels and reduce bleeding
Objectives:
\- To see if eculizumab increases platelet levels more after a transfusion. To see if it reduces the chance of bleeding too much.
Eligibility:
\- Adults 18-75 years old who have thrombocytopenia and alloimmune platelet refractoriness.
Design:
* Participants will be screened with medical history, physical exam, and blood tests.
* Participants will have the procedures listed below. They can have them while they are in the hospital. Or they can go to the outpatient clinic for them. Each visit may take up to 3 hours.
* Participants will get a meningitis vaccine if needed. Then they will get the study drug as an infusion.
* Participants will have a platelet transfusion. Their blood will be drawn every 24 hours until the platelet count is less than 10,000 per 1 microliter of blood.
* They will take antibiotics for 14 days.
* Participants will have a checkup and blood drawn twice a week for 2 weeks. They will get more transfusions if needed.
Detailed Description:
Platelet transfusion can be a life-saving procedure in preventing or treating serious bleeding in patients who have low and/or dysfunctional platelets. Treatment of blood cancer and other blood diseases as well as bone marrow transplantation is not possible without platelet transfusion support. Unfortunately, 20-60% of chronically transfused patients will stop responding to these transfusions putting them at risk for serious bleeding complications. Data support the concept that in many patients, platelet counts fail to increase after a platelet transfusion because the transfused platelets are destroyed by the body s complement. In order to overcome this problem, we will inhibit complement activity with the medication eculizumab that specifically binds and suppresses complement. We hypothesize that when we treat patients who have platelet refractoriness with eculizumab, the platelet counts will increase to higher numbers after platelet transfusions, decreasing the risk of bleeding complications associated with having a low platelet count.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 15-H-0015 | None | None | View |