CompletedPhase 2

Complement Inhibition Using Eculizumab to Overcome Platelet Transfusion Refractoriness in Patients With Severe Thrombocytopenia

United States10 participantsStarted 2014-11-21

Plain-language 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.

Who can participate

Age range

18 Years – 75 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

* INCLUSION CRITERIA: * Ages 18-75 years inclusive. * Ability to comprehend the investigational nature of the study and provide informed consent. * Thrombocytopenia (due to congenital causes, bone marrow failure, hematologic malignancies, and treatment related), defined as \<10k/uL without bleeding or \<30K/uL with evidence of life threatening bleeding (intracranial hemorrhage, GI bleeding, pulmonary hemorrhage, uncontrolled epistaxis, hematuria). * Diagnosed with immune platelet refractoriness, characterized by all of the following: * Lack of adequate post-transfusion platelet count increment, defined by, CCI \<7500/ul at 10-60 min, and CCI \<5000/ul at 18-24 hrs (in those who had a CCI at 10-60 min greater than or equal to 5000/microl)after at least 2 consecutive transfusions. * Presence of anti-HLA class A and/or B antibody. EXCLUSION CRITERIA: * Active meningococcal infection. * Severe psychiatric illness. Mental deficiency sufficiently severe as to make making informed consent impossible. * Positive pregnancy test for women of childbearing age within 1 week. * HIV positive test within 3 months * Paroxysmal Nocturnal Hemoglobinuria (PNH) disease with evidence of intravascular hemolysis. * Presence ITP/autoimmune thrombocytopenia * Immune platelet refractoriness responsive to treatment with IVIG RE-ENROLLMENT CRITERIA: * Meet response criteria to the initial treatment or second treatment * Meets all initial inclusion/exclusion criteria

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Subjects With Sustained Platelet Transfusion Responsiveness

Timeframe: 24 hours

Trial details

NCT IDNCT02298933

SponsorNational Heart, Lung, and Blood Institute (NHLBI)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2017-08-08

Results submitted2018-12-12

View on ClinicalTrials.gov More Thrombocytopenia trials