CompletedPhase 2

Extended Dosing With Eltrombopag for Severe Aplastic Anemia

United States40 participantsStarted 2013-06-28

Plain-language summary

Background: \- Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve blood counts in these patients. However, researchers do not know how long the drug can and should be taken for this type of anemia. Objectives: \- To look at whether 6 months of treatment with eltrombopag can improve patient s blood counts. Eligibility: \- Individuals at least 2 years of age who are taking eltrombopag for severe aplastic anemia. Design: * Participants will take eltrombopag by mouth once a day for 6 months. * Blood samples will be collected every 2 weeks for the first 6 months. Bone marrow samples will be collected at 3 and 6 months. These samples will look at the effects of the study drug on the marrow. * Participants will continue to take the study drug for as long as it is effective and if the side effects are not severe.

Who can participate

Age range

2 Years – 100 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: * Previous diagnosis of refractory severe aplastic anemia and following at least one treatment course of immunosuppression with a regimen containing antithymocyte globulin, alemtuzumab or cyclophosphamide. * One or more of the following three clinically-significant cytopenias: platelet count less than or equal to 30,000/micro L or platelet-transfusion-dependence (requiring at least 4 platelet transfusions in the 8 weeks prior to study entry); neutrophil count less than 500/micro L; hemoglobin less than 9.0 g/dL or red cell transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior to study entry) * Age greater than or equal to 2 years old * Weight \> 12 kg EXCLUSION CRITERIA: * Infection not adequately responding to appropriate therapy * Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study entry. * Creatinine \> 2.5 mg/dL * Direct Bilirubin \> 2.0 mg/dL * SGOT or SGPT \>5 times the upper limit of normal * Hypersensitivity to eltrombopag or its components\<TAB\> * Female subjects who are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential * Unable to understand the investigational nature of the study or give informed consent * Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient s ability to tolerate protocol therapy, or that…

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 Participants With Drug Response as Defined by Clinically-signficant Hematologic Improvements

Timeframe: 24 weeks

Trial details

NCT IDNCT01891994

SponsorNational Heart, Lung, and Blood Institute (NHLBI)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2017-10-16

Results submitted2018-11-14

View on ClinicalTrials.gov More Severe Aplastic Anemia (SAA) trials