TerminatedPhase 2

Using Fostamatinib to Treat Post-Hematopoietic Stem Cell Transplant Immune-mediated Cytopenias

Stopped: Clinical trial was halted prematurely due to low enrollment

United States1 participantsStarted 2023-03-10

Plain-language summary

Background: People who have a blood stem cell transplant can sometimes develop cytopenia. This means that their levels of one or more types of blood cell, such as the red cells or platelets, are lower than they should be. This can occur because a person s immune system might attack these cells after a stem cell transplant. Cytopenia can lead to anemia, severe bleeding, infections, and other problems. Treatments are needed to help keep blood cell levels stable after blood stem cell transplant. Objective: To test a study drug (fostamatinib) in people who have cytopenia after a blood stem cell transplant. Eligibility: People aged 18 to 75 years who have cytopenia after a blood stem cell transplant. Design: Participants will be screened. They will have a physical exam. They will have blood, urine, and stool tests. Fostamatinib is an oral tablet taken by mouth. Participants will take the pills 2 times a day for 12 weeks. Participants will have a medical assessment every 2 weeks; their vital signs will be checked, and they will have blood and stool tests. Participants must come to the NIH clinic for these visits in weeks 4 and 12. Other visits may be done by telephone or telehealth; the blood and stool tests can be sent to the researchers from a local lab. After 4 weeks, some participants may begin taking a higher dose of the drug. Participants will return for a final medical assessment 2 weeks after they finish taking the drug. Participants who complete this study and show evidence that fostamatinib has increased their blood cell counts may enroll in an extension study to continue taking fostamatinib.

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 * Female patients of reproductive potential agree to avoid pregnancy through abstinence or the use two forms of highly effective birth control during and for 1 month after the last study treatment and agree not to donate eggs during this time * male patients of reproductive potential agree to avoid pregnancy of a partner through abstinence or the use two forms of highly effective birth control during and for 1 month after the last study treatment and agree not to donate sperm during this time. * Diagnosis of an immune mediated cytopenia (anemia and/or thrombocytopenia) in a patient that either: * Failed or relapsed after at least one line of therapy including steroids, IVIG, TPO mimetics, rituximab, azathioprine, cyclophosphamide, cyclosporine, tacrolimus, danazol, vincristine, ESA or splenectomy * Or remains transfusion dependent (\>=1 transfusion(s)/2 weeks) * Or is steroid dependent * Subjects are \>=60 days post-allogeneic transplant with: * Thrombocytopenia, defined as average platelets count \<30 x 10\^9/L for 3 consecutive available readings at least 2 weeks apart, after other cell lines have engrafted, with no counts \>40 x 10\^9/L unless from rescue transfusions. Subjects failed at least one line of therapy outlined above with a clinical diagnosis of immune mediated thrombocytopenia. * Anemia, transfusion dependen…

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 Who Were Able to Maintain Hematologic Recovery

Timeframe: Baseline, Week 2, Week 4, Week 6, Week 8, Week 10

Trial details

NCT IDNCT05502783

SponsorNational Heart, Lung, and Blood Institute (NHLBI)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2023-06-16

Results submitted2025-10-28

View on ClinicalTrials.gov More Immune Mediated Anemia trials