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 range18 Years – 75 Years

SexALL

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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…

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