RECRUITING

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

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Conditions

Immune Mediated AnemiaImmune Mediated ThrombocytopeniaChronic GVHDAllogeneic Hematopoietic Stem Cell TransplantGraft Versus Host DiseaseSpleen Tyrosine Kinase (Syk) Inhibitor

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Phase II Study Using Fostamatinib to Treat Post-Hematopoietic Stem Cell Transplant Immune-Mediated Cytopenias

Quick Facts

Study Start:2023-03-10
Study Completion:2026-04-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05502783

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Ages 18-75 years inclusive
  2. * Ability to comprehend the investigational nature of the study and provide informed consent
  3. * 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
  4. * 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.
  5. * Diagnosis of an immune mediated cytopenia (anemia and/or thrombocytopenia) in a patient that either:
  6. * 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
  7. * Or remains transfusion dependent (\>=1 transfusion(s)/2 weeks)
  8. * Or is steroid dependent
  9. * Subjects are \>=60 days post-allogeneic transplant with:
  10. * 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.
  11. * Anemia, transfusion dependent, or defined as hemoglobin \<=9 g/dL for 3 consecutive available readings at least 2 weeks apart, after other cell lines have engrafted OR if hemoglobin 9-10 g/dL, subject must have symptomatic anemia or ongoing treatment for immune hemolytic anemia that have failed at least one line of therapy outlined above. Symptomatic anemia is defined as anemia with fatigue, weakness, shortness of breath, palpitations/fast heartbeat, lightheadedness, and/or chest pain, and these symptoms are attributed to anemia. Laboratory evaluation are recommended but not required for the diagnosis, such as a positive DAT, low haptoglobin \<lower limit of normal (LLN), indirect bilirubin \> upper limit of normal (ULN), or lactate dehydrogenase (LDH) \>ULN.
  12. * Subjects must test negative for HIV, HBV, and HCV by standard serologic tests within the previous six months
  13. * Subjects on other standard of care therapeutic regimens for GVHD or cytopenias should be on a stable dose of medication (no change \>=25%) for at least 15 days prior to enrollment.
  14. * Patients with a history of hypertension should be maintained on a stable antihypertensive regimen and with controlled blood pressure (Systolic blood pressure \< 140 mmHg and diastolic blood pressure \<90 mmHg) for at least one week prior to enrollment.
  15. * Peripheral blood or bone marrow T-cell chimerism \>=50% donor cells
  16. * Immune mediated anemia in subjects with auto or alloantibodies identified due to ABO or non-ABO mismatch transplant, or thrombocytopenia due to identified HLA/HPA antibody. Other causes of immune mediated cytopenias include clinically diagnosed (with or without serologic confirmation) idiopathic thrombocytopenic purpura or autoimmune hemolytic anemia. Subjects with cytopenias attributable to GVHD will be included. Subjects with idiopathic immune mediated cytopenias can also be included. Subjects with evidence for graft rejection per the investigator's opinion ARE NOT eligible for treatment.
  1. * Severe psychiatric illness or mental deficiency sufficient to make making informed consent impossible
  2. * Positive pregnancy test for women of childbearing age within 1 week or being actively lactating
  3. * Immune mediated cytopenia responsive to the standard of care treatment
  4. * Immune mediated cytopenia due to other autoimmune causes, such as systemic lupus erythrocytosis, chronic lymphocytic leukemia
  5. * Patients who have previously received or currently take fostamatinib post-allogeneic transplant
  6. * Non-immune mediated cytopenias. Etiologies including, but not limited to, cytopenias due to HIV infection, lymphoproliferative disorders, myelodysplasia/acute leukemia, drug-induced thrombocytopenia, thrombotic microangiopathies, acute bleeding, consumptive coagulopathy, fever, infections leading to cytopenia, medications induced cytopenias, thrombotic microangiopathies (disseminated intravascular coagulation), splenomegaly or hemophagocytic lymphohistiophagocytosis, relapse of primary disease.
  7. * Patients with neutropenia, defined as absolute neutrophil count \<=1.0 x 10\^9/L, will be excluded
  8. * Uncontrolled hypertension (systolic blood pressure \>=140mmHg or diastolic blood pressure \>=90mmHg)
  9. * ALT or AST \>=3 times the upper limit of normal, or direct bilirubin \>=2 times the upper limit of normal
  10. * Patients who have a history of medical disorders, that in the investigator's opinion, could affect the conduct of the study or the absorption, metabolism or excretion of the study drug are excluded.
  11. * Patients with lymphoma/chronic lymphocytic leukemia, hepatitis, or HIV associated with ITP/wAIHA
  12. * Patients with evidence of graft rejection (based on clinical suspicion supported by BM biopsy data and/or chimerism studies and/or MLR)
  13. * Subjects recently treated (within 30 days) with cytokine-targeting biologics (anti-TNF, IL6) or Bcell or plasma-cell depleting antibody.
  14. * Other cancers except that for which the transplant was done \< 2 years before study entry, except non-melanoma skin cancer or carcinoma in situ of the uterine cervix or breast

Contacts and Locations

Study Contact

Jamie Y Hur, D.O.
CONTACT
(301) 402-2399
jamie.hur@nih.gov

Principal Investigator

Jamie Y Hur, D.O.
PRINCIPAL_INVESTIGATOR
National Heart, Lung, and Blood Institute (NHLBI)

Study Locations (Sites)

National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
United States

Collaborators and Investigators

Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

  • Jamie Y Hur, D.O., PRINCIPAL_INVESTIGATOR, National Heart, Lung, and Blood Institute (NHLBI)

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-03-10
Study Completion Date2026-04-01

Study Record Updates

Study Start Date2023-03-10
Study Completion Date2026-04-01

Terms related to this study

Keywords Provided by Researchers

  • Allogeneic Hematopoietic Stem Cell Transplant
  • Graft Versus Host Disease
  • Spleen Tyrosine Kinase (Syk) Inhibitor

Additional Relevant MeSH Terms

  • Immune Mediated Anemia
  • Immune Mediated Thrombocytopenia
  • Chronic GVHD