Immune Mediated AnemiaPhase 2NCT05502783
Using Fostamatinib to Treat Post-Hematopoietic Stem Cell Transplant Immune-mediated Cytopenias
A Phase 2 study testing fostamatinib in people with Immune Mediated Anemia and Immune Mediated Thrombocytopenia, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Immune Mediated Anemia, Immune Mediated Thrombocytopenia, Chronic GVHD
- What is being tested?
- fostamatinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Mar 2023to 16 Jun 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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 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.
- Subjects must test negative for HIV, HBV, and HCV by standard serologic tests within the previous six months
- 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.
- 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.
- Peripheral blood or bone marrow T-cell chimerism >=50% donor cells
- 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.
- Steroid dependence is defined as inability to tolerate a corticosteroid taper after demonstrating a response to an initial corticosteroid dose (typically 1-2 mg/kg/day). Patients will meet our definition of steroid dependence if their cytopenias relapse or progress before achieving a 50% decrease in the initial corticosteroid dose and/or are unable to have their steroid dose tapered to a dose of less than 20 mg/day of prednisone.
Who cannot join
- Severe psychiatric illness or mental deficiency sufficient to make making informed consent impossible
- Positive pregnancy test for women of childbearing age within 1 week or being actively lactating
- Immune mediated cytopenia responsive to the standard of care treatment
- Immune mediated cytopenia due to other autoimmune causes, such as systemic lupus erythrocytosis, chronic lymphocytic leukemia
- Patients who have previously received or currently take fostamatinib post-allogeneic transplant
- 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.
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- Patients with neutropenia, defined as absolute neutrophil count <=1.0 x 10^9/L, will be excluded
- Uncontrolled hypertension (systolic blood pressure >=140mmHg or diastolic blood pressure >=90mmHg)
- ALT or AST >=3 times the upper limit of normal, or direct bilirubin >=2 times the upper limit of normal
- 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.
- Patients with lymphoma/chronic lymphocytic leukemia, hepatitis, or HIV associated with ITP/wAIHA
- Patients with evidence of graft rejection (based on clinical suspicion supported by BM biopsy data and/or chimerism studies and/or MLR)
- Subjects recently treated (within 30 days) with cytokine-targeting biologics (anti-TNF, IL6) or Bcell or plasma-cell depleting antibody.
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: A Phase II Study Using Fostamatinib to Treat Post-Hematopoietic Stem Cell Transplant Immune-Mediated Cytopenias
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.
Read more
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.
Read the detailed description
Study Description:
This open label phase II trial is designed to evaluate the efficacy of fostamatinib in the treatment of post-transplant cytopenias as assessed by hematologic improvement in anemia and/or thrombocytopenia following a 12-week treatment course. Patients who respond to the 12-week treatment course on this single arm study are eligible and have the option to enroll on the extended access trial.
Objectives:
The primary objective is to assess efficacy of fostamatinib for stable hematologic recovery during post-hematopoietic stem cell transplant immune mediated anemia and/or thrombocytopenia.
The secondary objective is to assess efficacy of fostamatinib for clinically-relevant outcomes in post-hematopoietic stem cell transplant patients.
The exploratory objective is to evaluate changes in serologic markers that may be associated with cytopenias while on treatment to identify key elements for fostamatinib response.
Endpoints:
Primary endpoints:
The proportion of subjects with hematologic recovery that is stable, defined as improvement documented in 2 consecutive available readings at least 2 weeks apart, without recent blood product transfusion support in the past 7 days.
-Hematologic recovery is defined as:
--Hemoglobin >=10 g/dL (or at least >=2 g/dL above baseline) in subjects enrolled with posttransplant anemia. In subjects with symptomatic anemia, a hemoglobin increase of at least >=2 g/dL above baseline is required
OR
--Platelets >= 50 x 10^9/L (or at least >=20 x 10^9/L above baseline) in subjects enrolled with posttransplant thrombocytopenia
OR
--Both of the above criteria in subjects with posttransplant Evans syndrome
Secondary endpoints:
-Proportion of subjects who achieve objective hematologic recovery within the 12-week treatment course defined as:
--Hemoglobin >=9 g/dL (or at least >=1 g/dL above baseline) in subjects enrolled with anemia or at least >=1 g/dL above baseline in subjects with symptomatic anemia
OR
--Platelets >= 30 x 10^9/L (or at least >=10 x 10^9/L above baseline) in subjects enrolled with thrombocytopenia
OR
-- Either of the above criteria in subjects with Evans syndrome
- Average weekly requirement of transfused blood component or growth factor requirement (total units of PRBC or Platelets/week, total dose of growth factor/week) by week 12, compared to the week prior to the start of the study drug.
- Change in corticosteroid dose over time, measured by median daily weight-based prednisone-equivalent corticosteroid dose in a week, from week 1 to week 12
- Change in other immunosuppressant dose over time, measured by median daily dose of the immunosuppressant in a week, from week 1 to week 12
- Number of patients who achieved >=50% steroid dose reduction by week 12 compared to week 1
- Incidence and severity of cGVHD according to 2014 NIH Consensus Criteria, at baseline before the initiation of treatment, and at week 12
Exploratory endpoints:
- Absolute percentage change in B cell chimerism pre-treatment, vs weeks 4 and 12.
- cPRA change in the preformed HLA antibodies before and after treatment in subjects with thrombocytopenia or Evans syndrome
- Percent MFI change of preformed HLA antibodies before and after treatment. This applies for antibodies with an MFI >1,000.
- Anti-RBC alloantibody titer change in patients with hemolytic anemia before and after treatment
- Assessment of serial cytokines pre-treatment and weeks 4 and 12. Cytokines include CRP, IL-1, IL-2, IL-6, IFN gamma, TNF alpha, EPO and TPO
- Immunoglobulin level changes pre-treatment, and weeks 4 and 12.
- Absolute reticulocyte count, lactate dehydrogenase, haptoglobin changes pre-treatment, and at each biweekly study visit, in patients with anemia or Evans syndrome
- Change in the weekly rate and severity of bleeding according to the total score of the ITP-Bleeding Scale (IBLS), from week -1 (one week before the start of the study drug) to week 12, in subjects with thrombocytopenia or Evans syndrome
- Being tested
- fostamatinib
- Keywords
- Allogeneic Hematopoietic Stem Cell Transplant, Graft Versus Host Disease, Spleen Tyrosine Kinase (Syk) Inhibitor
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Bronchiolitis Obliterans Syndrome, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
fostamatinibDrug
Participants will receive fostamatinib 100 mg BID for 4 weeks. On the 4-week evaluation, a) if cytopenia improves (hemoglobin =10 g/dL, platelets = 50 x 109/L), patients will continue the same dose for a total of 12 weeks, b) if refractory cytopenias persist (hemoglobin < 10 g/dL, platelets < 50 x 109/L), the dose will be increased to 150 mg BID. Subjects with persistent (=2 readings, 2 weeks apart) loss of hematologic response after week 5 can increase their dose to 150 mg BID until at end of the study on week 12.
| Study group | Role | What they receive |
|---|---|---|
| Fostamatinib in Participants Post-Hematopoietic Stem Cell Transplant with Immune-Mediated Cytopenias | Experimental | Participants Post-Hematopoietic Stem Cell Transplant with Immune-Mediated Cytopenias will receive fostamatinib 100 mg BID by mouth for 4 weeks. On the 4-week evaluation, a) if cytopenia improves (hemoglobin =10 g/dL, platelets = 50 x 109/L), patients will continue the same dose for a total of 12 weeks, b) if refractory cytopenias persist (hemoglobin < 10 g/dL, platelets < 50 x 109/L), the dose will be increased to 150 mg BID. Subjects with persistent (=2 readings, 2 weeks apart) loss of hematologic response after week 5 can increase their dose to 150 mg BID until at end of the study on week 12. Receives: Drug: fostamatinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Were Able to Maintain Hematologic Recovery
Stable hematologic recovery (improvement documented in 2 consecutive available readings) without recent blood product transfusion support (in the past 48-72 hours).
Hematologic recovery is defined as:
- Hemoglobin =10 g/dL (or at least =2 g/dL above baseline) in participants enrolled with posttransplant anemia. In participants with symptomatic anemia, a hemoglobin increase of at least =2 g/dL above baseline is required OR
- Platelets = 50 x 10^9/L (or at least =20 x 10^9/L above baseline) in participants enrolled with posttransplant thrombocytopenia OR
- Both of the above criteria in participants with posttransplant Evans syndrome
Measured: Baseline, Week 2, Week 4, Week 6, Week 8, Week 10
Secondary outcomes (7)
Number of Participants Who Achieve Objective Hematologic Recovery
Objective hematologic recovery defined as:
- Hemoglobin =9 g/dL (or at least =1 g/dL above baseline) in participants enrolled with anemia or at least =1 g/dL above baseline in participants with symptomatic anemia OR
- Platelets = 30 x 10^9/L (or at least =20 x 10^9/L above baseline) in participants enrolled with thrombocytopenia OR
- Both of the above criteria in participants with Evans syndrome
Measured: Up to 10 weeks
Median Change in Requirement of Transfused Blood Component
Median change in requirement of weekly transfused blood component or growth factor injections requirement (total units of Packed Red Blood Cells or Platelets). Data from the specified time points were aggregated and summarized to calculate the median value.
Measured: Baseline, Week 2, Week 4, Week 6, Week 8, Week 10
Median Change in Requirement of Growth Factor Injections
Median change in requirement of weekly growth factor injections. Data from the specified time points were aggregated and summarized to calculate the median value.
Measured: Baseline, Week 2, Week 4, Week 6, Week 8, Week 10
Median Change in Requirement Corticosteroid Dose (Prednisone)
Median Change in requirement corticosteroid dose (Prednisone) , measured by median daily weight-based prednisone-equivalent corticosteroid dose from week 1 to week 12. Data from the specified time points were aggregated and summarized to calculate the median value.
Measured: Baseline, Week 2, Week 4, Week , Week 8, Week 10
Median Change in Other Immunosuppressant Dose
Median Change in other immunosuppressant dose, measured by median daily dose of the immunosuppressant. Data from the specified time points were aggregated and summarized to calculate the median value.
Measured: Baseline, Week 2, Week 4, Week 6, Week 8, Week 10, Week 12
Number of Participants Who Developed Mild, Moderate or Severe Chronic Graft vs Host Disease (GVHD) According to 2014 NIH Consensus Criteria
Number of Participants Who Developed Mild, Moderate or Severe Chronic Graft vs Host Disease (GVHD) according to 2014 NIH Consensus Criteria
Diagnosis is based on organ involvement and severity scoring, with criteria for skin, mouth, eyes, liver, lungs, gastrointestinal tract, joints/fascia, and genital tract, though criteria have been revised to exclude findings clearly attributable to non-GVHD causes. Severity is categorized as mild, moderate, or severe based on the number of organs involved and the specific severity score for each organ (0-3), with lungs requiring specific pulmonary function tests for scoring.
Mild: Involves 1 or 2 organs with a score of no more than 1 for each (lung score must be 0).
Moderate: Involves 3 or more organs with a score of no more than 1 for each; OR at least 1 organ (not the lung) has a score of 2; OR the lung score is 1.
Severe: Involves at least 1 organ with a score of 3; OR the lung score is 2 or 3.
Measured: Up to 10 weeks
Number of Patients Who Achieved 50% Steroid Dose Reduction
Number of patients who achieved 50% steroid dose reduction by week 12 compared to week 1
Measured: Up to Week 10
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2022
- Study start10 Mar 2023
- Primary completion16 Jun 2023
- Study completion16 Jun 2023
Why it stopped: Clinical trial was halted prematurely due to low enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Immune Mediated AnemiaStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Jamie Y Hur, D.O., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05502783
- Sponsor's protocol ID
- 10000758
- Other IDs
- 000758-H
- Status last verified
- Oct 2025
- First submitted
- 12 Aug 2022
- Last updated
- 5 Dec 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study Using Fostamatinib to Treat Post-Hematopoietic Stem Cell Transplant Immune-Mediated Cytopenias
- ClinicalTrials.gov ID
- NCT05502783
- Acronym / sponsor ID
- 10000758
- Keywords
- Allogeneic Hematopoietic Stem Cell Transplant, Graft Versus Host Disease, Spleen Tyrosine Kinase (Syk) Inhibitor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Clinical trial was halted prematurely due to low enrollment
- Enrolment
- 1 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 10 Mar 2023 (actual)
- Primary completion
- 16 Jun 2023 (actual)
- Study completion
- 16 Jun 2023 (actual)
- First posted
- 16 Aug 2022 (actual)
- Results
- Posted 5 Dec 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Study Description:
This open label phase II trial is designed to evaluate the efficacy of fostamatinib in the treatment of post-transplant cytopenias as assessed by hematologic improvement in anemia and/or thrombocytopenia following a 12-week treatment course. Patients who respond to the 12-week treatment course on this single arm study are eligible and have the option to enroll on the extended access trial.
Objectives:
The primary objective is to assess efficacy of fostamatinib for stable hematologic recovery during post-hematopoietic stem cell transplant immune mediated anemia and/or thrombocytopenia.
The secondary objective is to assess efficacy of fostamatinib for clinically-relevant outcomes in post-hematopoietic stem cell transplant patients.
The exploratory objective is to evaluate changes in serologic markers that may be associated with cytopenias while on treatment to identify key elements for fostamatinib response.
Endpoints:
Primary endpoints:
The proportion of subjects with hematologic recovery that is stable, defined as improvement documented in 2 consecutive available readings at least 2 weeks apart, without recent blood product transfusion support in the past 7 days.
-Hematologic recovery is defined as:
--Hemoglobin >=10 g/dL (or at least >=2 g/dL above baseline) in subjects enrolled with posttransplant anemia. In subjects with symptomatic anemia, a hemoglobin increase of at least >=2 g/dL above baseline is required
OR
--Platelets >= 50 x 10^9/L (or at least >=20 x 10^9/L above baseline) in subjects enrolled with posttransplant thrombocytopenia
OR
--Both of the above criteria in subjects with posttransplant Evans syndrome
Secondary endpoints:
-Proportion of subjects who achieve objective hematologic recovery within the 12-week treatment course defined as:
--Hemoglobin >=9 g/dL (or at least >=1 g/dL above baseline) in subjects enrolled with anemia or at least >=1 g/dL above baseline in subjects with symptomatic anemia
OR
--Platelets >= 30 x 10^9/L (or at least >=10 x 10^9/L above baseline) in subjects enrolled with thrombocytopenia
OR
-- Either of the above criteria in subjects with Evans syndrome
- Average weekly requirement of transfused blood component or growth factor requirement (total units of PRBC or Platelets/week, total dose of growth factor/week) by week 12, compared to the week prior to the start of the study drug.
- Change in corticosteroid dose over time, measured by median daily weight-based prednisone-equivalent corticosteroid dose in a week, from week 1 to week 12
- Change in other immunosuppressant dose over time, measured by median daily dose of the immunosuppressant in a week, from week 1 to week 12
- Number of patients who achieved >=50% steroid dose reduction by week 12 compared to week 1
- Incidence and severity of cGVHD according to 2014 NIH Consensus Criteria, at baseline before the initiation of treatment, and at week 12
Exploratory endpoints:
- Absolute percentage change in B cell chimerism pre-treatment, vs weeks 4 and 12.
- cPRA change in the preformed HLA antibodies before and after treatment in subjects with thrombocytopenia or Evans syndrome
- Percent MFI change of preformed HLA antibodies before and after treatment. This applies for antibodies with an MFI >1,000.
- Anti-RBC alloantibody titer change in patients with hemolytic anemia before and after treatment
- Assessment of serial cytokines pre-treatment and weeks 4 and 12. Cytokines include CRP, IL-1, IL-2, IL-6, IFN gamma, TNF alpha, EPO and TPO
- Immunoglobulin level changes pre-treatment, and weeks 4 and 12.
- Absolute reticulocyte count, lactate dehydrogenase, haptoglobin changes pre-treatment, and at each biweekly study visit, in patients with anemia or Evans syndrome
- Change in the weekly rate and severity of bleeding according to the total score of the ITP-Bleeding Scale (IBLS), from week -1 (one week before the start of the study drug) to week 12, in subjects with thrombocytopenia or Evans syndrome
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fostamatinib in Participants Post-Hematopoietic Stem Cell Transplant with Immune-Mediated Cytopenias | Experimental | Drug: fostamatinibParticipants Post-Hematopoietic Stem Cell Transplant with Immune-Mediated Cytopenias will receive fostamatinib 100 mg BID by mouth for 4 weeks. On the 4-week evaluation, a) if cytopenia improves (hemoglobin =10 g/dL, platelets = 50 x 109/L), patients will continue the same dose for a total of 12 weeks, b) if refractory cytopenias persist (hemoglobin < 10 g/dL, platelets < 50 x 109/L), the dose will be increased to 150 mg BID. Subjects with persistent (=2 readings, 2 weeks apart) loss of hematologic response after week 5 can increase their dose to 150 mg BID until at end of the study on week 12. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 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:
Key exclusion criteria (14 listed)
- Severe psychiatric illness or mental deficiency sufficient to make making informed consent impossible
- Positive pregnancy test for women of childbearing age within 1 week or being actively lactating
- Immune mediated cytopenia responsive to the standard of care treatment
- Immune mediated cytopenia due to other autoimmune causes, such as systemic lupus erythrocytosis, chronic lymphocytic leukemia
- Patients who have previously received or currently take fostamatinib post-allogeneic transplant
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| fostamatinibParticipants will receive fostamatinib 100 mg BID for 4 weeks. On the 4-week evaluation, a) if cytopenia improves (hemoglobin =10 g/dL, platelets = 50 x 109/L), patients will continue the same dose for a total of 12 weeks, b) if refractory cytopenias persist (hemoglobin < 10 g/dL, platelets < 50 x 109/L), the dose will be increased to 150 mg BID. Subjects with persistent (=2 readings, 2 weeks apart) loss of hematologic response after week 5 can increase their dose to 150 mg BID until at end of the study on week 12. | Drug | Fostamatinib in Participants Post-Hematopoietic Stem Cell Transplant with Immune-Mediated Cytopenias |
Primary outcome
Number of Participants Who Were Able to Maintain Hematologic Recovery
Time frame: Baseline, Week 2, Week 4, Week 6, Week 8, Week 10
Stable hematologic recovery (improvement documented in 2 consecutive available readings) without recent blood product transfusion support (in the past 48-72 hours). Hematologic recovery is defined as: * Hemoglobin =10 g/dL (or at least =2 g/dL above baseline) in participants enrolled with posttransplant anemia. In participants with symptomatic anemia, a hemoglobin increase of at least =2 g/dL above baseline is required OR * Platelets = 50 x 10^9/L (or at least =20 x 10^9/L above baseline) in participants enrolled with posttransplant thrombocytopenia OR * Both of the above criteria in participants with posttransplant Evans syndrome
7 secondary outcomes
Number of Participants Who Achieve Objective Hematologic Recovery
Time frame: Up to 10 weeks
Median Change in Requirement of Transfused Blood Component
Time frame: Baseline, Week 2, Week 4, Week 6, Week 8, Week 10
Median Change in Requirement of Growth Factor Injections
Time frame: Baseline, Week 2, Week 4, Week 6, Week 8, Week 10
Median Change in Requirement Corticosteroid Dose (Prednisone)
Time frame: Baseline, Week 2, Week 4, Week , Week 8, Week 10
Median Change in Other Immunosuppressant Dose
Time frame: Baseline, Week 2, Week 4, Week 6, Week 8, Week 10, Week 12
Number of Participants Who Developed Mild, Moderate or Severe Chronic Graft vs Host Disease (GVHD) According to 2014 NIH Consensus Criteria
Time frame: Up to 10 weeks
Number of Patients Who Achieved 50% Steroid Dose Reduction
Time frame: Up to Week 10
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jamie Y Hur, D.O. principal investigator
National Heart, Lung, and Blood Institute (NHLBI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing fostamatinib in people with Immune Mediated Anemia and Immune Mediated Thrombocytopenia, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 5 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Who Were Able to Maintain Hematologic Recovery (time frame: Baseline, Week 2, Week 4, Week 6, Week 8, Week 10). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Dec 2025.