Immune Mediated AnemiaPhase 2NCT05509582
Extension Study (Extended Access) of Syk-inhibition Using Fostamatinib to Treat Posttransplant 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
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Nov 2025to 7 Nov 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Subjects who were enrolled on phase II trial of fostamatinib and deemed responders at the time of rollover to the extended access trial which is defined as:
- Hemoglobin >= 9 g/dL (or at least >= 1 g/dL above baseline) in subjects enrolled with posttransplant anemia without transfusion support, at least once during the 12-week phase II trial.
- OR
- --Platelets >= 30 X 10^9/L (or at least >= 10 X 10^9/L above baseline) without transfusion support, at least once during the 12-week phase II trial, in subjects enrolled with posttransplant thrombocytopenia
- OR
- --Either of the above criteria in subjects with posttransplant Evans syndrome
Show all 9Show fewer
- Completed the end of study visit (week 12) on the initial protocol (A Phase II Study of Syk-inhibition using Fostamatinib to treat Post-Transplant Immune-mediated Cytopenias).
- 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.
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
- Uncontrolled hypertension (systolic blood pressure >140mmHg or diastolic blood pressure >90mmHg)
- ALT or AST >3 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 evidence of graft rejection (based on clinical suspicion supported by BM biopsy data and/or chimerism studies and/or MLR)
- Neutropenia, defined as absolute neutrophil count <= 1.0 X 10^9/L
- 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.
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: Extension Study (Extended Access) of Syk-inhibition Using Fostamatinib to Treat Post-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. Up to 20% of people who have blood stem cell transplants develop cytopenias, which 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 evaluate the long-term effects of a study drug (fostamatinib) in people with cytopenia after a blood stem cell transplant.
Eligibility:
People who responded well to fostamatinib in an earlier study.
Design:
Participants will be screened. They will have a physical exam and blood tests.
Fostamatinib is an oral tablet taken by mouth. Participants will take the pills at the same dose and frequency as they did during the previous study. They will take the pills for up to 21 months. The dosage of the drug may be reduced over time if their blood cell levels are stable.
Participants will have a medical assessment every month. This can be with their local doctor or at the NIH clinic.
Participants will have blood tests every 3 months.
Participants will have a follow-up visit after they stop taking the drug. Their vital signs will be taken, and they will have blood drawn. They will answer questions about their health.
Read the detailed description
Study Description:
This is an extension trial of a phase II study which is designed to evaluate the long-term safety and efficacy of fostamatinib in the treatment of post-transplant cytopenias as assessed by hematologic improvement in anemia and/or thrombocytopenia following an additional 12-84 weeks of treatment on the initial phase II trial. The subjects who are deemed responders to the initial 12-week treatment, will be candidates to enroll onto the extension trial for up to 2 years of treatment.
Objectives:
The primary objective is to evaluate the efficacy for the use of fostamatinib in subjects with post-transplant anemia and/or thrombocytopenia at the end of total 24 weeks of treatment.
The secondary objectives are to evaluate the long-term efficacy of subjects on fostamatinib while weaning off fostamatinib treatment either partially or completely.
Endpoints:
The primary endpoint is:
-Proportion of subjects who are able to maintain hematologic recovery (as defined below) by the end of 12 weeks on the extended access trial (total of 24 weeks fostamatinib treatment).
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
The secondary endpoints:
- Proportion of subjects who are able to taper off fostamatinib by >33% while maintaining hematologic recovery as outlined above at the end of the treatment.
- Proportion of subjects who are able to completely taper off fostamatinib while maintaining hematologic recovery as outlined above at the end of the treatment.
- Change in corticosteroid dose in the total 24 weeks on fostamatinib, measured by median daily weight-based prednisone-equivalent corticosteroid dose in a week, from week 1 to week 24
- Change in the dose of other immunosuppressive agents in the total 24 weeks on fostamatinib, measured by median daily dose of the immunosuppressant in a week, from week 1 to week 24
- 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
Subjects will receive fostamatinib at the same dose as the dose they receive at the time of rollover. The dose could be reduced to 100mg daily, 150 mg daily, 100 mg twice a day if a dose-limiting adverse event occurred. After the initial 12 weeks on the extended access (total of 24 weeks of treatment), subjects will have the option to start tapering fostamatinib if they are maintaining hematologic recovery. Subjects with persistent (=2 readings, 2 weeks apart) loss of hematologic response, defined as Hb<9g/dL and/or PLT< 30 X 10^9/L can increase their dose to next higher dose. Once count stabilization again occurs, a slow dose reduction to next lowest dose (100mg or 150mg) can be performed to identify the lowest dose necessary to keep counts over these thresholds.
| Study group | Role | What they receive |
|---|---|---|
| Fostamatinib Arm | Experimental | The subjects will receive oral fostamatinib daily for up to 2 years. Receives: Drug: fostamatinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects who are able to maintain hematologic recovery
Proportion of subjects who are able to maintain hematologic recovery (as defined below) by the end of 12 weeks on the extended access trial (total of 24 weeks fostamatinib treatment). Hematologic recovery is defined as: Hemoglobin >=10 g/dL (or at least >=2 g/dL above baseline) in subjects enrolled with post-transplant anemia. In subjects with symptomatic anemia, a hemoglobin increase of at least >=2 g/dL above baseline is required OR Platelets >= 50 x 109/L (or at least =20 x 10^9/L above baseline) in subjects enrolled with post-transplant thrombocytopenia OR Both of the above criteria in subjects with post-transplant Evan s syndrome
Measured: 12 weeks
Secondary outcomes (4)
Proportion of subjects who are able to taper off fostamatinib by >33% while maintaining hematologic recovery
Hematologic recovery is defined as: Hemoglobin >=10 g/dL (or at least >=2 g/dL above baseline) in subjects enrolled with post-transplant 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 post-transplant thrombocytopenia OR Both of the above criteria in subjects with post-transplant Evan s syndrome
Measured: 106 weeks
Change in the dose of other immunosuppressive agents
Change in the dose of other immunosuppressive agents in the total 24 weeks on fostamatinib, measured by median daily dose of the immunosuppressant in a week, from week 1 to week 24.
Measured: 12 weeks
Proportion of subjects who are able to completely taper off fostamatinib while maintaining hematologic recovery
Hematologic recovery is defined as: Hemoglobin >=10 g/dL (or at least >=2 g/dL above baseline) in subjects enrolled with post-transplant 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 post-transplant thrombocytopenia OR Both of the above criteria in subjects with post-transplant Evan s syndrome
Measured: 106 weeks
Change in corticosteroid dose
Change in corticosteroid dose in the total 24 weeks on fostamatinib, measured by median daily weight-based prednisone-equivalent corticosteroid dose from week 1 to week 24.
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2022
- Study start7 Nov 2025
- Primary completion7 Nov 2025
- Study completion7 Nov 2025
Why it stopped: No responders in parent protocol 000758; this follow-on protocol not activated and PI leaving NIH
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 0 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
- NCT05509582
- Sponsor's protocol ID
- 10000760
- Other IDs
- 000760-H
- Status last verified
- Nov 2025
- First submitted
- 19 Aug 2022
- Last updated
- 10 Nov 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Extension Study (Extended Access) of Syk-inhibition Using Fostamatinib to Treat Post-Transplant Immune-mediated Cytopenias
- ClinicalTrials.gov ID
- NCT05509582
- Acronym / sponsor ID
- 10000760
- Keywords
- Allogeneic Hematopoietic Stem Cell Transplant, Graft Versus Host Disease, Spleen Tyrosine Kinase (Syk) Inhibitor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: No responders in parent protocol 000758; this follow-on protocol not activated and PI leaving NIH
- Enrolment
- 0 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 7 Nov 2025 (actual)
- Primary completion
- 7 Nov 2025 (actual)
- Study completion
- 7 Nov 2025 (actual)
- First posted
- 22 Aug 2022 (actual)
- Results
- Not posted in the source record
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. Up to 20% of people who have blood stem cell transplants develop cytopenias, which 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 evaluate the long-term effects of a study drug (fostamatinib) in people with cytopenia after a blood stem cell transplant.
Eligibility:
People who responded well to fostamatinib in an earlier study.
Design:
Participants will be screened. They will have a physical exam and blood tests.
Fostamatinib is an oral tablet taken by mouth. Participants will take the pills at the same dose and frequency as they did during the previous study. They will take the pills for up to 21 months. The dosage of the drug may be reduced over time if their blood cell levels are stable.
Participants will have a medical assessment every month. This can be with their local doctor or at the NIH clinic.
Participants will have blood tests every 3 months.
Participants will have a follow-up visit after they stop taking the drug. Their vital signs will be taken, and they will have blood drawn. They will answer questions about their health.
Detailed description
Study Description:
This is an extension trial of a phase II study which is designed to evaluate the long-term safety and efficacy of fostamatinib in the treatment of post-transplant cytopenias as assessed by hematologic improvement in anemia and/or thrombocytopenia following an additional 12-84 weeks of treatment on the initial phase II trial. The subjects who are deemed responders to the initial 12-week treatment, will be candidates to enroll onto the extension trial for up to 2 years of treatment.
Objectives:
The primary objective is to evaluate the efficacy for the use of fostamatinib in subjects with post-transplant anemia and/or thrombocytopenia at the end of total 24 weeks of treatment.
The secondary objectives are to evaluate the long-term efficacy of subjects on fostamatinib while weaning off fostamatinib treatment either partially or completely.
Endpoints:
The primary endpoint is:
-Proportion of subjects who are able to maintain hematologic recovery (as defined below) by the end of 12 weeks on the extended access trial (total of 24 weeks fostamatinib treatment).
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
The secondary endpoints:
- Proportion of subjects who are able to taper off fostamatinib by >33% while maintaining hematologic recovery as outlined above at the end of the treatment.
- Proportion of subjects who are able to completely taper off fostamatinib while maintaining hematologic recovery as outlined above at the end of the treatment.
- Change in corticosteroid dose in the total 24 weeks on fostamatinib, measured by median daily weight-based prednisone-equivalent corticosteroid dose in a week, from week 1 to week 24
- Change in the dose of other immunosuppressive agents in the total 24 weeks on fostamatinib, measured by median daily dose of the immunosuppressant in a week, from week 1 to week 24
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fostamatinib Arm | Experimental | Drug: fostamatinibThe subjects will receive oral fostamatinib daily for up to 2 years. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Subjects who were enrolled on phase II trial of fostamatinib and deemed responders at the time of rollover to the extended access trial which is defined as:
- OR
- --Platelets >= 30 X 10^9/L (or at least >= 10 X 10^9/L above baseline) without transfusion support, at least once during the 12-week phase II trial, in subjects enrolled with posttransplant thrombocytopenia
- OR
- --Either of the above criteria in subjects with posttransplant Evans syndrome
Key exclusion criteria (8 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
- Uncontrolled hypertension (systolic blood pressure >140mmHg or diastolic blood pressure >90mmHg)
- ALT or AST >3 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.
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 |
|---|---|---|
| fostamatinibSubjects will receive fostamatinib at the same dose as the dose they receive at the time of rollover. The dose could be reduced to 100mg daily, 150 mg daily, 100 mg twice a day if a dose-limiting adverse event occurred. After the initial 12 weeks on the extended access (total of 24 weeks of treatment), subjects will have the option to start tapering fostamatinib if they are maintaining hematologic recovery. Subjects with persistent (=2 readings, 2 weeks apart) loss of hematologic response, defined as Hb<9g/dL and/or PLT< 30 X 10^9/L can increase their dose to next higher dose. Once count stabilization again occurs, a slow dose reduction to next lowest dose (100mg or 150mg) can be performed to identify the lowest dose necessary to keep counts over these thresholds. | Drug | Fostamatinib Arm |
Primary outcome
Proportion of subjects who are able to maintain hematologic recovery
Time frame: 12 weeks
Proportion of subjects who are able to maintain hematologic recovery (as defined below) by the end of 12 weeks on the extended access trial (total of 24 weeks fostamatinib treatment). Hematologic recovery is defined as: Hemoglobin >=10 g/dL (or at least >=2 g/dL above baseline) in subjects enrolled with post-transplant anemia. In subjects with symptomatic anemia, a hemoglobin increase of at least >=2 g/dL above baseline is required OR Platelets >= 50 x 109/L (or at least =20 x 10^9/L above baseline) in subjects enrolled with post-transplant thrombocytopenia OR Both of the above criteria in subjects with post-transplant Evan s syndrome
4 secondary outcomes
Proportion of subjects who are able to taper off fostamatinib by >33% while maintaining hematologic recovery
Time frame: 106 weeks
Change in the dose of other immunosuppressive agents
Time frame: 12 weeks
Proportion of subjects who are able to completely taper off fostamatinib while maintaining hematologic recovery
Time frame: 106 weeks
Change in corticosteroid dose
Time frame: 12 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Nov 2025.
- 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 9 inclusion and 8 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: withdrawn, as of the sponsor's last update on 10 Nov 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Proportion of subjects who are able to maintain hematologic recovery (time frame: 12 weeks). 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 10 Nov 2025.