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

Conditions

• Chronic GVHD
• Immune Mediated Anemia
• Immune Mediated Thrombocytopenia

Eligibility Criteria

Sex

ALL

Age

18 Years - 75 Years

Healthy Volunteers

Not accepted

Interventions

• fostamatinib — DRUG
  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 Details

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.

Key Dates

Start date

Mar 10, 2023

Status verified

Oct 2025

Primary completion

Jun 16, 2023

Completion

Jun 16, 2023

Study Design

Enrollment

1 participants (actual)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Fostamatinib in Participants Post-Hematopoietic Stem Cell Transplant with Immune-Mediated Cytopenias
  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.

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 ]

Locations (1)

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