Extension Study (Extended Access) of Syk-inhibition Using Fostamatinib to Treat Posttransplant 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
  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 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. 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.

Key Dates

Start date

Nov 7, 2025

Status verified

Nov 2025

Primary completion

Nov 7, 2025

Completion

Nov 7, 2025

Study Design

Enrollment

0 participants (actual)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Fostamatinib Arm
  The subjects will receive oral fostamatinib daily for up to 2 years.

Primary Outcome Measure

Proportion of subjects who are able to maintain hematologic recovery [ Time Frame: 12 weeks ]

Locations (1)

Find similar trials in Bethesda, MD