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Extension Study (Extended Access) of Syk-inhibition Using Fostamatinib to Treat Posttransplant Immune-mediated Cytopenias

NCT05509582 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2025-11-10

No results posted yet for this study

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. 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.

Conditions

  • Immune Mediated Anemia
  • Immune Mediated Thrombocytopenia
  • Chronic GVHD

Interventions

DRUG

fostamatinib

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.

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    lead NIH

Principal Investigators

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

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-11-07
Primary Completion
2025-11-07
Completion
2025-11-07
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05509582 on ClinicalTrials.gov