Extension Study (Extended Access) of Syk-inhibition Using Fostamatinib to Treat Posttransplant Immune-mediated Cytopenias
- Conditions
- Immune Mediated ThrombocytopeniaChronic GVHDImmune Mediated Anemia
- Interventions
- Registration Number
- NCT05509582
- Brief 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....
- 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
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 20
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Fostamatinib Arm fostamatinib The subjects will receive oral fostamatinib daily for up to 2 years.
- Primary Outcome Measures
Name Time Method Proportion of subjects who are able to maintain hematologic recovery 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
- Secondary Outcome Measures
Name Time Method Change in corticosteroid dose 12 weeks 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.
Proportion of subjects who are able to taper off fostamatinib by >33% while maintaining hematologic recovery 106 weeks 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
Change in the dose of other immunosuppressive agents 12 weeks 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.
Proportion of subjects who are able to completely taper off fostamatinib while maintaining hematologic recovery 106 weeks 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
Trial Locations
- Locations (2)
Atrium Health
🇺🇸Charlotte, North Carolina, United States
National Institutes of Health Clinical Center
🇺🇸Bethesda, Maryland, United States