Fostamatinib for Immune Thrombocytopenia (ITP)

Recruiting in Palo Alto (17 mi)

+1 other location

Overseen byJamie Y Hur, D.O.

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

No Placebo Group

Prior Safety Data

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

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.

Research Team

Jamie Y Hur, D.O.

Principal Investigator

National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria

This trial is for people who've had a blood stem cell transplant and are experiencing low blood cell counts. They must have responded well to fostamatinib in a previous study, not be pregnant or breastfeeding, have certain minimum levels of platelets and hemoglobin, agree to use effective birth control, and not suffer from severe psychiatric illness or other health conditions that could interfere with the study.

Inclusion Criteria

I agree to use two forms of birth control or abstain from sex to avoid pregnancy during and for 1 month after the study.

Subjects who were enrolled on phase II trial of fostamatinib and deemed responders at the time of rollover to the extended access trial

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)

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Exclusion Criteria

Positive pregnancy test for women of childbearing age within 1 week or being actively lactating

I do not have a severe mental illness that prevents me from understanding this study.

ALT or AST >3 times the upper limit of normal

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Treatment Details

Interventions

Fostamatinib (Syk Inhibitor)

Trial OverviewThe trial is testing the long-term effects of fostamatinib on patients with cytopenia after a stem cell transplant. Participants will continue taking the same dose they received in an earlier phase of research for up to 21 months, with regular medical assessments and blood tests every month and every three months respectively.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Fostamatinib ArmExperimental Treatment1 Intervention

The subjects will receive oral fostamatinib daily for up to 2 years.