Fostamatinib for Immune Thrombocytopenia (ITP)

Recruiting at 1 trial location

Overseen By Richard W Childs, M.D.

Age: 18+

Sex: Any

Trial Phase: Phase 2

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

Must be taking: Fostamatinib

Disqualifiers: Severe psychiatric illness, Uncontrolled hypertension, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 5

Duration: 21 months

20 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

What data supports the effectiveness of the drug Fostamatinib for treating immune thrombocytopenia (ITP)?

Fostamatinib has been shown to increase platelet counts in adults with chronic ITP who did not respond well to previous treatments. In clinical trials, more patients achieved stable platelet levels with Fostamatinib compared to a placebo, and many maintained these levels for over a year. ^{1 2 3 4 5}

Is fostamatinib safe for humans?

Fostamatinib has been studied in clinical trials for immune thrombocytopenia (ITP) and has shown a generally manageable safety profile, with common side effects including diarrhea, high blood pressure, nausea, and increased liver enzymes. No serious safety risks were identified in these studies. ^{2 3 4 5 6}

How is the drug Fostamatinib different from other treatments for immune thrombocytopenia (ITP)?

Fostamatinib is unique because it is the first drug that works by inhibiting spleen tyrosine kinase (Syk), a protein involved in the destruction of platelets, which is a new approach compared to other treatments. It is taken orally and is specifically used for adults with chronic ITP who haven't responded well to other treatments. ^{1 2 4 5 7}

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)

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive oral fostamatinib daily for up to 21 months, with monthly medical assessments and blood tests every 3 months

21 months

Monthly visits (in-person or with local doctor), blood tests every 3 months

Follow-up

Participants have a follow-up visit after stopping the drug, including vital signs check and blood tests

4 weeks

1 visit (in-person)

Treatment Details

Interventions

• Fostamatinib

Trial Overview The 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

Group I: Fostamatinib Arm Experimental Treatment 1 Intervention

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

Fostamatinib is already approved in United States for the following indications:

Approved in United States as Tavalisse for:

• Chronic immune thrombocytopenia (ITP)

Who Is Running the Clinical Trial?

National Heart, Lung, and Blood Institute (NHLBI)

Lead Sponsor

Trials

3,987

Recruited

47,860,000+

Findings from Research

Fostamatinib is an effective treatment for chronic immune thrombocytopenia (ITP) in patients who have not responded adequately to at least one prior therapy, as demonstrated in two phase 3 randomized controlled trials (FIT1 and FIT2) across North America, Australia, and Europe.

The drug works by inhibiting spleen tyrosine kinase, which plays a crucial role in the immune response, thereby helping to increase platelet counts in ITP patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fostamatinib for the treatment of chronic immune thrombocytopenia. Connell, NT., Berliner, N.[2021]

Fostamatinib (TAVALISSE™) is a spleen tyrosine kinase (SYK) inhibitor developed by Rigel Pharmaceuticals, recently approved in the US for treating thrombocytopenia in adults with chronic immune thrombocytopenia (ITP) who did not respond adequately to prior treatments.

The approval was based on positive outcomes from the phase III FIT clinical trial program, highlighting its efficacy in increasing platelet counts in patients with ITP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fostamatinib: First Global Approval. Markham, A.[2022]

Fostamatinib is an oral spleen tyrosine kinase inhibitor that effectively increases platelet counts in adults with immune thrombocytopenia who did not respond adequately to previous treatments, as shown in two Phase III trials.

The treatment is generally well tolerated, with lower rates of severe bleeding-related side effects, although common adverse events include diarrhea, nausea, and liver function test elevations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fostamatinib for the treatment of immune thrombocytopenia in adults. Moore, DC., Gebru, T., Muslimani, A.[2021]

Fostamatinib is an effective oral treatment for adults with chronic immune thrombocytopenia (ITP) who have not responded to previous therapies, showing durable responses and maintaining platelet counts above 50 × 10^9/L for at least 12 months.

The drug works by inhibiting the spleen tyrosine kinase (SYK) pathway, which is involved in platelet destruction, but it can cause side effects such as diarrhea, hypertension, nausea, and increased liver enzymes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fostamatinib in chronic immune thrombocytopenia: a profile of its use in the USA. McKeage, K., Lyseng-Williamson, KA.[2022]

In a phase 3 study involving 34 Japanese patients with chronic primary immune thrombocytopenia (ITP), fostamatinib demonstrated significant efficacy, with 36% of patients achieving stable platelet responses compared to none in the placebo group.

Fostamatinib also led to fewer bleeding symptoms and a reduced need for rescue medication, with adverse events being mild to moderate and manageable, indicating a favorable safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fostamatinib for the treatment of Japanese patients with primary immune thrombocytopenia: A phase 3, placebo-controlled, double-blind, parallel-group study. Kuwana, M., Ito, T., Kowata, S., et al.[2023]

References

1. United States pubmed.ncbi.nlm.nih.gov

Fostamatinib for the treatment of chronic immune thrombocytopenia. [2021]

2. New Zealand pubmed.ncbi.nlm.nih.gov

Fostamatinib: First Global Approval. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Fostamatinib for the treatment of immune thrombocytopenia in adults. [2021]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Fostamatinib in chronic immune thrombocytopenia: a profile of its use in the USA. [2022]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Fostamatinib: A Review in Chronic Immune Thrombocytopenia. [2021]

6. England pubmed.ncbi.nlm.nih.gov

Fostamatinib for the treatment of Japanese patients with primary immune thrombocytopenia: A phase 3, placebo-controlled, double-blind, parallel-group study. [2023]

7. England pubmed.ncbi.nlm.nih.gov

Fostamatinib for persistent/chronic adult immune thrombocytopenia. [2021]