Fostamatinib for Immune Thrombocytopenia (ITP)

No longer 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests fostamatinib, a medication designed to help individuals with low blood cell counts (cytopenia) following a blood stem cell transplant. The goal is to evaluate fostamatinib's long-term effectiveness in maintaining stable blood cell levels and preventing issues like anemia or severe bleeding. Participants should have previously responded well to fostamatinib in an earlier study and completed that study's requirements. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

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.

Is there any evidence suggesting that fostamatinib is likely to be safe for humans?

Research shows that fostamatinib, a drug under study for individuals with low blood cell levels after a stem cell transplant, has been tested in other conditions. In studies involving patients with chronic immune thrombocytopenia (ITP), fostamatinib was generally well-tolerated. Common side effects included diarrhea and nausea, affecting many patients. Serious side effects were rare, occurring in about 1% of patients, and included issues like pneumonia and high blood pressure.

The FDA has already approved fostamatinib for treating ITP, indicating that its safety is well-understood and considered acceptable for that use. While the side effects can be uncomfortable, most people can manage them. However, discussing these findings with a healthcare provider is important to understand how they might relate to an individual's specific situation. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Fostamatinib is unique because it tackles immune thrombocytopenia (ITP) in a new way by targeting spleen tyrosine kinase (SYK), a key player in the immune system's attack on platelets. Unlike standard treatments like corticosteroids or immunoglobulins, which mainly suppress the immune system broadly, fostamatinib specifically inhibits the signals that lead to platelet destruction. This targeted approach not only has the potential to effectively increase platelet counts but may also reduce some of the side effects associated with broader immune suppression. Researchers are excited about fostamatinib because it offers a novel mechanism of action that could provide a more precise treatment option for people with ITP.

What evidence suggests that fostamatinib might be an effective treatment for cytopenia?

Research shows that fostamatinib, the treatment under study in this trial, can increase platelet counts in people with certain blood conditions. In earlier studies, 90% of patients with ongoing immune thrombocytopenia (ITP) reached a safe platelet level after treatment, improving their blood's ability to clot and stop bleeding. Another study found that most patients who responded to fostamatinib saw their platelet counts improve within the first few weeks. These results suggest that fostamatinib might help maintain stable blood cell levels after a stem cell transplant, potentially reducing the risk of serious bleeding and other complications. ^{6 7 8 9 10}

Who Is on the Research Team?

Jamie Y Hur, D.O.

Principal Investigator

National Heart, Lung, and Blood Institute (NHLBI)

Are You a Good Fit for This Trial?

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 for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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 (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 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]

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]

Citations

1. tavalissehcp.com tavalissehcp.com/efficacy

TAVALISSE® (fostamatinib disodium hexahydrate) tablets ROBUST IMPROVEMENTS IN PLATELET COUNTS WERE OBSERVED AFTER PRIOR STEROID AND/OR TPO-RA USE · 56% of 2nd-line patients responded within 4 weeks · 76% of 2nd-line ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7540289/

Fostamatinib is an effective second‐line therapy in patients ... Response to fostamatinib of ≥50 000/µl was achieved in 9/10 (90%) patients with persistent ITP, 11/19 (58%) patients with 1 to <2 years of ITP, and 59 (51%) of ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT02076412

Study Details | NCT02076412 | A Efficacy and Safety ... The purpose of this study is to determine whether fostamatinib is safe and effective in the treatment of persistent/chronic Immune Thrombocytopenic Purpura ...

4. rigel.com rigel.com/news-media/press-releases/detail/318/new-tavalisse-data-analyses-to-be-presented-at

New TAVALISSE® Data Analyses To Be Presented at ... --In a Post-hoc Analysis of Patients With at Least One Year of Treatment, 91% of ITP Patients Achieved Platelet Counts ≥ 50,000/µL and Incidence ...

5. tavalisse.com tavalisse.com/whattoexpect

TAVALISSE can help you reach your treatment goals Most patients who had a positive response to taking TAVALISSE saw increased platelet counts during their first weeks on treatment, while others took longer to ...

6. tavalissehcp.com tavalissehcp.com/safety

TAVALISSE® (fostamatinib disodium hexahydrate) tablets Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of ...

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2018/209299lbl.pdf

TAVALISSE™ (fostamatinib - accessdata.fda.gov The data described below reflect exposure to TAVALISSE in 102 patients with chronic ITP who had received one or more prior ITP treatment(s). Groups were ...

8. ema.europa.eu ema.europa.eu/en/documents/product-information/tavlesse-epar-product-information_en.pdf

Tavlesse, INN-fostamatinib - EMA TAVLESSE is indicated for the treatment of chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (see section 5.1). 4.2 ...

9. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/n/tr82698683820701/tr82698683820701_ch01_bp1/

Clinical Review - Fostamatinib (Tavalisse) - NCBI - NIH In the FIT1 trial, among patients in the fostamatinib group, the most common AEs (≥ 5%) were diarrhea (41%), nausea (29%), increased alanine transaminase (ALT) ...

10. tavalissehcp.com tavalissehcp.com/

Homepage | TAVALISSE® (fostamatinib disodium ... Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of ...