What side effects are associated with this type of intervention?

Common treatments for Myelodysplastic Syndromes (MDS) include hypomethylating agents (HMAs) like azacitidine and decitabine, which have similar efficacy and toxicity profiles, including cytopenias, infections, gastrointestinal toxicity, and fatigue. Lenalidomide, used for lower-risk MDS, can cause cytopenias, fatigue, and gastrointestinal issues. Thrombopoietin mimetics, although not routinely used due to potential risks, may transiently improve platelet counts but can increase the risk of progression to acute myeloid leukemia (AML) and cause bone marrow fibrosis. These treatments share side effects such as cytopenias and infections, which are also relevant to enzyme inhibition therapies like Fostamatinib.

What prior FDA approvals exist?

FDA-approved treatments for Myelodysplastic Syndromes (MDS) include hypomethylating agents such as azacitidine and decitabine. These agents work by inhibiting DNA methylation, which can help to restore normal function to genes that control cell growth and differentiation. While the context does not mention specific enzyme inhibitors like Fostamatinib, it does highlight the use of hypomethylating agents, which are a cornerstone of MDS treatment.

What other types of research exist?

Promising novel alternatives to Fostamatinib for Myelodysplastic Syndromes patients in 2024 include emerging FLT3 inhibitors like quizartinib, gilteritinib, and crenolanib, as well as milademetan, a murine double minute 2 inhibitor.

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Tyrosine Kinase Inhibitor

Fostamatinib for Leukemia

Phase 1

Waitlist Available

Led By Guillermo M Bravo

Research Sponsored by M.D. Anderson Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Eastern Cooperative Oncology Group (ECOG) performance status =< 2

Both females of childbearing potential and males with female partners of childbearing potential must agree to use contraception during the study period and for at least one month after the last dose

Must not have

Female patients with reproductive potential who do not have a negative urine or blood beta-human chorionic gonadotropin (beta human chorionic gonadotropin [HCG]) pregnancy test at screening

Uncontrolled or poorly controlled hypertension, defined as systolic blood pressure >= 135 mmHg or diastolic blood pressure >= 85 mmHg, whether or not the subject is receiving anti-hypertensive treatment

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

Summary

This trial is testing a pill called fostamatinib, which may help stop the growth of certain blood cancers in patients who haven't responded to other treatments. It works by blocking enzymes that cancer cells need to grow. Fostamatinib is already approved in the USA and Europe for treating a different condition in adults.

Who is the study for?

Adults with lower-risk myelodysplastic syndromes or chronic myelomonocytic leukemia who didn't improve after treatment with hypomethylating agents. They must have had at least 4 cycles of such therapy, not be pregnant or breastfeeding, able to consent, and willing to use contraception. Excluded are those with very low white blood cells, uncontrolled high blood pressure, other recent cancers (with some exceptions), or recent stem cell transplants.

What is being tested?

The trial is testing fostamatinib's effectiveness and optimal dosage for patients whose tumor growth hasn't been halted by standard treatments. It works by blocking enzymes that cancer cells need to grow.

What are the potential side effects?

While the specific side effects of fostamatinib in this context aren't listed here, common ones may include diarrhea, high blood pressure, nausea, fatigue and liver enzyme elevations. Each patient's experience can vary.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can take care of myself and am up and about more than half of my waking hours.

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I agree to use contraception during and for a month after the study.

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I have MDS or CMML and my risk level is not high according to IPSS-R.

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I am 18 years old or older.

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My kidneys work well enough (creatinine clearance over 30 mL/min).

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am a woman who can have children and have not had a negative pregnancy test.

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My blood pressure is high, above 135/85 mmHg, even with treatment.

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I am not pregnant or breastfeeding.

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I am not currently on any experimental treatments or had chemotherapy, radiotherapy, or immunotherapy in the last 14 days.

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I have not received any treatment for my MDS or CMML.

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I have an infection that isn't getting better with antibiotics.

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I am willing to use effective birth control during the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2013 Phase 2 trial • 101 Patients • NCT01499303

24%

BLOOD ALKALINE PHOSPHATASE INCREASED

24%

NEUTROPENIA

24%

THROMBOCYTOPENIA

19%

CONSTIPATION

19%

ASPARTATE AMINOTRANSFERASE INCREASED

19%

BACK PAIN

19%

PYREXIA

19%

NAUSEA

19%

COUGH

19%

FATIGUE

14%

DYSPNOEA

14%

BLOOD CREATININE INCREASED

14%

ALANINE AMINOTRANSFERASE INCREASED

14%

DIARRHOEA

14%

BLOOD BILIRUBIN INCREASED

10%

ABDOMINAL DISTENSION

10%

ANAEMIA

10%

HYPOTENSION

10%

ARTHRALGIA

10%

HEADACHE

10%

OEDEMA PERIPHERAL

10%

NEUTROPHIL COUNT DECREASED

10%

BLOOD UREA INCREASED

10%

ANXIETY

BLOOD LACTATE DEHYDROGENASE INCREASED

ABDOMINAL PAIN

HYPERTENSION

HYPONATRAEMIA

MYALGIA

NEUTROPENIC SEPSIS

WHITE BLOOD CELL COUNT DECREASED

100%

80%

60%

40%

20%

Study treatment Arm

100mg BID

200mg BID

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (fostamatinib)Experimental Treatment1 Intervention

Patients receive fostamatinib PO BID on days 1-28. Treatment repeats every 28 days for up to 6 cycles (week 24) in the absence of disease progression or unacceptable toxicity.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fostamatinib

FDA approved

0 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Myelodysplastic Syndromes (MDS) include hypomethylating agents (HMAs) like azacitidine and decitabine, which inhibit DNA methylation to reactivate tumor suppressor genes and induce cancer cell differentiation or death. Enzyme inhibitors, such as fostamatinib, target specific enzymes involved in cell signaling pathways that promote cancer cell growth and survival. These mechanisms are important for MDS patients as they help in selecting therapies that effectively target the disease's underlying pathophysiology, potentially improving outcomes and quality of life.

Lenalidomide as a disease-modifying agent in patients with del(5q) myelodysplastic syndromes: linking mechanism of action to clinical outcomes.