What side effects are associated with this type of intervention?

Common treatments for Myelodysplastic Syndrome (MDS) include lenalidomide, erythropoiesis-stimulating agents (ESAs), and supportive care measures. Lenalidomide can cause side effects such as cytopenias, including neutropenia and thrombocytopenia, as well as fatigue and gastrointestinal symptoms. ESAs, like epoetin alfa and darbepoetin alfa, may lead to hypertension, thromboembolic events, and pure red cell aplasia. Supportive care, including transfusions, can result in iron overload and associated complications. The safety profile of Etavopivat (FT-4202) is still under investigation, but it aims to improve anemia with potentially fewer side effects compared to existing therapies.

What prior FDA approvals exist?

The FDA has approved several treatments for Myelodysplastic Syndrome (MDS), including hypomethylating agents (HMAs) such as azacitidine and decitabine, which are used to manage the disease. Erythropoiesis-stimulating agents (ESAs) like epoetin alfa and darbepoetin alfa are also approved to treat anemia in MDS patients. While thrombopoietin mimetics like romiplostim have been evaluated, they are not routinely recommended due to potential risks of progression to acute myeloid leukemia (AML).

What other types of research exist?

A promising novel alternative to Etavopivat (FT-4202) for Myelodysplastic Syndrome patients is PTC-028, a tubulin polymerization inhibitor that has shown efficacy in preclinical models.

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Etavopivat for Myelodysplastic Syndrome

Phase 2

Waitlist Available

Research Sponsored by Forma Therapeutics, Inc.

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 4(first 6 participants), 16, 24, and 48 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a medication called etavopivat to see if it can help treat anemia in adults with certain types of Myelodysplastic Syndromes (MDS). These patients have a form of anemia that may not respond well to usual treatments. Etavopivat works by improving the function of red blood cells and is being developed for the treatment of sickle cell disease and other hemoglobin disorders.

Who is the study for?

Adults with low to intermediate risk Myelodysplastic Syndromes (MDS) and anemia can join this trial. They must have less than 5% bone marrow blasts, not be pregnant or breastfeeding, agree to use contraception, and cannot have had certain treatments like luspatercept recently. People with severe infections, other cancers within the last two years, significant heart or kidney disease are excluded.

What is being tested?

The trial is testing Etavopivat's safety and effectiveness in treating anemia for MDS patients who are at very low to intermediate risk. It will include adults who haven't responded well to other treatments or for whom those treatments aren't suitable.

What are the potential side effects?

While specific side effects of Etavopivat aren't listed here, common ones may include gastrointestinal symptoms like nausea or diarrhea, potential liver enzyme changes, fatigue, and possible allergic reactions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 4(first 6 participants), 16, 24, and 48 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~4(first 6 participants), 16, 24, and 48 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 4(first 6 participants), 16, 24, and 48 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

The hematologic improvement based on an erythroid response (HI -E) ≥ 8 weeks duration in patients with MDS after 16 weeks of etavopivat treatment

Secondary study objectives

Blood Transfusion

Change from baseline in mean daily dose of iron chelation therapy

Body Weight Changes

+10 more

Awards & Highlights

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: Etavopivat 400 mg QD dailyExperimental Treatment1 Intervention

Non-transfusion dependent (NTD), Low transfusion burden (LTB) , and High transfusion burden (HTB) patients

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Etavopivat

2024

Completed Phase 1

~20

Do I qualify?Apply below to find out

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 Syndrome (MDS) include erythropoiesis-stimulating agents (ESAs), which boost red blood cell production by stimulating the bone marrow, and thrombopoietin receptor agonists, which increase platelet production to reduce bleeding risks. DNA hypomethylating agents, such as azacitidine, work by reversing abnormal DNA methylation patterns, thereby restoring normal function to genes involved in blood cell production. The investigational drug etavopivat (FT-4202), a pyruvate kinase-R (PKR) activator, aims to enhance red blood cell metabolism and improve their survival and function. These treatments are crucial for MDS patients as they address the underlying issues of ineffective hematopoiesis, thereby improving blood counts and reducing the need for transfusions.

Recent advances in the treatment of lower-risk non-del(5q) myelodysplastic syndromes (MDS).Anemia as the Main Manifestation of Myelodysplastic Syndromes.Strategic Role of Nuclear Inositide Signalling in Myelodysplastic Syndromes Therapy.