What side effects are associated with this type of intervention?

Common treatments for anemia, such as erythropoiesis-stimulating agents (ESAs), hypomethylating agents like azacitidine, and immunosuppressive therapies, have various side effects. ESAs can increase the risk of thrombosis. Hypomethylating agents, including azacitidine, may cause gastrointestinal symptoms (nausea, vomiting, diarrhea) and hematologic toxicities like neutropenia. Immunosuppressive therapies, such as anti-thymocyte globulin (ATG) plus cyclosporine, can lead to infections and other immune-related complications. These side effects should be carefully monitored during treatment.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of anemia, particularly in the context of myelodysplastic syndromes (MDS). Azacitidine, a hypomethylating agent, is used to improve blood cell production in MDS patients. Lenalidomide and pomalidomide, both immunomodulatory drugs, have also been approved for their roles in enhancing erythropoiesis and increasing fetal hemoglobin production. These treatments aim to modify the bone marrow environment to alleviate anemia and improve patient outcomes.

What other types of research exist?

Promising alternatives for anemia treatment in 2024 include lenalidomide combined with epoetin alfa, which has shown efficacy in myelodysplastic syndromes, and thrombopoietin receptor agonists like eltrombopag, which can increase platelet counts and potentially improve anemia in certain conditions.

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INCB000928 for Myelodysplastic Syndrome (LIMBER Trial)

Phase 1 & 2

Waitlist Available

Research Sponsored by Incyte Corporation

Eligibility Criteria Checklist

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

Must have

For MDS participants: Not requiring cytoreductive therapy other than hydroxyurea

Participants who are transfusion-dependent or present with symptomatic anemia

Must not have

History of clinically significant or uncontrolled cardiac disease

Undergoing treatment with a strong or potent inhibitor or inducer of CYP3A4/5 within 28 days or 5 half-lives (whichever is longer) before the first dose of study drug or expected to receive such treatment during the study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up approximately upto 7 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new drug, INCB000928, for patients with MDS or MM who need regular blood transfusions or have severe anemia. The goal is to see if the drug can improve blood cell counts and reduce the need for transfusions by acting on specific pathways in the body.

Who is the study for?

This trial is for adults with Myelodysplastic Syndromes (MDS) or Multiple Myeloma (MM) who need regular blood transfusions or have anemia symptoms. MDS patients must have tried other anemia treatments without success and can't be on certain therapies. MM patients should have already tried standard treatments without success. Participants must not be pregnant, planning to become pregnant, or father a child.

What is being tested?

The study tests INCB000928's safety and how well it works as a single therapy in people with MDS or MM who are dependent on transfusions or suffer from symptomatic anemia. It's a Phase 1/2 trial where researchers will also look at how the body processes the drug and its effects on the disease.

What are the potential side effects?

Specific side effects of INCB000928 aren't listed, but common ones for cancer drugs include nausea, fatigue, risk of infection, bleeding issues, liver problems, allergic reactions and changes in heart rhythm which will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

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

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I have MDS and only use hydroxyurea for treatment.

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I need regular blood transfusions or have symptoms of anemia.

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My diagnosis of multiple myeloma is confirmed through tissue examination.

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I have MDS and treatments like ESAs or lenalidomide didn’t work for me.

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I have MM and standard treatments like chemotherapy or immunotherapy didn't work for me.

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I have been diagnosed with MDS, CMML, or an unclassifiable MDS/MPN overlap.

Exclusion Criteria

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

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I have a serious heart condition that is not under control.

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I am not on strong medication that affects liver enzymes within the last 28 days or expected during the study.

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I have been diagnosed with a long-term liver condition.

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I am currently on treatment for a long-term or active infection.

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I have had or am a candidate for a stem cell transplant from a donor.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ approximately upto 7 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~approximately upto 7 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and approximately upto 7 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Number of treatment-related adverse events

Secondary study objectives

AUC0-t

Cmax

Duration of RBC-TI period

+14 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: INCB000928Experimental Treatment1 Intervention

INCB000928 will be administered in participants with MDS or MM who are transfusion-dependent or present with symptomatic anemia.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

INCB000928

2021

Completed Phase 2

~70

0 / 11Eligibility 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 anemia include erythropoiesis-stimulating agents (ESAs) like epoetin and darbepoetin, which stimulate the bone marrow to produce more red blood cells. Hypomethylating agents such as azacitidine and decitabine can also be used, particularly in myelodysplastic syndromes, by reactivating silenced genes that promote normal cell differentiation and proliferation. These treatments are crucial for anemia patients as they address the underlying causes of reduced red blood cell production, thereby improving oxygen delivery to tissues and alleviating symptoms like fatigue and weakness.

Clinical experience with fetal hemoglobin induction therapy in patients with β-thalassemia.[Regional clinical audit, guideline targets, and local and regional benchmarks].