What side effects are associated with this type of intervention?

Common treatments for Myelodysplastic Syndrome (MDS) include erythropoiesis-stimulating agents (ESAs), thrombopoietin mimetics, and hypomethylating agents. ESAs can cause hypertension and increase the risk of thromboembolic events. Thrombopoietin mimetics, such as romiplostim, may transiently improve platelet counts but have been associated with an increased risk of progression to acute myeloid leukemia (AML) and bone marrow fibrosis. Hypomethylating agents, like azacitidine and decitabine, are generally better tolerated but can cause myelosuppression, leading to infections, anemia, and bleeding. These side effects should be carefully managed in clinical settings.

What prior FDA approvals exist?

The FDA has approved several treatments for Myelodysplastic Syndrome (MDS), including hypomethylating agents such as azacitidine and decitabine, which are commonly used due to their efficacy and tolerability. Thrombopoietin receptor agonists like romiplostim and eltrombopag have also been evaluated, particularly for their role in managing thrombocytopenia in MDS patients. Additionally, erythropoiesis-stimulating agents (ESAs) are used to treat anemia associated with MDS. These treatments aim to improve blood counts, reduce transfusion needs, and enhance the quality of life for patients with MDS.

What other types of research exist?

Promising novel alternatives to GLB-001 for Myelodysplastic Syndrome patients include: 1) BAY 2402234, a dihydroorotate dehydrogenase (DHODH) inhibitor that induces differentiation and shows efficacy across multiple AML subtypes. 2) ON 01910.Na, which has demonstrated encouraging efficacy and drug tolerance in higher-risk MDS patients unresponsive to hypomethylating agents. 3) Magnetic Fe3O4 nanoparticles with 2-methoxyestradiol, which enhance the therapeutic activity of MDS by affecting cell-cycle progression and apoptosis.

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GLB-001 for Acute Myeloid Leukemia

Phase 1

Recruiting

Research Sponsored by GluBio Therapeutics Inc.

Eligibility Criteria Checklist

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

Must have

Participants must have specific screening laboratory values for WBC, AST, ALT, serum total bilirubin, estimated serum creatinine clearance, INR, aPTT, life expectancy, ECOG Performance Status, and negative pregnancy test for female Participants of child-bearing potential.

Participants is ≥ 18 years of age at the time of signing the Informed Consent Form (ICF).

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new drug, GLB-001, in patients with certain types of blood cancer that haven't responded to other treatments. The goal is to find out how safe the drug is and what dose works best.

Who is the study for?

This trial is for adults over 18 with relapsed or refractory acute myeloid leukemia (R/R AML) or higher-risk myelodysplastic syndromes (HR-MDS). They must have failed or be ineligible for other treatments and meet specific health criteria like blood counts, liver and kidney function. Pregnant women are excluded.

What is being tested?

GLB-001, an oral medication, is being tested in this Phase 1 study on patients with certain types of blood cancers. The study has two parts: dose escalation to assess safety and dosage levels, followed by dose expansion to find the most effective yet tolerable dose for future studies.

What are the potential side effects?

While not specified here, typical side effects in such trials may include nausea, fatigue, diarrhea, risk of infection due to low white blood cell count, bleeding complications from low platelets and potential liver toxicity.

Eligibility Criteria

Inclusion Criteria

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

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My blood tests and health status meet the study's requirements.

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I am 18 years or older and can sign the consent form.

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I have been diagnosed with AML or HR-MDS according to WHO criteria.

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My AML or HR-MDS has not improved with available treatments.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Dose-limiting Toxicity (DLT)

Incidence of Adverse Events (AEs)

Maximum Tolerated Dose (MTD)/Maximum Administered Dose (MAD)

+1 more

Secondary study objectives

CR Rate in Participants with Higher Risk Myelodysplastic Syndromes (HR-MDS)

CR with Incomplete Hematologic Recovery (CRi) Rate in Participants with AML

CR with Partial Hematological Recovery (CRh) Rate in Participants with AML

+31 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

2Treatment groups

Experimental Treatment

Group I: Dose Expansion of GLB-001 as a Monotherapy in Participants with R/R AML and R/R HR-MDS-Phase 1bExperimental Treatment1 Intervention

Part 1b (Dose Expansion) will confirm tolerability of the selected doses and schedules and evaluate whether efficacy is in a range that warrants further clinical development for R/R AML and R/R HR-MDS participants.

Group II: Dose Escalation of GLB-001 as a Monotherapy in Participants with R/R AML and R/R HR-MDS-Phase 1aExperimental Treatment1 Intervention

Part 1a (Dose Escalation) of the study will enroll R/R AML and R/R HR-MDS participants and will evaluate the safety, tolerability, PK, PD and preliminary efficacy of GLB-001 administered orally, and determine the maximum tolerated dose/maximum administered dose (MTD/MAD) in R/R AML or R/R HR-MDS patients who are eligible for dose limiting toxicity (DLT) evaluation.

0 / 4Eligibility 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 Syndrome (MDS) include DNA methyltransferase inhibitors like azacitidine and decitabine, which work by reversing abnormal DNA methylation patterns, thereby reactivating tumor suppressor genes and promoting normal cell differentiation. Histone deacetylase inhibitors modify the chromatin structure to reactivate gene expression. Erythropoiesis-stimulating agents (ESAs) like epoetin alfa boost red blood cell production, addressing anemia. Thrombopoietin mimetics, although controversial, aim to increase platelet counts. These treatments are crucial for MDS patients as they target the underlying genetic and epigenetic abnormalities, improve blood cell counts, and alleviate symptoms, thereby enhancing quality of life and potentially delaying disease progression.

Lenalidomide as a disease-modifying agent in patients with del(5q) myelodysplastic syndromes: linking mechanism of action to clinical outcomes.Myelodysplastic syndrome.Combination therapy with DNA methyltransferase inhibitors in hematologic malignancies.