What side effects are associated with this type of intervention?

Common treatments for Myelodysplastic Syndrome (MDS) include DNA methyltransferase inhibitors like azacitidine and decitabine, and thrombopoietin mimetics like romiplostim and eltrombopag. DNA methyltransferase inhibitors can cause side effects such as cytopenias, gastrointestinal symptoms, and injection site reactions. Thrombopoietin mimetics may lead to increased blast cell counts, potential progression to acute myeloid leukemia (AML), and bone marrow fibrosis. Patients undergoing these treatments should be closely monitored for these adverse effects.

What prior FDA approvals exist?

FDA-approved treatments for Myelodysplastic Syndrome (MDS) include hypomethylating agents like azacitidine and decitabine, which inhibit DNA methylation to reactivate tumor suppressor genes. Thrombopoietin receptor agonists such as romiplostim have been explored to improve platelet counts, though their use is controversial due to potential leukemic transformation. Additionally, lenalidomide is approved for patients with del(5q) MDS, targeting specific genetic abnormalities to manage hematologic complications and improve outcomes.

What other types of research exist?

Promising novel alternatives to CTX-712 for Myelodysplastic Syndrome patients include: 1) KPT-330, a CRM1 inhibitor showing anti-leukemic activity and potential for sensitizing chronic myeloid leukemia to other treatments. 2) CC-90002, an anti-CD47 therapeutic antibody with demonstrated antitumor activity and favorable pharmacokinetic properties in hematological malignancies. Both options are supported by preclinical and early clinical data, making them viable candidates for further consideration in 2024.

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CTX-712 for Acute Myeloid Leukemia and Myelodysplastic Syndrome

Phase 1 & 2

Recruiting

Led By Guillermo Garcia-Manero, MD

Research Sponsored by Chordia 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 measured from date of first dose to 28 days after last dose of ctx-712.

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug called CTX-712 to see if it is safe and effective for patients with certain types of blood cancer that have not responded to other treatments. The study will first determine the best dose and then check how well the drug works and if it is safe.

Who is the study for?

Adults (18+) with relapsed/refractory acute myeloid leukemia or high-risk myelodysplastic syndromes, who have undergone 1-4 prior treatments. Participants must have adequate organ function and blood counts, agree to contraception if of childbearing potential, and not be suffering from severe lung disease or recent major surgery.

What is being tested?

The trial is testing CTX-712's safety and effectiveness in two phases. Phase 1 determines the best dose through a '3+3' method where small groups receive increasing doses. Phase 2 evaluates its therapeutic activity at this established dose in patients with specific types of leukemia.

What are the potential side effects?

While specific side effects for CTX-712 are not listed, common ones for cancer drugs include nausea, fatigue, risk of infection due to low blood cell counts, liver issues indicated by altered blood tests, and potential allergic reactions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ measured from date of first dose to 28 days after last dose of ctx-712.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~measured from date of first dose to 28 days after last dose of ctx-712.

This trial's timeline: 3 weeks for screening, Varies for treatment, and measured from date of first dose to 28 days after last dose of ctx-712. for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Phase 1: The maximum tolerated dose MTD.

Secondary study objectives

Phase 1 and 2: Objective response rate, defined as the proportion of patients achieving a response.

Awards & Highlights

No Placebo-Only Group

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

Trial Design

3Treatment groups

Experimental Treatment

Group I: Phase 2Experimental Treatment1 Intervention

CTX-712 administered at the recommended dose by the expansion cohort

Group II: Dose Expansion CohortExperimental Treatment1 Intervention

Drug: CTX-712 administered at a dose to be determined from the data of dose escalation cohort

Group III: Dose Escalation CohortExperimental Treatment1 Intervention

Drug: CTX-712 administered at 20 mg, 40 mg, 80 mg, 100 mg, 140 mg weekly

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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 hypomethylating agents such as azacitidine and decitabine, which work by inhibiting DNA methyltransferase, leading to gene derepression and reactivation of silenced genes, thereby promoting normal cell differentiation and apoptosis of malignant cells. Thrombopoietin mimetics like romiplostim aim to increase platelet production but have a controversial risk of promoting leukemic transformation. Erythropoiesis-stimulating agents (ESAs) like erythropoietin boost red blood cell production, improving anemia symptoms. These treatments are crucial for managing MDS as they target different aspects of the disease, improving blood counts and reducing transfusion needs, thereby enhancing patients' quality of life.

Decitabine Induces Gene Derepression on Monosomic Chromosomes: In Vitro and In Vivo Effects in Adverse-Risk Cytogenetics AML.Lenalidomide as a disease-modifying agent in patients with del(5q) myelodysplastic syndromes: linking mechanism of action to clinical outcomes.Combination therapy with DNA methyltransferase inhibitors in hematologic malignancies.