What side effects are associated with this type of intervention?

Common treatments for Acute Myeloid Leukemia (AML) include novel therapeutic agents like BGB-11417 and DNA methyltransferase inhibitors such as Azacitidine. Known side effects of Azacitidine include cytopenias (low blood cell counts), febrile neutropenia (fever with low white blood cell count), thrombocytopenia (low platelet count), anemia, and gastrointestinal symptoms like nausea, vomiting, diarrhea, and constipation. When combined with other agents like Venetoclax, additional side effects can include infections, hypokalemia (low potassium levels), and peripheral edema. Novel agents like BGB-11417 may have unique side effects that are still being studied, but they often share similar profiles with other targeted therapies, including fatigue, gastrointestinal disturbances, and hematologic toxicities.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of Acute Myeloid Leukemia (AML) that are similar to BGB-11417 and Azacitidine. Azacitidine itself is an FDA-approved DNA methyltransferase inhibitor used in AML treatment. Venetoclax, another FDA-approved drug, is a BCL-2 inhibitor often used in combination with hypomethylating agents like Azacitidine for AML. Additionally, Decitabine, another DNA methyltransferase inhibitor, is approved for AML treatment. These drugs have shown efficacy in improving survival and response rates in AML patients, particularly those who are not candidates for intensive chemotherapy.

What other types of research exist?

Promising alternatives for AML treatment include: 1) Venetoclax combined with Azacitidine, which has shown efficacy in patients with poor-risk cytogenetics and TP53 mutations. 2) IDH1 inhibitors like BAY1436032 combined with Azacitidine, particularly for patients with IDH1 mutations. 3) Decitabine combined with Sapacitabine, which has shown comparable outcomes to Decitabine alone in older patients unsuitable for intensive therapy. 4) Azacitidine combined with Gilteritinib, which has demonstrated a higher overall response rate compared to Azacitidine plus placebo.

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Epigenetic Modifying Agent

BGB-11417 for Acute Myeloid Leukemia and Myelodysplastic Syndrome

Phase 1 & 2

Recruiting

Research Sponsored by BeiGene

Eligibility Criteria Checklist

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

Must have

Adequate organ function defined as: Creatinine clearance ≥ 50 milliliters/minute (mL/min) (or between 30 and 49 mL/min in unfit AML cohort); Adequate liver function; Life expectancy of > 12 weeks; Ability to comply with the requirements of the study.

Confirmed diagnosis of one of the following by 2016 World Health Organization criteria: AML, nonacute promyelocytic leukemia; MDS; MDS/MPN; Eastern Cooperative Oncology Group performance status of 0 to 2.

Must not have

Known central nervous system involvement by leukemia.

A diagnosis of acute promyelocytic leukemia.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up approximately 24 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new drug, BGB-11417, alone and with azacitidine, in patients with certain blood cancers to see if it is safe and effective, and to find the best dose.

Who is the study for?

This trial is for adults with certain blood disorders like AML, MDS, or MDS/MPN. They should be relatively active (able to care for themselves), have kidneys and liver working well, and expected to live more than 12 weeks. People can't join if they've had leukemia in the brain, certain other cancers in the last 2 years, specific blood diseases including myelofibrosis or polycythemia vera, or previous treatments with similar drugs.

What is being tested?

The study tests BGB-11417 alone and combined with azacitidine to find a safe dose and see how effective it is against acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). Posaconazole is also used as part of the treatment regimen.

What are the potential side effects?

Possible side effects include nausea, vomiting, diarrhea; low blood cell counts leading to increased infection risk; fatigue; liver issues; kidney problems. The exact side effects will depend on individual reactions to BGB-11417 and azacitidine.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with AML, MDS, or MDS/MPN and can care for myself.

Exclusion Criteria

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

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My leukemia has spread to my brain or spinal cord.

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I have been diagnosed with acute promyelocytic leukemia.

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I have a history of certain blood disorders or leukemia, with or without a specific genetic change.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ approximately 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~approximately 24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and approximately 24 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

Part 3 AML Cohort: Complete Remission (CR) Plus CR With Partial Hematologic Recovery (CRh) Rate

Part 3 MDS Cohort: Modified Overall Response (mOR) Rate

Secondary study objectives

Part 3 AML (Treated with Monotherapy): Complete Response + Morphologic complete Remission with Partial Hematologic Recovery

Part 3 AML Cohort: CR With Incomplete Hematologic Recovery (CRi) Rate

Part 3 AML Cohort: Duration Of Response (DOR)

+12 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

4Treatment groups

Experimental Treatment

Group I: Parts 1 and 2: MDS CohortsExperimental Treatment2 Interventions

Participants with MDS will receive BGB-11417 and azacitidine on a 28-day cycle.

Group II: Parts 1 and 2: AML CohortsExperimental Treatment2 Interventions

Participants with AML will receive BGB-11417 and azacitidine on a 28-day cycle.

Group III: Part 3: AML and MDS CohortsExperimental Treatment3 Interventions

Participants with AML and MDS will receive BGB-11417 and azacitidine on a 28-day cycle. A subset of the participants will receive a modified second cycle of treatment to explore drug-drug interactions (DDI) with posaconazole.

Group IV: Part 3: AML and MDS CohortExperimental Treatment1 Intervention

Participants with MDS and R/R AML (China only) will receive BGB-11417 on a 28-day cycle.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Posaconazole

2018

Completed Phase 4

~4080

Azacitidine

2012

Completed Phase 3

~1440

0 / 5Eligibility 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

The most common treatments for Acute Myeloid Leukemia (AML) include novel therapeutic agents and DNA methyltransferase inhibitors. Novel agents like BGB-11417 often target specific molecular pathways or mutations within leukemia cells, aiming to inhibit their growth and survival. DNA methyltransferase inhibitors, such as azacitidine, work by reversing abnormal DNA methylation patterns that silence tumor suppressor genes, thereby restoring normal cell function and promoting cancer cell death. These treatments are crucial for AML patients as they offer targeted approaches that can be more effective and potentially less toxic than traditional chemotherapy, improving outcomes and quality of life.

Epigenetic deregulation in myeloid malignancies.Role of epigenetic in leukemia: From mechanism to therapy.Emerging Epigenetic Therapeutic Targets in Acute Myeloid Leukemia.