What side effects are associated with this type of intervention?

Common treatments for leukemia, such as Enasidenib, dasatinib, nilotinib, and ponatinib, have a range of side effects. Enasidenib can cause differentiation syndrome, hyperbilirubinemia, and leukocytosis. Dasatinib and nilotinib are associated with myelosuppression, cardiovascular events, and gastrointestinal issues. Ponatinib, effective for the T315I mutation, can also lead to cardiovascular complications. Other common side effects across these treatments include fatigue, rash, and liver enzyme abnormalities.

What prior FDA approvals exist?

The FDA has approved several targeted therapies for the treatment of leukemia. Enasidenib, an IDH2 inhibitor, is approved for relapsed or refractory acute myeloid leukemia (AML) with an IDH2 mutation. Other notable approvals include Ivosidenib, an IDH1 inhibitor for AML with an IDH1 mutation, and Venetoclax, a BCL-2 inhibitor used in combination with azacitidine, decitabine, or low-dose cytarabine for newly diagnosed AML in adults who are 75 years or older or have comorbidities precluding intensive induction chemotherapy. Additionally, Midostaurin, a FLT3 inhibitor, is approved for newly diagnosed FLT3-mutated AML in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation. These targeted therapies represent significant advancements in the personalized treatment of leukemia.

What other types of research exist?

Promising novel alternatives to Enasidenib for leukemia patients include: 1) Venetoclax, particularly in combination with low-dose cytarabine (LoDAC) or hypomethylating agents (HMAs) like azacitidine, which has shown efficacy in AML. 2) GD2-CAR T cell therapy for H3K27M-mutated diffuse midline gliomas, which may have potential applications in leukemia. 3) ONC201, a selective DRD2 antagonist, which is being studied for its anticancer properties. These alternatives target different pathways and mechanisms, offering potential new avenues for treatment.

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IDH2 Inhibitor

Enasidenib for Chronic Myelomonocytic Leukemia

Phase 1 & 2

Recruiting

Led By Tian Yi Zhang, MD

Research Sponsored by Stanford University

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 12 months

Awards & highlights

Summary

This trial is testing enasidenib to see if it can help patients with certain blood disorders make more red blood cells and need fewer transfusions. The medication works by boosting the body's natural ability to produce red blood cells. Enasidenib is approved by the US FDA for adults with specific types of blood cancer.

Who is the study for?

This trial is for adults with lower risk myelodysplastic syndrome (MDS) or nonproliferative chronic myelomonocytic leukemia (CMML), without IDH2 mutation, who have anemia symptoms like fatigue and shortness of breath. Participants must not have had certain therapies recently, be able to take oral meds, and use effective contraception if applicable. They can't join if they have other causes of anemia, significant heart disease, less than 3 months life expectancy, active infections including HIV or hepatitis B/C.

What is being tested?

The study tests whether enasidenib mesylate can safely improve anemia and reduce the need for blood transfusions in MDS/CMML patients without the IDH2 mutation. It's a phase 1b/2 trial where everyone gets increasing doses of enasidenib to see how well it works and what effects it has on their body.

What are the potential side effects?

Possible side effects from enasidenib may include digestive issues such as nausea or vomiting, liver problems indicated by changes in blood tests, tiredness, decreased appetite or weight loss. Since this is a dose escalation study assessing safety too, close monitoring will occur to identify any new or unexpected side effects.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 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

Clinical Response: Hematological Improvement - Erythroid (HI-E)

Clinical Response: Hematological Improvement - Neutrophils (HI-N)

Clinical Response: Hematological Improvement - Platelets (HI-P)

+4 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Enasidenib mesylatExperimental Treatment1 Intervention

Participants will self administer the enasidenib orally everyday.

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 leukemia often target specific genetic mutations or dysregulated molecular pathways involved in the disease. For instance, Enasidenib inhibits the mutant IDH2 enzyme, which is crucial in blocking the differentiation of hematopoietic cells, thereby promoting the maturation of malignant cells. This mechanism is significant for leukemia patients as it directly addresses the underlying genetic abnormalities driving the disease, potentially leading to more effective and targeted therapies. Other treatments, such as those targeting DNA methylation or signal transduction pathways, work by correcting the epigenetic and signaling abnormalities that contribute to leukemia progression. These targeted approaches are essential as they offer the potential for more precise and less toxic treatments compared to traditional chemotherapy.

Emerging Epigenetic Therapeutic Targets in Acute Myeloid Leukemia.Molecular targeting in acute myeloid leukemia.AG-221, a First-in-Class Therapy Targeting Acute Myeloid Leukemia Harboring Oncogenic IDH2 Mutations.

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Who is running the clinical trial?

Stanford UniversityLead Sponsor

2,448 Previous Clinical Trials

17,492,346 Total Patients Enrolled

42 Trials studying Leukemia

11,843 Patients Enrolled for Leukemia

Tian Yi ZhangLead Sponsor

Celgene CorporationIndustry Sponsor

444 Previous Clinical Trials

58,189 Total Patients Enrolled

82 Trials studying Leukemia

2,641 Patients Enrolled for Leukemia

Media Library

Enasidenib (IDH2 Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05282459 — Phase 1 & 2

Leukemia Research Study Groups: Enasidenib mesylat

~13 spots leftby Sep 2025

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