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OncoMatch/Clinical Trials/NCT04493164

CPX-351 and Ivosidenib for the Treatment of IDH1 Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Is NCT04493164 recruiting? Yes, currently enrolling (May 2026). This Phase 2 trial studies multiple treatments including Ivosidenib and Liposome-encapsulated Daunorubicin-Cytarabine for acute myeloid leukemia with gene mutations.

Phase 2RecruitingM.D. Anderson Cancer CenterNCT04493164Data as of May 2026

Treatment: Ivosidenib · Liposome-encapsulated Daunorubicin-CytarabineThis phase II trial investigates how well CPX-351 and ivosidenib work in treating patients with acute myeloid leukemia or high-risk myelodysplastic syndrome that has IDH1 mutation. The safety of this drug combination will also be studied. IDH1 is a type of genetic mutation (change). Chemotherapy drugs, such as CPX-351, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Ivosidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. The purpose of this trial is to learn if CPX-351 in combination with ivosidenib can help to control IDH1-mutated acute myeloid leukemia or high-risk myelodysplastic syndrome.

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Extracted eligibility criteria

Cancer type

Acute Myeloid Leukemia

Myelodysplastic Syndrome

Myeloproliferative Neoplasm

Biomarker criteria

Required: IDH1 R132

IDH1-R132 mutated disease status as assessed by local laboratory

Allowed: IDH1 R100

2HG-producing IDH1 variants outside of R132 (i.e. R100) may be eligible after discussion with the principal investigator (PI)

Performance status

ECOG 0–2(Ambulatory, capable of self-care)

Prior therapy

Cannot have received: cpx-351 (cpx-351)

Lab requirements

Kidney function

creatinine clearance ≥ 30 ml/min based on the Cockcroft-Gault equation

Liver function

direct bilirubin ≤ 2 x ULN, ALT and/or AST ≤ 3 x ULN unless deemed to be related to underlying leukemia

Cardiac function

No symptomatic congestive heart failure (NYHA Class III or IV), unstable angina, or an ejection fraction < 45%

Adequate hepatic function (direct bilirubin ≤ 2 x ULN, ALT and/or AST ≤ 3 x ULN unless deemed to be related to underlying leukemia). Adequate renal function including creatinine clearance ≥ 30 ml/min based on the Cockcroft-Gault equation. Patients with symptomatic congestive heart failure (NYHA Class III or IV), unstable angina, or an ejection fraction < 45% [excluded].

Structured fields extracted by AI. May contain errors — verify against the official protocol.

US trial sites

  • M D Anderson Cancer Center · Houston, Texas

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