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CPX-351 and Ivosidenib for IDH1-Mutated AML or High-Risk MDS

This study is investigating a combination of two drugs, CPX-351 and ivosidenib, for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) that has a specific genetic change called an IDH1 mutation. CPX-351 is a chemotherapy drug that works to stop cancer cell growth. Ivosidenib may also stop cancer cell growth by blocking certain enzymes. The main goal is to see how many patients respond to this treatment combination. Researchers will also look at the safety of these drugs together. You may be able to join if you are 18 or older, have an IDH1 mutation, and meet certain health criteria. This study plans to enroll 30 participants.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It is a Phase II trial, which typically involves a smaller number of participants (30 planned for this study) to evaluate effectiveness and safety.
What's involved
Participants will receive CPX-351 intravenously (through a vein) on specific days and take ivosidenib by mouth daily. This treatment schedule will involve induction and consolidation phases.
Compensation
Not stated in the trial record.
Follow-up
The study will measure how well the treatment works for up to 3 years.

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NCT04493164

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

Recruiting
PHASE2Ages 18+InterventionalTreatment
M.D. Anderson Cancer Center
~30 participants
Updated 2026-08-25 on ClinicalTrials.gov
What's tested:IvosidenibLiposome-encapsulated Daunorubicin-Cytarabine

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Overall response rate (ORR)
Measured over Up to 3 years

Conditions

Acute Myeloid Leukemia With Gene Mutations
Myelodysplastic Syndrome
Myeloproliferative Neoplasm
Recurrent Acute Myeloid Leukemia
Refractory Acute Myeloid Leukemia

Explore related trials

Acute Myeloid LeukemiaMyelodysplastic SyndromeMyeloproliferative NeoplasmIDH1 Gene

Where it's being run

1 sites across 1 states
Texas1

Study leadership

  • Courtney DiNardo, MD · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center

Who to contact

Courtney DiNardo, MD
[email protected]
713-794-1141
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2
IDH1-R132 mutated disease status as assessed by local laboratory. 2HG-producing IDH1 variants outside of R132 (i.e. R100) may be eligible after discussion with the principal investigator (PI)
Treatment naive or relapsed/refractory AML who are eligible for intensive chemotherapy. Patients with high-risk MDS or MPN (defined as International Prognostic Scoring System Revised \[IPSS-R\] score ≥ 4 or dynamic \[D\]-IPSS ≥ 3) may also be eligible after discussion with the PI
Adequate hepatic function (direct bilirubin ≤ 2 x upper limit of normal (ULN), Alanine aminotransferase (ALT) and/or aspartate aminotransferase (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.
Willing and able to provide informed consent
In the absence of rapidly proliferative disease, the interval from prior treatment to time of initiation will be at least 7 days for cytotoxic or non-cytotoxic (immunotherapy) agents.
Male subjects must agree to refrain from unprotected sex and sperm donation from initial study drug administration until 90 days after the last dose of study drug

Exclusion

Patients who have previously received CPX-351.
Patients with any concurrent uncontrolled clinically significant medical condition including infection, laboratory abnormality, or psychiatric illness, which could place the patient at unacceptable risk of study treatment.
The use of other chemotherapeutic agents or anti-leukemic agents is not permitted during study with the following exceptions (1) intrathecal chemotherapy for prophylactic use or for controlled CNS leukemia. (2) use of hydroxyurea, and/or cytarabine (1 or 2 doses; up to 2 g/m2) for patients with rapidly proliferative disease is allowed before the start of study therapy.
Patients with active graft-versus-host-disease (GVHD) status post stem cell transplant (patients without active GVHD on chronic suppressive immunosuppression and/or phototherapy for chronic skin GVHD are permitted after discussion with the PI).
Patients with any severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications.
Patients with symptomatic congestive heart failure (NYHA Class III or IV), unstable angina, or an ejection fraction \< 45%.
Patients with prior anthracycline exposure of \> 360 mg/m2 daunorubicin (or equivalent), or \> 210 mg/m2 daunorubicin (or equivalent) in patients with prior mediastinal radiation.
QTc interval using Fridericia's formula (QTcF) \> 470 msec. A prolonged QTc interval in the setting of right bundle branch block is permitted after discussion with the PI.
Nursing women, women of childbearing potential (WOCBP) with positive urine or serum pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception
Subjects with a known medical history of progressive multifocal leukoencephalopathy (PML).
Subjects taking strong CYP3A4 inducers are excluded from the study unless they can be transferred to other medications within ≥ 5 half-lives prior to dosing
Patients with a diagnosis of acute promyelocytic leukemia (APL).
Unresolved toxicities \> grade 1 from prior treatment including chemotherapy, targeted therapy, immunotherapy, experimental agents, radiation, or surgery.

What this trial measures

  • Overall response rate (ORR)Up to 3 years

    Defined as complete remission (CR) + complete remission with hematologic recovery (CRh) + complete remission with incomplete blood count recovery (CRi) + morphologic leukemia-free state (MLFS) + and partial remission (PR). Will be assessed based on revised International Working Group (IWG) response criteria for acute myeloid leukemia (AML). Estimated along 95% confidence interval.