Actively Recruiting

Phase 2
Age: 18Years +
All Genders
NCT04493164

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

Led by M.D. Anderson Cancer Center · Updated on 2025-11-21

30

Participants Needed

1

Research Sites

282 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This 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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Eastern Cooperative Oncology Group (ECOG) performance status of 64 2
  • IDH1-R132 mutated disease status confirmed by local laboratory
  • Treatment naive or relapsed/refractory AML patients eligible for intensive chemotherapy
  • Patients with high-risk MDS or myeloproliferative neoplasm with IPSS-R score 64 4 or D-IPSS 64 3 may be eligible after discussion
  • Adequate liver function with direct bilirubin 64 2 times upper limit of normal, ALT and/or AST 64 3 times upper limit of normal unless related to leukemia
  • Adequate kidney function with creatinine clearance 64 30 ml/min based on Cockcroft-Gault equation
  • Willing and able to give informed consent
  • At least 7 days interval from prior treatment to study start unless rapidly proliferative disease
  • Male subjects agree to avoid unprotected sex and sperm donation from first dose until 90 days after last dose
Not Eligible

You will not qualify if you...

  • Previous treatment with CPX-351
  • Uncontrolled serious medical conditions including infection, lab abnormalities, or psychiatric illness
  • Use of other chemotherapy or anti-leukemic agents during the study except specified exceptions
  • Active graft-versus-host disease after stem cell transplant unless controlled and approved
  • Severe gastrointestinal or metabolic conditions interfering with oral medication absorption
  • Symptomatic congestive heart failure (NYHA Class III or IV), unstable angina, or ejection fraction less than 45%
  • Prior anthracycline exposure over specified dose limits
  • QTc interval over 470 msec unless approved
  • Nursing women or women of childbearing potential who are pregnant or unwilling to use contraception
  • History of progressive multifocal leukoencephalopathy (PML)
  • Use of strong CYP3A4 inducers unless switched prior to dosing
  • Diagnosis of acute promyelocytic leukemia (APL)
  • Unresolved toxicities greater than grade 1 from prior treatments

AI-Screening

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Trial Site Locations

Total: 1 location

1

M D Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

C

Courtney DiNardo, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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