Study on CPX-351 and Ivosidenib for Treating IDH1 Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Sponsor: M.D. Anderson Cancer Center

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT04493164

Provider View

This clinical trial is exploring how well the combination of CPX-351 and ivosidenib works in treating patients with acute myeloid leukemia or high-risk myelodysplastic syndrome that has a specific genetic change called IDH1 mutation. The study also looks at the safety of this drug combination.

Patient Parameters

Program Overview

This phase II study is testing a combination of two drugs, CPX-351 and ivosidenib, to see if they can effectively treat patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) that have a specific genetic mutation known as IDH1. CPX-351 is a type of chemotherapy that works by stopping cancer cells from growing and spreading. Ivosidenib is a drug that may block certain enzymes needed for cancer cell growth. The study aims to find out if this combination can help control the disease and to assess the safety of using these drugs together.

Description

The main goal of this study is to determine how well the combination of CPX-351 and ivosidenib works in patients with IDH1-mutated acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). Researchers will look at the overall response rate, which includes different levels of remission and response to the treatment.

Secondary goals include assessing the safety of the drug combination and measuring how long patients respond to the treatment, as well as their overall survival.

The study will also explore additional factors, such as minimal residual disease and specific biomarkers that might predict how well the treatment works or if there is resistance.

Treatment Plan:

  • Induction Phase: Patients receive CPX-351 through an IV on days 1, 3, and 5, and take ivosidenib by mouth daily for 28 days. If complete remission is not achieved, a second cycle may be given.

  • Consolidation Phase: Patients who achieve remission receive CPX-351 on days 1 and 3, and ivosidenib daily for 28 days. This cycle can be repeated up to two times.

  • Maintenance Phase: Patients continue taking ivosidenib daily for up to two years if the disease does not progress and side effects are manageable. Those benefiting from the treatment may continue beyond two years after consulting with the study doctor.

After completing the treatment, patients will have follow-up visits at 30 days, then monthly for three years.

Eligibility Criteria

Inclusion Criteria

  • Must have a performance status score of 0 to 2, indicating full activity or some restriction in strenuous activities.
  • Must have the IDH1-R132 mutation confirmed by a local lab. Other IDH1 variants may be eligible after discussion with the study doctor.
  • Must be newly diagnosed or have relapsed/refractory AML eligible for intensive chemotherapy. High-risk MDS or MPN patients may also be eligible.
  • Must have adequate liver function and kidney function.
  • Must be willing and able to provide informed consent.
  • Must have at least a 7-day gap from prior treatment to starting the study, unless the disease is rapidly progressing.
  • Male participants must agree to use effective contraception and avoid sperm donation during the study and for 90 days after the last dose.

Exclusion Criteria

  • Cannot have previously received CPX-351.
  • Cannot have uncontrolled medical conditions, infections, or psychiatric illnesses that pose a risk during the study.
  • Cannot use other chemotherapy or anti-leukemic agents during the study, except for specific exceptions like intrathecal chemotherapy or hydroxyurea for rapidly growing disease.
  • Cannot have active graft-versus-host disease after a stem cell transplant.
  • Cannot have severe gastrointestinal or metabolic conditions affecting drug absorption.
  • Cannot have severe heart conditions or a low ejection fraction.
  • Cannot have had high doses of certain chemotherapy drugs in the past.
  • Cannot have a prolonged QTc interval unless approved by the study doctor.
  • Cannot be pregnant, breastfeeding, or unwilling to use effective contraception.
  • Cannot have a history of progressive multifocal leukoencephalopathy (PML).
  • Cannot take strong CYP3A4 inducers unless they can switch to other medications before starting the study.
  • Cannot have acute promyelocytic leukemia (APL).
  • Cannot have unresolved severe side effects from previous treatments.

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