CPX-351 + Gilteritinib for Acute Myeloid Leukemia

Recruiting in Palo Alto (17 mi)

+211 other locations

Overseen by Todd M Cooper

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Children's Oncology Group

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This phase III trial compares standard chemotherapy to therapy with liposome-encapsulated daunorubicin-cytarabine (CPX-351) and/or gilteritinib for patients with newly diagnosed acute myeloid leukemia with or without FLT3 mutations. Drugs used in chemotherapy, such as daunorubicin, cytarabine, and gemtuzumab ozogamicin, 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. CPX-351 is made up of daunorubicin and cytarabine and is made in a way that makes the drugs stay in the bone marrow longer and could be less likely to cause heart problems than traditional anthracycline drugs, a common class of chemotherapy drug. Some acute myeloid leukemia patients have an abnormality in the structure of a gene called FLT3. Genes are pieces of DNA (molecules that carry instructions for development, functioning, growth and reproduction) inside each cell that tell the cell what to do and when to grow and divide. FLT3 plays an important role in the normal making of blood cells. This gene can have permanent changes that cause it to function abnormally by making cancer cells grow. Gilteritinib may block the abnormal function of the FLT3 gene that makes cancer cells grow. The overall goals of this study are, 1) to compare the effects, good and/or bad, of CPX-351 with daunorubicin and cytarabine on people with newly diagnosed AML to find out which is better, 2) to study the effects, good and/or bad, of adding gilteritinib to AML therapy for patients with high amounts of FLT3/ITD or other FLT3 mutations and 3) to study changes in heart function during and after treatment for AML. Giving CPX-351 and/or gilteritinib with standard chemotherapy may work better in treating patients with acute myeloid leukemia compared to standard chemotherapy alone.

Research Team

Todd M Cooper

Principal Investigator

Children's Oncology Group

Eligibility Criteria

This trial is for patients under 22 years old newly diagnosed with acute myeloid leukemia (AML), either with or without FLT3 gene mutations. They must have certain levels of cancer cells in their blood or bone marrow and agree to use contraception if of reproductive potential. Excluded are those with specific genetic syndromes, prior significant cancer treatments, pregnant or breastfeeding women, and those not using effective contraception.

Inclusion Criteria

I am at least 5 years old.

I have been recently diagnosed with a type of leukemia called AML.

All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Exclusion Criteria

I am currently pregnant.

I do not have a genetic heart rhythm disorder or congenital heart block.

I have been diagnosed with Shwachman Diamond syndrome.

Treatment Details

Interventions

• CPX-351 (Liposomal Anthracycline and Cytarabine)
• Gilteritinib Fumarate (FLT3 Inhibitor)

Trial Overview The study compares standard chemotherapy against therapy combining CPX-351 (liposome-encapsulated daunorubicin-cytarabine) and/or gilteritinib in AML patients. It aims to see if these new therapies are more effective or cause fewer heart problems than traditional treatments, especially for those with FLT3 mutations.

Participant Groups

Group I: Arm BD Low Risk Group 2 Experimental Treatment 18 Interventions

Arm BD Low Risk Group 2: See Detailed Description. (CLOSED TO ACCRUAL 11/19/2024)

Group II: Arm BD High Risk Group Experimental Treatment 16 Interventions

Arm BD High Risk Group: See Detailed Description. (CLOSED TO ACCRUAL 11/19/2024)

Group III: Arm BC Low Risk Group 2 Experimental Treatment 18 Interventions

Arm BC Low Risk Group 2: See Detailed Description. (CLOSED TO ACCRUAL 11/19/2024)

Group IV: Arm BC High Risk Group Experimental Treatment 16 Interventions

Arm BC High Risk Group: See Detailed Description. (CLOSED TO ACCRUAL 11/19/2024)

Group V: Arm B Low Risk Group 2 Experimental Treatment 15 Interventions

Arm B Low Risk Group 2: See Detailed Description. (CLOSED TO ACCRUAL 11/19/2024)

Group VI: Arm B Low Risk Group 1 Experimental Treatment 15 Interventions

Arm B Low Risk Group 1: See Detailed Description. (CLOSED TO ACCRUAL 11/19/2024)

Group VII: Arm B High Risk Group Experimental Treatment 15 Interventions

Arm B High Risk Group: See Detailed Description. (CLOSED TO ACCRUAL 11/19/2024)

Group VIII: Arm AD Low Risk Group 2 Experimental Treatment 18 Interventions

Arm AD Low Risk Group 2: See Detailed Description.

Group IX: Arm AD High Risk Group Experimental Treatment 17 Interventions

Arm AD High Risk Group: See Detailed Description.

Group X: Arm AC Low Risk Group 2 Experimental Treatment 18 Interventions

Arm AC Low Risk Group 2: See Detailed Description.

Group XI: Arm AC High Risk Group Experimental Treatment 17 Interventions

Arm AC High Risk Group: See Detailed Description.

Group XII: Arm A Low Risk Group 2 Experimental Treatment 17 Interventions

Arm A Low Risk Group 2: See Detailed Description.

Group XIII: Arm A Low Risk Group 1 Experimental Treatment 16 Interventions

Arm A Low Risk Group 1: See Detailed Description. (CLOSED TO ACCRUAL 11/19/2024)

Group XIV: Arm A High Risk Group Experimental Treatment 16 Interventions

Arm A High Risk Group: See Detailed Description.

Who Is Running the Clinical Trial?

Children's Oncology Group

Lead Sponsor

Trials

467

Recruited

241,000+

Dr. Doug Hawkins

Children's Oncology Group

Chief Executive Officer since 2020

MD from University of Washington School of Medicine

Dr. Leo Mascarenhas

Children's Oncology Group

Chief Medical Officer since 2024

MD, MS from Cedars-Sinai Guerin Children’s

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Dr. Douglas R. Lowy

National Cancer Institute (NCI)

Chief Executive Officer since 2023

MD from New York University School of Medicine

Dr. Monica Bertagnolli

National Cancer Institute (NCI)

Chief Medical Officer since 2022

MD from Harvard Medical School