CPX-351 vs Intensive Chemotherapy in Patients With de Novo Intermediate or Adverse Risk AML Stratified by Genomics

NCT ID: NCT05260528

Last Updated: 2024-01-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

210 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-05-03

Study Completion Date

2027-10-31

Brief Summary

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The trial is a randomized, open-label phase II study comparing CPX-351 vs conventional intensivechemotherapy in patients with newly diagnosed de novo AML and intermediate- or adverse-risk genetics (according to 2017 ELN criteria)

Detailed Description

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Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy characterized by clonal expansion of myeloid blasts.

Interestingly comparing de novo and stringently defined secondary AMLs occurring after a documented phase of MDS, Lindsley et al. could identify among de novo AMLs a molecular subgroup, termed 'secondary-type AML', defined by mutations in either SRSF2, SF3B1, U2AF1, ZRSR2, ASXL1, EZH2, BCOR and/or STAG2 genes. Among de novo AML patients, 33.3% had secondary-type mutations.

It has been shown that patients older than 60 years of age harboring secondary-type AML, as defined by this 8-gene molecular signature, had inferior outcome to those without 'secondary-type' mutations when treated with conventional 7+3 chemotherapy, combining cytarabine and an anthracycline (ALFA 1200 study). This was notably true among patients with 'intermediate-risk' disease per European LeukemiaNet criteria.

The incidence of 'secondary-type' AML mutations increases with age and with cytogenetic risk category. Notably, roughly 50% of de novo AML patients with intermediate risk older than 50 years of age harbor such secondary-type mutations, New therapeutic options are thus necessary in patients older than 50 years with de novo AML classified adverse risk but also intermediate risk and associated to secondary-type mutation

This study will evaluate the rate of MRD negative remissions with CPX-351 used as induction and consolidation therapy according to its marketing authorization (AMM), as compared to intensive chemotherapy in a population of non-MRC AMLs enriched in secondary-like mutations. In addition,P-gp activity will be explore as a putative biomarker.

Duration of the enrollment period: 36 months Duration of treatment: 6 months Duration of the participation for a patient: 18 months (post randomization) (including approximately 6 months treatment, and 12 months of post-treatment follow up) Overall duration of the study: 58 months including the analysis of the results

Conditions

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Acute Myeloid Leukemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Standard arm

Group Type ACTIVE_COMPARATOR

Cytarabine and Idarubicin

Intervention Type DRUG

Induction 1: Cytarabine 200 mg/m2 i.v. (continuously) d1-7 + Idarubicin 12mg/m2 d1, 2, 3 i.v (60 min)

Induction 2: Cytarabine 1500 mg/m2 i.v. q12h d1-3

Consolidation: Cytarabine 1500 mg/m2 i.v. q12h d1-3

Investigational arm

Group Type EXPERIMENTAL

CPX-315

Intervention Type DRUG

Induction 1:CPX-351 44 mg/m2 daunorubicin / 100 mg/m2 cytarabine i.v. (90 min) d1,3,5

Induction 2: CPX-351 44 mg/m2 daunorubicin / 100 mg/m2 cytarabine i.v. (90 min) d1,3

Consolidation therapy:CPX-351 29 mg/m2 daunorubicin / 65 mg/m2 cytarabine i.v. (90 min) d1,3

Interventions

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Cytarabine and Idarubicin

Induction 1: Cytarabine 200 mg/m2 i.v. (continuously) d1-7 + Idarubicin 12mg/m2 d1, 2, 3 i.v (60 min)

Induction 2: Cytarabine 1500 mg/m2 i.v. q12h d1-3

Consolidation: Cytarabine 1500 mg/m2 i.v. q12h d1-3

Intervention Type DRUG

CPX-315

Induction 1:CPX-351 44 mg/m2 daunorubicin / 100 mg/m2 cytarabine i.v. (90 min) d1,3,5

Induction 2: CPX-351 44 mg/m2 daunorubicin / 100 mg/m2 cytarabine i.v. (90 min) d1,3

Consolidation therapy:CPX-351 29 mg/m2 daunorubicin / 65 mg/m2 cytarabine i.v. (90 min) d1,3

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. De novo AML
2. No MRC-defining cytogenetic lesion
3. No t(15;17), t(8;21), inv(16) or t(16;16)
4. No NPM1 gene mutation
5. No FLT3 mutated AML (FLT3 ITD or TKD)
6. Not previously treated except for short course hydroxyurea in patients presenting with high WBC count and/or tumor symptoms,
7. Age ≥ 50 years,
8. Performance status ≤ 2 (ECOG grading),
9. Patient must have adequate organ function as indicated detailed with laboratory values in the section IV of the protocol
10. Female patient of childbearing potential with a negative serum pregnancy test (β-hCG) within 72 hours prior to receiving the first dose of CPX-351 or 7+3. Female patient who is not actively breastfeeding at the time of study entry.
11. Female patient is either post-menopausal, free from menses for \> 2 years, surgically sterilized or willing to use 2 adequate barrier methods of contraception to prevent pregnancy, or agrees to not become pregnant throughout the study, starting with study screening
12. Male patient agrees to use an adequate method of contraception for the duration of the study. Men should be advised not to father a child while receiving CPX-351 or 7+3 and for 3 months after the last dose of study treatment .
13. Patient is available for periodic blood sampling, study related assessments, and appropriate clinical management at the treating institution for the duration of the study.
14. Patient has the ability to understand and willingness to sign an informed consent form indicating the investigational nature of the study.
15. Patient registered to the French Social Security.

Exclusion Criteria

1. Prior history of documented MDS, MPN or MDS/MPN, tAML
2. Prior history of radiation therapy or chemotherapy for a solid tumor or lymphoma (exceptions to be considered: local radiotherapy for prostate cancer)
3. Patient has active and uncontrolled infection.
4. Patient has uncontrolled intercurrent illness or circumstances that could limit compliance with the study, including but not limited to the following: symptomatic congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmia, pancreatitis, or psychiatric or social conditions that may interfere with patient compliance.
5. Patient is currently participating or has participated in a study with an investigational compound or device within 30 days of initial dosing with study drug.
6. Patient has known human immunodeficiency virus (HIV) infection or HIV-related malignancy.
7. Patient has clinically active hepatitis B or hepatitis C infection.
8. Patient has a known allergy or hypersensitivity to any component of CPX-351, idarubicin or cytarabine.
9. Patient with a "currently active" second malignancy, other than nonmelanoma skin cancer and carcinoma in situ of the cervix, should not be enrolled. Patients are not considered to have a "currently active" malignancy if they have completed therapy for a prior malignancy, are disease free from prior malignancies for \>1 year or are considered by their physician to be at less than 30% risk of relapse.
10. Patients with clinical evidence of CNS leukemia.
11. Cardiac ejection fraction \<50% or considered as abnormal by echocardiography or multi-gated acquisition (MUGA) scan.
12. Patient is pregnant or breastfeeding within the projected duration of the study.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Jazz Pharmaceuticals

INDUSTRY

Sponsor Role collaborator

Acute Leukemia French Association

OTHER

Sponsor Role collaborator

Centre Hospitalier Universitaire de Nice

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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CHU Amiens Picardie site Sud

Amiens, , France

Site Status RECRUITING

CH Avignon

Avignon, , France

Site Status RECRUITING

CHRU Jean Minjoz

Besançon, , France

Site Status RECRUITING

Centre Hospitalier de Béziers

Béziers, , France

Site Status RECRUITING

Hôpital Avicenne APHP

Bobigny, , France

Site Status RECRUITING

Institut d'hématologie de Basse Normandie (IHBN)

Caen, , France

Site Status RECRUITING

Hôpital d'Instruction des Armée (HIA)

Clamart, , France

Site Status RECRUITING

CHU Estaing

Clermont-Ferrand, , France

Site Status RECRUITING

Centre Hospitalier Sud Francilien (CHSF)

Corbeil-Essonnes, , France

Site Status RECRUITING

CHU Henri Mondor

Créteil, , France

Site Status RECRUITING

Centre Hospitalier de Versailles, Site André Mignot

Le Chesnay, , France

Site Status RECRUITING

Hôpital Claude HURIEZ, CHU Lille

Lille, , France

Site Status RECRUITING

CHU de Limoges

Limoges, , France

Site Status RECRUITING

Hoptial de la Conception APHM

Marseille, , France

Site Status RECRUITING

CHR Metz-Thionville Site Mercy

Metz, , France

Site Status RECRUITING

Groupe hospitalier de la région de Mulhouse et Sud-Alsace, Hôpital Emile Muller

Mulhouse, , France

Site Status RECRUITING

Centre Antoine Lacassagne

Nice, , France

Site Status NOT_YET_RECRUITING

CHU de Nice

Nice, , France

Site Status RECRUITING

Institut de cancérologie du Gard

Nîmes, , France

Site Status RECRUITING

CHR Orléans

Orléans, , France

Site Status RECRUITING

Hopital Necker

Paris, , France

Site Status RECRUITING

Hôpital de la Pitié Salpêtrière

Paris, , France

Site Status RECRUITING

Hôpital Saint-Antoine

Paris, , France

Site Status RECRUITING

Hôpital Saint-Louis

Paris, , France

Site Status RECRUITING

Hopital Lyon Sud

Pierre-Bénite, , France

Site Status RECRUITING

CH de Roubaix

Roubaix, , France

Site Status RECRUITING

Centre Henri Becquerel

Rouen, , France

Site Status RECRUITING

CHU de Saint Etienne

Saint-Priest-en-Jarez, , France

Site Status RECRUITING

Hopital Bretonneau

Tours, , France

Site Status RECRUITING

Institut Gustave Roussy

Villejuif, , France

Site Status NOT_YET_RECRUITING

Countries

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France

Central Contacts

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valerie Foussat

Role: CONTACT

04 92 03 63 77

Facility Contacts

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Delphine Lebon

Role: primary

Safia Chebrek

Role: primary

Yohan Desbrosses

Role: primary

Alain Saad

Role: primary

Thorsten Braun

Role: primary

Sylvain Chantepie

Role: primary

Pierre Arnautou

Role: primary

Romain Guieze

Role: primary

Stéphanie Haiat

Role: primary

Cécile Pautas

Role: primary

Juliette Lambert

Role: primary

Celine Berthon

Role: primary

Pascal Turlure

Role: primary

Regis Costello

Role: primary

Houria Debarri

Role: primary

Mario Ojeda-Uribe

Role: primary

Lauris Gastaud

Role: primary

Thomas Cluzeau, MD

Role: primary

Samy Chraibi

Role: primary

Diana Carp

Role: primary

Ambroise Marcais

Role: primary

Madalina Uzunov

Role: primary

Ollivier Legrand

Role: primary

Florence Rabian

Role: primary

Mael Heiblig

Role: primary

Isabelle Plantier

Role: primary

Emilie Lemasle-Hue

Role: primary

Emmanuelle Tavernier

Role: primary

Alban Villate

Role: primary

Sephane De Botton

Role: primary

Other Identifiers

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21-PP-26

Identifier Type: -

Identifier Source: org_study_id

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