A Study Evaluating the Safety and Efficacy of Idasanutlin in Combination With Cytarabine and Daunorubicin in Patients Newly Diagnosed With Acute Myeloid Leukemia (AML) and the Safety and Efficacy of Idasanutlin in the Maintenance of First AML Complete Remission

NCT ID: NCT03850535

Last Updated: 2022-06-21

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-03-25

Study Completion Date

2020-09-10

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This Phase Ib/II, open-label, multicenter, non-randomized study will evaluate the safety, efficacy, and pharmacokinetics of idasanutlin when it is given in combination with cytarabine and daunorubicin in induction, in combination with cytarabine in consolidation, and as a single agent in maintenance for treating participants with acute myeloid leukemia (AML).

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Acute Myeloid Leukemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Dose-Escalation Phase

Participants with newly diagnosed, previously untreated, favorable or intermediate risk AML (according to European LeukemiaNet \[ELN\] 2017 criteria) will undergo the treatment sequence of induction, consolidation, and maintenance. For induction, participants will be treated with idasanutlin plus cytarabine and daunorubicin. At the investigator's discretion for consolidation, either participants will be treated with idasanutlin and cytarabine or they will undergo Allo-HSCT. For maintenance, participants will be treated with single-agent idasanutlin.

Group Type EXPERIMENTAL

Idasanutlin

Intervention Type DRUG

Participants will self-administer idasanutlin tablets by mouth (PO) once daily (QD) for 5 days of each 28-day treatment cycle for up to 2 cycles of induction, up to 4 cycles of consolidation, and 12 cycles of maintenance, according to the study's protocol.

Cytarabine

Intervention Type DRUG

Cytarabine will be administered for 7 days of each 28-day cycle for up to 2 cycles of induction treatment at a dose of 200 milligrams per meter squared (mg/m\^2) of body surface area, once daily (QD) by intravenous (IV) infusion. At the investigator's discretion, cytarabine will also be administered as part of chemotherapy consolidation at a dose of 1.5 g/m\^2 QD as IV infusion for 5 days of each 28-day cycle and for up to 4 cycles of treatment.

Daunorubicin

Intervention Type DRUG

Daunorubicin will be administered for 3 days of each 28-day cycle for up to 2 cycles of induction treatment at a dose of 60 mg/m\^2 QD as IV infusion.

Allogeneic Hematopoietic Stem Cell Transplant (Allo-HSCT)

Intervention Type PROCEDURE

After induction treatment, participants who achieve remission will either receive chemotherapy consolidation treatment or undergo Allo-HSCT (as per local guidelines) at the investigator's discretion.

Post-Consolidation Phase

Participants who are idasanutlin treatment-naive, had received induction and chemotherapy consolidation for AML outside of the study, and were in minimal residual disease (MRD)-positive remission after induction will be enrolled in this cohort to receive maintenance treatment with single-agent idasanutlin.

Group Type EXPERIMENTAL

Idasanutlin

Intervention Type DRUG

Participants will self-administer idasanutlin tablets by mouth (PO) once daily (QD) for 5 days of each 28-day treatment cycle for up to 2 cycles of induction, up to 4 cycles of consolidation, and 12 cycles of maintenance, according to the study's protocol.

Expansion Phase: Favorable/Intermediate-Risk AML

Participants with newly diagnosed, previously untreated, favorable or intermediate risk AML (according to ELN 2017 criteria) will undergo the treatment sequence of induction, consolidation, and maintenance. For induction, participants will be treated with idasanutlin (at the recommended Phase 2 dose) plus cytarabine and daunorubicin. At the investigator's discretion for consolidation, either participants will be treated with idasanutlin and cytarabine or they will undergo Allo-HSCT. For maintenance, participants will be treated with single-agent idasanutlin.

Group Type EXPERIMENTAL

Idasanutlin

Intervention Type DRUG

Participants will self-administer idasanutlin tablets by mouth (PO) once daily (QD) for 5 days of each 28-day treatment cycle for up to 2 cycles of induction, up to 4 cycles of consolidation, and 12 cycles of maintenance, according to the study's protocol.

Cytarabine

Intervention Type DRUG

Cytarabine will be administered for 7 days of each 28-day cycle for up to 2 cycles of induction treatment at a dose of 200 milligrams per meter squared (mg/m\^2) of body surface area, once daily (QD) by intravenous (IV) infusion. At the investigator's discretion, cytarabine will also be administered as part of chemotherapy consolidation at a dose of 1.5 g/m\^2 QD as IV infusion for 5 days of each 28-day cycle and for up to 4 cycles of treatment.

Daunorubicin

Intervention Type DRUG

Daunorubicin will be administered for 3 days of each 28-day cycle for up to 2 cycles of induction treatment at a dose of 60 mg/m\^2 QD as IV infusion.

Allogeneic Hematopoietic Stem Cell Transplant (Allo-HSCT)

Intervention Type PROCEDURE

After induction treatment, participants who achieve remission will either receive chemotherapy consolidation treatment or undergo Allo-HSCT (as per local guidelines) at the investigator's discretion.

Expansion Phase: High-Risk AML

Participants with newly diagnosed, previously untreated, high-risk AML (defined as adverse risk according to ELN 2017 criteria, and secondary AML) will undergo the treatment sequence of induction, consolidation, and maintenance. For induction, participants will be treated with idasanutlin (at the recommended Phase 2 dose) plus cytarabine and daunorubicin. At the investigator's discretion for consolidation, either participants will be treated with idasanutlin and cytarabine or they will undergo Allo-HSCT. For maintenance, participants will be treated with single-agent idasanutlin.

Group Type EXPERIMENTAL

Idasanutlin

Intervention Type DRUG

Participants will self-administer idasanutlin tablets by mouth (PO) once daily (QD) for 5 days of each 28-day treatment cycle for up to 2 cycles of induction, up to 4 cycles of consolidation, and 12 cycles of maintenance, according to the study's protocol.

Cytarabine

Intervention Type DRUG

Cytarabine will be administered for 7 days of each 28-day cycle for up to 2 cycles of induction treatment at a dose of 200 milligrams per meter squared (mg/m\^2) of body surface area, once daily (QD) by intravenous (IV) infusion. At the investigator's discretion, cytarabine will also be administered as part of chemotherapy consolidation at a dose of 1.5 g/m\^2 QD as IV infusion for 5 days of each 28-day cycle and for up to 4 cycles of treatment.

Daunorubicin

Intervention Type DRUG

Daunorubicin will be administered for 3 days of each 28-day cycle for up to 2 cycles of induction treatment at a dose of 60 mg/m\^2 QD as IV infusion.

Allogeneic Hematopoietic Stem Cell Transplant (Allo-HSCT)

Intervention Type PROCEDURE

After induction treatment, participants who achieve remission will either receive chemotherapy consolidation treatment or undergo Allo-HSCT (as per local guidelines) at the investigator's discretion.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Idasanutlin

Participants will self-administer idasanutlin tablets by mouth (PO) once daily (QD) for 5 days of each 28-day treatment cycle for up to 2 cycles of induction, up to 4 cycles of consolidation, and 12 cycles of maintenance, according to the study's protocol.

Intervention Type DRUG

Cytarabine

Cytarabine will be administered for 7 days of each 28-day cycle for up to 2 cycles of induction treatment at a dose of 200 milligrams per meter squared (mg/m\^2) of body surface area, once daily (QD) by intravenous (IV) infusion. At the investigator's discretion, cytarabine will also be administered as part of chemotherapy consolidation at a dose of 1.5 g/m\^2 QD as IV infusion for 5 days of each 28-day cycle and for up to 4 cycles of treatment.

Intervention Type DRUG

Daunorubicin

Daunorubicin will be administered for 3 days of each 28-day cycle for up to 2 cycles of induction treatment at a dose of 60 mg/m\^2 QD as IV infusion.

Intervention Type DRUG

Allogeneic Hematopoietic Stem Cell Transplant (Allo-HSCT)

After induction treatment, participants who achieve remission will either receive chemotherapy consolidation treatment or undergo Allo-HSCT (as per local guidelines) at the investigator's discretion.

Intervention Type PROCEDURE

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

RO5503781 RG7388

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Eastern Cooperative Oncology Group (ECOG) performance status ≤2
* Adequate hepatic and renal function
* For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, and agreement to refrain from donating eggs with a failure rate of \<1% per year during the treatment period and for at least 6 months after the final dose of idasanutlin, cytarabine, or daunorubicin. Women must refrain from donating eggs during this same period.
* For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive, measures, and agreement to refrain from donating sperm that together result in a failure rate of \<1% per year during the treatment period and for 6 months after the final dose of idasanutlin, cytarabine, or daunorubicin. Men must refrain from donating sperm during this same period.


\- Documented/confirmed newly diagnosed acute myeloid leukemia (AML) not previously treated according to World Health Organization (WHO)


\- Documented/confirmed AML according to WHO in remission after induction, within 21 days of end of last chemotherapy consolidation cycle, and were minimum residual disease (MRD) positive at the end of induction as per local laboratory assessment

Exclusion Criteria

* Clinical evidence of central nervous system (CNS) leukemia
* Any Grade ≥2 non-hematologic toxicities prior to starting therapy
* Current treatment with any other investigational or commercial agents or therapies administered with the intention to treat their malignancy with the exception of hydroxyurea (HU) or 6-mercaptopurine (6-MP)
* Treatment-related AML
* Acute promyelocytic leukemia
* History of other malignancy that could affect compliance with the protocol or interpretation of results
* Any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study, impair the ability of the investigator to evaluate the patient, or impair the patient's ability to complete the study
* Echocardiogram (ECHO) or multiple-gated acquisition (MUGA) scan showing ejection fraction ≤40%
* Non-malignant medical illnesses that are uncontrolled or whose control may be jeopardized by this study treatment, such as hereditary coagulation disorders, insulin-dependent diabetes mellitus not optimally controlled with medical management (e.g., presence of ketoacidosis), or active GI conditions affecting absorption
* Infection considered by the investigator to be clinically uncontrolled or of unacceptable risk to the patient upon the induction of neutropenia, that is, patients who are or should be on antimicrobial agents for the treatment of active infection
* Febrile patients within 72 hours of study treatment initiation
* Patients with a history of active or chronic infectious hepatitis unless serology demonstrates clearance of infection
* Patients who are unable to interrupt treatment with moderate to strong CYP2C8 inducers and inhibitors
* Patients who are unable to temporarily interrupt treatment with oral or parenteral anticoagulants/anti-platelet agents during treatment phase
* Patients who have a history of clinically significant liver cirrhosis
* Patients with extramedullary AML with no evidence of systemic involvement
* Pregnant or breastfeeding patients
* Known history of HIV-positive status
* Patients who might refuse to receive blood products and/or have a hypersensitivity to blood products
* Prior treatment with an MDM2 antagonist
* Patients with clinically relevant QTc prolongation, a family history of long QT syndrome


\- Adverse risk patients as per European LeukemiaNet (ELN) 2017 criteria


* Any ongoing Grade ≥2 hematologic adverse events prior to starting therapy
* Previous hematopoietic stem cell transplant (HSCT)


\- Secondary AML, defined as AML evolving from antecedent hematologic disorder (AHD)
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Hoffmann-La Roche

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

UCLA Jonsson Comprehensive Cancer Center

Los Angeles, California, United States

Site Status

Sarah Cannon Research Institute at HealthONE

Denver, Colorado, United States

Site Status

Robert H. Lurie Comprehensive Cancer Center of Northwestern University

Chicago, Illinois, United States

Site Status

University of Iowa Hospitals and Clinics; Investigational Drug Services

Iowa City, Iowa, United States

Site Status

University of Kansas Clinical Research Center; Clinical Trials Office

Fairway, Kansas, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

State University of NY

Brooklyn, New York, United States

Site Status

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status

Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, United States

Site Status

The Ohio State University Comprehensive Cancer Center

Columbus, Ohio, United States

Site Status

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, United States

Site Status

The University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status

The Alfred Hospital

Prahan, Victoria, Australia

Site Status

Royal Perth Hospital

Perth, Western Australia, Australia

Site Status

Centre Léon Bérard

Lyon, , France

Site Status

Institut Paoli Calmettes

Marseille, , France

Site Status

CHU de Nantes - Hotel Dieu

Nantes, , France

Site Status

Hôpital Saint-Louis

Paris, , France

Site Status

Institut Universitaire du Cancer de Toulouse-Oncopole

Toulouse, , France

Site Status

Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori (IRST); Onco-Ematologia

Meldola, Emilia-Romagna, Italy

Site Status

ASST Papa Giovanni XXIII; Dipartimento Interaziendale di Farmacia Clinica

Bergamo, Lombardy, Italy

Site Status

Hospital de la Santa Creu i Sant Pau

Barcelona, , Spain

Site Status

Hospital Universitario Vall d'Hebron

Barcelona, , Spain

Site Status

Hospital Universitario Fundacion Jimenez Diaz.

Madrid, , Spain

Site Status

Hospital Universitario Virgen del Rocío; Servicio de Neuropediatra

Seville, , Spain

Site Status

Hospital Universitari i Politècnic La Fe

Valencia, , Spain

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Australia France Italy Spain

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2018-002964-25

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GO40800

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.