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Comparison Between Two Dose Levels of Daunorubicin and Between One vs. Two Induction Cycles for Adult Patients With AML

Phase 3
Completed
Conditions
Leukemia, Myelocytic, Acute
Interventions
Drug: study part 1 - dose daunorubicin
Procedure: induction cycles
Registration Number
NCT02140242
Lead Sponsor
Technische Universität Dresden
Brief Summary

The proposed trial will address two clinically important questions for younger patients with newly diagnosed acute myeloid leukemia (AML): the optimal dose of daunorubicin in induction therapy and the necessity of a second induction cycle in patients with a good response after the first induction. The primary endpoint is the rate of good responders. Secondary outcomes will be relapse-free survival, overall survival and minimal residual disease kinetics. Patients will be recruited in about 40 treatment centers of the Study Alliance Leukemia study group over a period of 40 months. The results will be of great clinical relevance: First, the study could facilitate the establishment or confirmation of the optimal daunorubicin dose.

Detailed Description

In the first part of the trial, patients will be randomly assigned to receive either 90 mg/m2 or 60 mg/m2 daunorubicin in the first induction cycle in addition to standard dosed cytarabine. Assuming a superiority of 90 mg/m2, 436 patients will be recruited. In the second part of the trial, good responders will be randomized to receive either a second or no further induction cycle. Assuming a non-inferiority of the single induction regarding the rate of complete remissions, a number of 360 patients will be included in the second part. Furthermore, in case of a non-inferiority of single versus double induction in good responders, about half of all younger AML patients could be spared a second induction cycle, leading to a reduction in treatment-related mortality, fewer days spent in hospital and improved quality of life.

As a result of the preplanned interim analysis of part I, the sponsor decided to suspend randomization in trial part I and to offer all patients the standard dose of 60 mg/m2 daunorubicin in both induction cycles (part I and II of the trial). Because of this an Amendment was sent to and approved by regulatories and ethics comitee.

The inclusion age was raised to 65 years based on the current German treatment guidelines in which patients up to the age of 65 are considered eligible for intensive induction chemotherapy with DA60 \[Onkopedia-Leitlinie 2017\].

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
721
Inclusion Criteria
  • Newly diagnosed AML other than acute promyelocytic leukemia (APL) according to WHO criteria, i.e. bone marrow aspirate or biopsy must contain ≥20% blasts of all nucleated cells or differential blood count must contain ≥20% blasts. In acute erythroid leukemia, ≥20% blasts in all non-erythroid bone marrow cells. In AML defined by cytogenetic aberrations, the rate of blasts may be <20%. Secondary AMLs are eligible for inclusion.

  • Age 18- inkl.65 years

  • Eastern Cooperative Oncology Group (ECOG) performance status 0-2

  • Adequate liver and renal function as assessed by the following laboratory requirements to be conducted within 7 days prior to screening:

    • Total bilirubin ≤ 1.5 times the upper limit of normal
    • alanine transaminase (ALT) and aspartate transaminase (AST) ≤ 2.5 times upper limit of normal
    • Creatinine ≤ 1.5 times upper limit of normal
Exclusion Criteria
  • Adequate cardiac function, i.e. left ventricular ejection fraction (LVEF) of ≥ 50% as assessed by transthoracic two-dimensional echocardiography ("M Mode") or multiple gated acquisition scan (MUGA scan)

  • Signed informed consent

  • Women must fulfill at least one of the following criteria in order to be eligible for trial inclusion:

    • Post-menopausal (12 months of natural amenorrhea or 6 months of amenorrhea with Serum follicle stimulating hormone (FSH) > 40 U/ml)
    • Postoperative (i.e. 6 weeks) after bilateral ovariectomy with or without hysterectomy
    • Continuous and correct application of a contraception method with a Pearl Index of <1% (e.g. implants, depots, oral contraceptives, intrauterine device - IUD).
    • Sexual abstinence
    • Vasectomy of the sexual partner

Exclusion criteria:

  • Patients who are not eligible for standard chemotherapy as assessed by the treating physician
  • Central nervous system manifestation of AML
  • Cardiac disease: i.e. heart failure New York Heart Association (NYHA) III or IV; unstable coronary artery disease (MI more than 6 months prior to study entry is permitted); serious cardiac ventricular arrhythmias requiring anti-arrhythmic therapy
  • Patients undergoing renal dialysis
  • Chronic pulmonary disease with clinical relevant hypoxia
  • Known HIV or Hepatitis infection
  • Uncontrolled active infection
  • Medical conditions other than AML with an estimated life expectancy below 6 months
  • Previous treatment of AML except hydroxyurea up to 5 days
  • Relapsed or primary refractory AML
  • Acute promyelocytic leukemia
  • Previous anthracycline-containing chemotherapy
  • Treatment with any known non-marketed drug substance or experimental therapy within 4 weeks prior to enrollment
  • Incapability of understanding purpose and possible consequences of the trial
  • Pregnant or breastfeeding women
  • Evidence suggesting that the patient is not likely to follow the study protocol (e.g. lacking compliance)

Study & Design

Study Type
INTERVENTIONAL
Study Design
FACTORIAL
Arm && Interventions
GroupInterventionDescription
daunorubicin 60 mg/m2study part 1 - dose daunorubicinstudy part 1 - dose daunorubicin standard dose daunorubicin in induction 1 (60 mg/m2) on days 3-5
Double inductioninduction cyclesstudy part 2: induction cycles double induction (only patients with good response)
Single inductioninduction cyclesstudy part 2: induction cycles single induction (only patients with good response)
Primary Outcome Measures
NameTimeMethod
response rate after first inductionday 15

To investigate whether a higher dose of daunorubicin in induction chemotherapy leads to an increase in hematological good responders defined as having \<5% myeloid blasts on day 15 after start of induction therapy.

Rate complete remissionsday 35 after final induction

To investigate whether the rate of complete remissions (CR) after single induction is similar to that after double induction in patients with good response to induction I.

Secondary Outcome Measures
NameTimeMethod
rate cytogenetic and molecular complete remissionsday 35

To investigate whether a higher dose of daunorubicin in induction chemotherapy will lead to an increase in cytogenetic and molecular complete remissions.

event-free survival (EFS)5 years

To investigate whether a higher dose of daunorubicin will lead to improved event-free survival (EFS), relapse-free survival (RFS) and overall survival (OS). To investigate whether EFS, RFS and OS are similar after single versus double induction in patients with good response to induction I.

relapse-free survival (RFS)5 years

To investigate whether a higher dose of daunorubicin will lead to improved event-free survival (EFS), relapse-free survival (RFS) and overall survival (OS). To investigate whether EFS, RFS and OS are similar after single versus double induction in patients with good response to induction I.

overall survival (OS)5 years

To investigate whether a higher dose of daunorubicin will lead to improved event-free survival (EFS), relapse-free survival (RFS) and overall survival (OS). To investigate whether EFS, RFS and OS are similar after single versus double induction in patients with good response to induction I.

Correlation between Minimal Residual Disease (MRD) and EFS, RFS, OSday 35

To correlate the level of cytogenetic and molecular minimal residual disease after induction treatment with survival outcomes EFS, RFS and OS.

Rate of induction deathsday 60

Rate of induction deaths (until day 60 or beginning of consolidation treatment - whichever occurs first)

Incidence of serious infectious complicationsday 35

Incidence of serious infectious complications Grades 3-4 (Common Toxicity Criteria for Adverse Effects (CTCAE) V4.0

Sonographic cardiac left ventricular ejection fractionday 35

Sonographic cardiac left ventricular ejection fraction

Serum levels of pro-brain natriuretic peptide (por-BNP) and Troponin-Tday 35

Serum levels of pro-BNP and Trop-T

Incidence of CTCAE grade ≥3 cardiac complicationsday 35

Incidence of CTCAE grade ≥3 cardiac complications

Rate of early deathsweek 2

Rate of early deaths (2 weeks)

Trial Locations

Locations (38)

Fakultní nemocnice Olomouc

🇨🇿

Olomouc, Czechia

Interní klinika LF Masarykovy univerzity a Fakultní nemocnice Brno

🇨🇿

Brno, Czechia

Faculty Hospital Hradec Králové, II. Clinic of international medicine

🇨🇿

Hradec Králové, Czechia

Fakultní nemocnice Královské Vinohrady

🇨🇿

Praha, Czechia

Ústav hematologie a krevní transfuze (ÚHKT)

🇨🇿

Praha, Czechia

Sozialstiftung Bamberg Klinikum am Bruderwald

🇩🇪

Bamberg, Germany

Klinikum Bielefeld

🇩🇪

Bielefeld, Germany

Augusta Kliniken Bochum Hattingen

🇩🇪

Bochum, Germany

Ev. Diakonie-Krankenhaus gGmbH Bremen

🇩🇪

Bremen, Germany

Klinikum Chemnitz GmbH

🇩🇪

Chemnitz, Germany

Universitätsklinikum Jena

🇩🇪

Jena, Germany

Städtisches Krankenhaus Kiel

🇩🇪

Kiel, Germany

Gemeinschaftsklinikum Mittelrhein GmbH

🇩🇪

Koblenz, Germany

Westpfalz-Klinikum GmbH

🇩🇪

Kaiserslautern, Germany

Universitätsklinikum Erlangen

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Erlangen, Germany

Marienhospital Düsseldorf GmbH

🇩🇪

Düsseldorf, Germany

Universitätsklinikum Essen

🇩🇪

Essen, Germany

Klinikum Nürnberg-Nord

🇩🇪

Nürnberg, Germany

Asklepios Klinik St. Georg

🇩🇪

Hamburg, Germany

Universitätsklinikum Heidelberg

🇩🇪

Heidelberg, Germany

St. Bernward Krankenhaus Hildesheim

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Hildesheim, Germany

Universitätsklinikum Gießen und Marburg

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Marburg, Germany

Universitätsklinikum Münster

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Münster, Germany

St. Marien-Hospital Hamm

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Hamm, Germany

Carl.Thiem-Klinikum Cottbus gGmbH

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Cottbus, Germany

Universitätsklinikum Carl Gustav Carus Dresden

🇩🇪

Dresden, Germany

Johann Wolfgang Goethe-Universität Frankfurt am Main

🇩🇪

Frankfurt am Main, Germany

Universitätsklinikum Leipzig

🇩🇪

Leipzig, Germany

Robert-Bosch-Krankenhaus

🇩🇪

Stuttgart, Germany

Rems-Murr-Klinikum Winnenden

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Winnenden, Germany

Universitätsklinikum Halle (Saale)

🇩🇪

Halle, Germany

Klinikum Augsburg

🇩🇪

Augsburg, Germany

Uniklinik RWTH Aachen

🇩🇪

Aachen, Germany

Klinikum Altenburger Land GmbH

🇩🇪

Altenburg, Germany

Krankenhaus Düren gem. GmbH

🇩🇪

Düren, Germany

Diakonie-Klinikum Schwäbisch Hall gGmbH

🇩🇪

Schwäbisch Hall, Germany

Charite Campus Benjamin Franklin

🇩🇪

Berlin, Germany

Helios Klinikum Berlin-Buch

🇩🇪

Berlin, Germany

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