Venetoclax plus Azacitidine: Induction cycle 1:
100 mg venetoclax p.o. on day 1; 200 mg venetoclax p.o. on day 2; 400 mg venetoclax p.o. on days 3-28; 75 mg/m\^2 azacitidine s.c. on days 1-7
Induction cycles 2-3:
400 mg venetoclax p.o. on days 1-28; 75 mg/m\^2 azacitidine s.c. on days 1-7
Postremission cycles 1-9:
400 mg venetoclax p.o. on days 1-28; 75 mg/m\^2 azacitidine s.c. on days 1-7
standard of care chemotherapy plus gemtuzumab ozogamicin: Induction cycle 1:
200 mg/m\^2 cytarabine cont inf i.v. on days 1-7; 60 mg/m\^2 daunorubicin i.v. on days 3-5; 3 mg/m\^2 (max 1 vial) gemtuzumab ozogamicin i.v. on days 1+4+7
Induction cycle 2 (patients not in remission, moderate or non-responders):
3000/1000 mg/m\^2 cytarabine i.v. BID on days 1-3; 10 mg/m\^2 mitoxantrone i.v. on days 3-5
Postremission cycles 1-3:
3000/1000 mg/m\^2 cytarabine i.v. BID on days 1-3
Study summary
This phase II clinical trial evaluates the efficacy and tolerability of the non-intensive treatment with venetoclax and the hypomethylating agent azacitidine as compared to the standard of care chemotherapy plus gemtuzumab ozogamicin in newly diagnosed NPM1 mutated AML patients fit for intensive chemotherapy.
Eligibility
Sex
ALL
Min age
18 Years
Max age
70 Years
Healthy volunteers
No
Inclusion Criteria:
1. A signed informed consent
2. Newly diagnosed CD33-positive AML with NPM1 mutation according to WHO criteria
3. Age 18-70 years
4. Fit for intensive chemotherapy, defined by
* ECOG performance status of 0-2
* Adequate hepatic function: ALAT/ASAT/Bilirubin ≤ 2.5 x ULN unless considered due to leukemic organ involvement Note: Subjects with Gilbert's Syndrome may have a bilirubin \> 2.5 × ULN per discussion between the investigator and Coordinating investigator.
* Adequate renal function assessed by serum creatinine ≤ 1.5x ULN OR creatinine clearance (by Cockcroft Gault formula) ≥ 50 mL/min
5. WBC \< 25 x 109/L (\<25,000/µL), prior hydroxyurea is permitted to meet this criterion
6. Ability to understand and the willingness to sign a written informed consent.
7. Male subjects must agree to refrain from unprotected sex and sperm donation from time point of signing the informed consent until 7 months after the last dose of study drug.
8. Women of childbearing potential must have a negative serum or urine pregnancy test performed within 72 hours before first dose of study drug.
Exclusion Criteria:
1. Activating FLT3 mutation
2. Relapsed or refractory AML
3. AML after antecedent myelodysplasia (MDS) with prior cytotoxic treatment
4. Prior history of malignancy, other than MDS, unless the subject has been free of the disease for ≥ 1 year prior to start of study treatment (exceptions are basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix or of the breast, incidental histologic finding of prostate cancer (T1a or T1b using the tumor, node, metastasis clinical staging system))
5. Previous treatment with HMA or venetoclax
6. Previous treatment for AML except hydroxyurea
7. Cumulative previous exposure to anthracyclines of \> 200 mg/m\^2 doxorubicin equivalents
8. CNS involvement or extramedullary disease only
9. Known hypersensitivity to excipients of the preparation or any agent given in association with this study including venetoclax, azacitidine, cytarabine, daunorubicin, gemtuzumab-ozogamicin, or mitoxantrone
10. Known positivity for human immunodeficiency virus (HIV) and History of active or chronic infectious hepatitis unless serology demonstrates clearance of infection (i.e.
PCR undetectable viral load for hepatitis).
11. Inability to swallow oral medications
12. Any malabsorption condition
13. Cardiovascular disability status of New York Heart Association (NYHA) Class ≥ 2; unstable coronary artery disease (MI more than 6 months prior to study entry is permitted); serious cardiac ventricular arrhythmias requiring anti-arrhythmic therapy.
Note: Class 2 is defined as cardiac disease in which patients are comfortable at rest but ordinary physical activity results in fatigue, palpitations, dyspnea, or anginal pain.
14. Chronic respiratory disease that requires continuous oxygen use
15. Substance abuse, medical, psychological, or social conditions that may interfere with the subject's cooperation with the requirements of the trial or evaluation of the study results
16. Simultaneous participation in another interventional clinical trial
17. Pregnant or breastfeeding women. Breastfeeding has to be discontinued before onset of and during treatment and should be discontinued for at least 3 months after end of treatment.
18. Patients who are unwilling to follow strictly highly effective contraception requirements including hormonal contraceptives with an Pearl Index \< 1% per year in combination with a barrier method from time point of signing the informed consent until 7 months after the last dose of study drug unless one of the following criteria is met:
* post-menopausal (12 months natural amenorrhea or 6 months amenorrhea with serum FSH \> 40 U/ml)
* postoperative (6 weeks after bilateral ovarectomy with or without hysterectomy)
* medically confirmed ovarian failure
* vasectomy Note: At present, it is not known whether the effectiveness of hormonal contraceptives is reduced by venetoclax. For this reason, women must use a barrier method in addition to hormonal contraceptive methods.
19. History of clinically significant liver cirrhosis (e.g., Child-Pugh class B and C)
20. Live-virus vaccines given within 28 days prior to the initiation of study treatment Note: corona vaccines are not live-virus vaccines and are excluded from this criterion.
Primary outcome measure(s)
modified event-free survival (mEFS) — time interval from date of randomization until either primary treatment failure or hematologic relapse or molecular failure or death, whichever occurs first * Failure to achieve a CR/CRi/CRh after a maximum of two induction cycles in the control arm (SOC) or three induction cycles in the investigational arm (VEN+AZA), i.e. primary induction failure
* Hematologic relapse after previous CR/CRi/CRh
* Molecular failure, defined as either
* Molecular progression, defined as confirmed ≥ 1 log10 increase of NPM1 MRD level in any two samples in a patient without prior MRD negativity or
* Molecular relapse after previous MRD negativity, defined as confirmed ≥ 1 log10 between two consecutive positive samples in a patient who was previously tested as MRD negative
* Death
Trial sites (18)
Facility
City
Region
Status
Universitätsklinikum Essen
Essen
North Rhine-Westphalia
Recruiting
Universitätsklinikum Aachen
Aachen
Germany
Recruiting
Universitätsklinikum Augsburg
Augsburg
Germany
Recruiting
Klinikum Chemnitz gGmbH
Chemnitz
Germany
Recruiting
Universiätsklinikum Köln
Cologne
Germany
Not Yet Recruiting
Universitätsklinikum Dresden
Dresden
Germany
Recruiting
Universitätsklinikum Erlangen
Erlangen
Germany
Recruiting
Johann Wolfgang Goethe-Universität
Frankfurt am Main
Germany
Recruiting
Universitätsklinikum Halle
Halle
Germany
Recruiting
Universitätsklinikum Heidelberg
Heidelberg
Germany
Recruiting
Universitätsklinikum Schleswig-Holstein
Kiel
Germany
Recruiting
Universitätsklinikum Leipzig
Leipzig
Germany
Recruiting
Klinikum Mannheim gGmbH
Mannheim
Germany
Recruiting
Philipps-Universität Marburg Fachbereich Medizin
Marburg
Germany
Recruiting
Universitätsklinikum Münster
Münster
Germany
Recruiting
Klinikum Nürnberg-Nord
Nuremberg
Germany
Recruiting
Krankenhaus Barmherzige Brüder
Regensburg
Germany
Recruiting
Robert-Bosch-Krankenhaus
Stuttgart
Germany
Recruiting
More Technische Universität Dresden trials in Germany
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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