newly secondary AML according to WHO 2016 classification following an antecedent of Myeloproliferative Neoplasm including Essential Thrombocythemia (ET), Polycythemia Vera (PV), primary or secondary Myelofibrosis MedDRA version: 21.1 Level: PT Classification code 10000880 Term: Acute myeloid leukaemia System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Trade Name: CPX-351, VYXEOS
Product Name: Vyxeos
Product Code: CPX-351
Pharmaceutical Form: Powder for concentrate for solution for injection/infusion
Sponsors
FILO
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Diagnosis of newly secondary AML according to WHO 2016 classification following an antecedent of Myeloproliferative Neoplasm including Essential Thrombocythemia (ET), Polycythemia Vera (PV), primary or secondary Myelofibrosis 2. Age > 18 years. 3. Performance status 1.5 X institutional ULN Hepatic Serum total bilirubin: = 2.5 X ULN OR direct bilirubin = ULN for patients with total bilirubin levels = 2 mg/dL AST (SGOT) and ALT (SGPT): = 2.5 times ULN Alkaline Phosphatase: = 5 X ULN, if > 2.5 X ULN, then liver fraction should be = 2.5 X ULN *Creatinine clearance should be calculated per institutional standard 7. Life expectancy should be of 12 weeks at least according to investigator evaluation. 8. Female patients of childbearing potential must have a negative serum pregnancy test (ß-hCG) within 72 hours prior to receiving the first dose of CPX-351. Female patients who are not post-menopausal, free from menses for > 2 years or surgically sterilized, will have to use adequate barrier methods of contraception to prevent pregnancy or agree to abstain from becoming pregnant throughout the study, starting with Visit 1. 9. Male patients agree 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 and for 3 months post study. 10. Patients have the ability to understand and willingness to sign an informed consent form indicating the investigational nature of the study. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 42 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 42
Exclusion criteria
Exclusion criteria: Exclusion criteria 1. MPN/MDS mixed types 2. Prior therapy for AML transformation except for Hydroxyurea 3. Prior treatment with growth factors such as erythropoietin alfa (EPO) or granulocyte colony-stimulating factor (G-CSF), low-dose oral chemotherapy or Hypomethylating agents chemotherapy given in the chronic phase of MPN in the 30 days before inclusion, except for hydroxyurea. 4. Uncontrolled undercurrent 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. Active and uncontrolled infection. 6. Current participation or participation in a study with an investigational compound or device within 30 days of initial dosing with study drug. 7. Patients with acute promyelocytic leukemia 8. Known human immunodeficiency virus (HIV) infection or HIV-related malignancy. 9. Clinically active hepatitis B or hepatitis C infection. 10. Known allergy or hypersensitivity to any component of CPX-351. 11. "Currently active" second malignancy, other than non-melanoma skin cancer and in situ carcinoma of the cervix. 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 >3 years or are considered by their physician to be at less than 30% risk of relapse. 12. Clinical evidence of CNS leukemia. 13. Pregnancy or breastfeeding during the projected duration of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the Complete Remission (CR/CRi) rate after treatment with CPX-351 in post-MPN AML;Secondary Objective: Overall response rate (CR, CRi, PR), Time to CR, response duration, Event-Free Survival and overall survival -Early mortality (Day-30 and Day-60 mortality) -Rate of allogeneic SCT -Safety and toxicity profile of CPX-351 in this population -Duration of cytopenias -Post-induction MRD -Impact of pretreatment gene mutations on outcome (EFS) ;Primary end point(s): -Rate of CR and CRi;Timepoint(s) of evaluation of this end point: Evaluation after induction or consolidation | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): CR, CRi, PR according to ELN 2017 criteria, time from treatment start to first response, time from response to relapse or progression • Proportion of patients receiving an allo-SCT • Rate of day- 30 and day-60 mortality • Overall Survival, Event-Free Survival (EFS) • Toxicity profile, including duration of cytopenias, life threatening or fatal cytopenias, time spent in hospital for induction cycle and consolidation cycles • Pretreatment gene mutations and outcome (CR/CRi, EFS, OS) ;Timepoint(s) of evaluation of this end point: after induction or consolidation périod | — |
Countries
France
Contacts
Public ContactFILO Desk
FILO
Outcome results
None listed