To compare the event-free survival at 2 years of CPX-351 vs. conventional care regimens before allogeneic blood cell transplantation as first line treatment in patients with higher risk MDS and oligoblastic AML.
Allogeneic stem cell transplantation (alloHCT) is considered the only potentially curative treatment option for MDS patients and is therefore often considered the standard treatment for mainly higher-risk MDS patients up to the age of 75 years. One common approach to "bridge" higher-risk MDS from the time of diagnosis to transplantation is a treatment with hypomethylating agents such as azacitidine due to its anticipated low toxicity profile. Alternative strategies are intensive 7+3 chemotherapy with anthracycline and cytarabine or direct and immediate transplantation. By this strategy the time interval for donor search can be significantly prolonged leading to a higher proportion of success.Nevertheless, not every patient initially eligible for transplantation undergoes this procedure subsequently. A direct prospective comparison of different therapeutic approaches as outlined above versus CPX-351 prior to alloHCT has not been performed so far and is subject of the PALOMA trial. We hypothesize that CPX-351 will lead to higher and more durable response rates including a more favourable safety profile and long-term outcome compared to currently used conventional care regimens approaches prior to alloHCT.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
150
CPX-351 is a liposomal formulation with a fixed 5:1 molar ratio of cytarabine and daunorubicin. It will be administered as a 90-minute intravenous infusion.
Daunorubicin is commercially available as a powder for reconstitution in 20 mg vials. In this trial, daunorubicin should be administered as an IV infusion over 60 min.
Cytarabine is commercially available as vials/bottles for preparation of diluted infusion solution. Cytarabine will be administrated intravenously. In this trial, cytarabine is administered as a continuous infusion.
2-year EFS in both arms
To compare the event-free survival (EFS) at 2 years of CPX-351 vs. CCR before allogeneic blood cell transplantation (alloHCT) as first line treatment in patients with higher risk MDS and oligoblastic AML.
Time frame: 2 years
Response rate
To compare best and overall response rate of CPX-351 vs. CCR according to AML-ELN and MDS-IWG criteria
Time frame: 2 years
Toxicity Assessment
To compare the safety and tolerability of CPX-351 vs. CCR measured by NCI CTCAE v5.0
Time frame: 2 years
Proportion of patients proceeding to alloHCT
To compare the effects of CPX-351 vs. CCR on the proportion of patients proceeding to alloHCT
Time frame: 2 years
Minimal residual disease
To compare the effect of CPX-351 vs. CCR on minimal residual disease which will be assessed at all times of bone marrow puncture
Time frame: 2 years
Patient's quality of life
To compare the effect of CPX-351 vs. CCR on the quality of life. It will be measured using the EORTC-QLQ30 questionnaire
Time frame: 2 years
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Azacitidine at 75mg/m² for 7 days. Patients should receive a minimum of 2 and up to 6 cycles.
Ordensklinikum Linz Elisabethinen GmbH
Linz, Austria
RECRUITINGUniklinikum Salzburg - Landeskrankenhaus
Salzburg, Austria
WITHDRAWNUniversitätsklinikum Aachen
Aachen, Germany
RECRUITINGUniversitätsklinikum Augsburg
Augsburg, Germany
RECRUITINGCharité - Universitätsmedizin Berlin
Berlin, Germany
RECRUITINGHelios Klinikum Berlin-Buch GmbH
Berlin, Germany
RECRUITINGUniversitätsklinikum Bonn (UKB)
Bonn, Germany
RECRUITINGKlinikum Chemnitz-gGmbH
Chemnitz, Germany
RECRUITINGUniversitätsklinikum Köln
Cologne, Germany
RECRUITINGUniversitätsklinikum Carl Gustav Carus Dresden
Dresden, Germany
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