NCT03897127

Randomized Phase III Study of Standard Intensive Chemotherapy Versus Intensive Chemotherapy With CPX-351 in Adult Patients With Newly Diagnosed AML and Intermediate- or Adverse Genetics

Active, enrolled Phase 3 Last updated 14 July 2025

What this trial tests

Phase 3 trial testing Cytarabine in Acute Myeloid Leukemia in 882 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

4 September 2019

Primary endpoint
1 June 2027

1 June 2027

Quick facts

Lead sponsor	University of Ulm
Phase	Phase 3
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	882
Start date	4 September 2019
Primary completion	1 June 2027
Estimated completion	1 June 2027
Sites	62 locations across Austria, Germany

Drugs / interventions tested

• Cytarabine — full drug profile →
• Daunorubicin (daunorubicin) — full drug profile →
• CPX-351 (cpx-351) — full drug profile →

Conditions studied

• Acute Myeloid Leukemia — all drugs for Acute Myeloid Leukemia →

Sponsor

University of Ulm

Who can join

18 and older, any sex, with Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Overall survival (OS) in the restricted set of de novo patients
  Time frame: 2 years

Sponsor's own description

The trial is a randomized, open-label phase III study comparing CPX-351 vs conventional intensive induction and consolidation chemotherapy in patients with newly diagnosed AML and intermediate- or adverse-risk genetics (according to 2017 ELN criteria), including AML with myelodysplasia-related changes (AML-MRC) and therapy-related AML according to the World Health Organization (WHO) classification. Overall survival (OS) in the restricted set of de novo patients will be the primary endpoint.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Recent drug approvals for acute myeloid leukemia.
   Lai C, Doucette K, Norsworthy K. · · 2019 · cited 101× · PMID 31533852 · DOI 10.1186/s13045-019-0774-x
2. Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia.
   Rautenberg C, Stölzel F, Röllig C, Stelljes M, et al · · 2021 · cited 51× · PMID 34608129 · DOI 10.1038/s41408-021-00558-5
3. Single-agent and combination biologics in acute myeloid leukemia.
   Richard-Carpentier G, DiNardo CD. · · 2019 · cited 18× · PMID 31808888 · DOI 10.1182/hematology.2019000059
4. Measurable Residual Disease in High-Risk Acute Myeloid Leukemia.
   Cluzeau T, Lemoli RM, McCloskey J, Cooper T. · · 2022 · cited 12× · PMID 35267586 · DOI 10.3390/cancers14051278
5. Improving long-term outcomes with intensive induction chemotherapy for patients with AML.
   Röllig C. · · 2023 · cited 7× · PMID 38066853 · DOI 10.1182/hematology.2023000504
6. The beginning of a new therapeutic era in acute myeloid leukemia.
   Récher C. · · 2021 · cited 4× · PMID 35845213 · DOI 10.1002/jha2.252
7. Therapy-related AML: long-term outcome in a large cohort of AML-patients with intensive and non-intensive therapy.
   Gross S, Ihlow J, Busack L, Adamiak K, et al · · 2024 · cited 3× · PMID 39284846 · DOI 10.1038/s41408-024-01140-5
8. CPX-351 selectively benefits patients with AML and myelodysplasia-related mutations in the pivotal randomized trial.
   Shimony S, Murdock HM, Keating J, Tsai HK, et al · · 2026 · cited 1× · PMID 41628350 · DOI 10.1182/bloodadvances.2025019378