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Comparing treatments before stem cell transplantation for high-risk MDS and AML patients

Primary Comparison of Liposomal Anthracycline Based Treatment Versus Conventional Care Strategies Before Allogeneic Stem Cell Transplantation in Patients With Higher Risk MDS and Oligoblastic AML

Phase 2 Interventional GWT-TUD GmbH · NCT04061239

This study is testing whether a new treatment called CPX-351 can help adults with high-risk blood cancers feel better before they get a stem cell transplant, compared to standard treatments.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment150 (estimated)
Ages18 Years to 75 Years
SexAll
SponsorGWT-TUD GmbH Academic / other
Drugs / interventionschemotherapy, radiation
Locations28 sites (Linz and 27 other locations)
Trial IDNCT04061239 on ClinicalTrials.gov

What this trial studies

This trial aims to compare the effectiveness of CPX-351 against conventional care regimens in improving event-free survival for patients with higher-risk myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia (AML) prior to allogeneic stem cell transplantation. The study will enroll adult patients aged 18-75 who meet specific eligibility criteria, including certain laboratory values and performance status. Participants will receive either CPX-351 or standard treatments like hypomethylating agents or intensive chemotherapy, with the goal of determining which approach leads to better outcomes before transplantation.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-75 with high-risk MDS or oligoblastic AML who are eligible for allogeneic stem cell transplantation within the next six months.

Not a fit: Patients with low-risk MDS or AML, or those who are not candidates for stem cell transplantation, may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a more effective treatment option that improves survival rates for patients with high-risk MDS and AML before undergoing stem cell transplantation.

How similar studies have performed: While this specific comparison has not been previously conducted, similar studies have shown promising results with CPX-351 in other contexts.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Male and female adult patients, 18-75 years of age
* Diagnosis of high risk MDS including oligoblastic non-proliferative (WBC \<13 Gpt/l) AML up to 29% of bone marrow blasts
* Availability of BM blast count from central morphology
* Bone marrow blasts ≥ 5%
* IPSS score intermediate or high
* alloHCT intended within the next 6 months
* ECOG performance status of 0 or 1
* Signed informed consent
* Laboratory values fulfilling the following:
* Serum creatinine \< 2.0 mg/dL
* Serum total bilirubin \< 2.0 mg/dL
* Serum alanine aminotransferase or aspartate aminotransferase \< 3 times the ULN
* Cardiac ejection fraction (LVEF) ≥ 50% by echocardiography
* Contraception:
* Female subjects of childbearing potential† must agree to use a medically acceptable method of contraception for at least 2 months prior to the first dose of CPX-351 and consent of female patients to use a medically acceptable method of contraception throughout the entire study period and for 6 months following the last dose of CPX-351. Medically acceptable methods of contraception that may be used by the patient include abstinence, diaphragm and spermicide, intrauterine device (IUD), condom and vaginal spermicide, hormonal contraceptives (patients must be stable on hormonal contraceptives for at least the prior 3 months), surgical sterilization, or post-menopausal (≥2 years of amenorrhea). Medically acceptable methods of contraception that may be used by the male partner of a female patient are condom and spermicide or vasectomy (\>6 months prior to Day-1) and are to be used throughout the entire study period and for 6 months following the last dose of CPX-351.
* Male patients must be willing to refrain from sperm donation for 6 months following the last dose of CPX-351 and must use adequate contraception throughout the entire study period and for 6 months following the last dose of CPX-351.
* Combined oral contraceptive pills are not recommended. It is recommended that during the study two medically accepted methods of contraception (e.g. as hormonal contraceptive methods along with a condom) apply.

Exclusion Criteria:

* Patients with history of myeloproliferative neoplasms (MPN) (defined as a history of essential thrombocytosis or
* polycythemia vera, or idiopathic myelofibrosis prior to the diagnosis of AML) or combined MDS/MPN are not eligible.
* WHO-2016 defined AML entities: AML with t(15;17), PML-RARA; AML with t(8;21), RUNX1-RUNX1T1, AML with inv(16)/t(16;16), CBFβ-MYH11; AML with biallelic CEBPA mutation; AML with mutated FLT3 or NPM1.
* Clinical evidence of active CNS leukemia.
* Patients with a "currently active" second malignancy other than non-melanoma skin cancers. Patients are not considered to have a "currently active" malignancy if they have completed therapy and are considered by their physician to be at less than 30% risk of relapse within one year.
* Any major surgery or radiation therapy within four weeks prior screening.
* Patients with prior treatment of either CPX-351, hypomethylating agents, cytarabine or intensive chemotherapy for high-risk MDS or AML.
* Patients with prior cumulative anthracycline exposure of greater than 368 mg/m2 daunorubicin (or equivalent).
* Any serious medical condition, laboratory abnormality or psychiatric illness that would prevent obtaining informed consent.
* Patients with myocardial impairment of any cause (e.g. cardiomyopathy, ischemic heart disease, significant valvular dysfunction, hypertensive heart disease, and congestive heart failure) resulting in heart failure by New York Heart Association Criteria (NYHA Class III or IV staging).
* Active or uncontrolled infection. Patients with an infection receiving treatment (antibiotic, antifungal or antiviral treatment) may be entered into the study but must be afebrile and hemodynamically stable for ≥72 hrs.
* Current evidence of invasive fungal infection (blood or tissue culture); patients with recent fungal infection must have a subsequent negative cultures to be eligible; known HIV (new testing not required) or evidence of active hepatitis B or C infection (with rising transaminase values).
* Hypersensitivity to cytarabine, daunorubicin or liposomal products.
* History of Wilson's disease or other copper-metabolism disorder.
* Female patients who are pregnant or lactating.

Where this trial is running

Linz and 27 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions MDSAML
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.