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MDSPhase 2PALOMANCT04061239

Comparison of Therapies Before Stem Cell Transplantation in Patients With Higher Risk MDS and Oligoblastic AML

A Phase 2 study testing CPX-351, Daunorubicin and Cytarabine in people with MDS and AML, run by GWT-TUD GmbH at 27 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
MDS, AML
What is being tested?
CPX-351, Daunorubicin and Cytarabine
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
150 participants
Where
27 sitesin 2 countries
Study dates
19 Aug 2019to 7 Aug 2026
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

27 sites in 2 countries.

27 sites in 2 countries

Germany26 sites

  • Universitätsklinikum Aachen

    Aachen, 52074

  • Universitätsklinikum Augsburg

    Augsburg, 86156

  • Charité - Universitätsmedizin Berlin

    Berlin, 12200

  • Helios Klinikum Berlin-Buch GmbH

    Berlin, 13125

  • Universitätsklinikum Bonn (UKB)

    Bonn, 53127

  • Klinikum Chemnitz-gGmbH

    Chemnitz, 09113

  • Universitätsklinikum Köln

    Cologne, 50937

  • Universitätsklinikum Carl Gustav Carus Dresden

    Dresden, 01307

  • Universitätsklinikum Düsseldorf

    Düsseldorf, 40225

  • Universitätsklinikum Essen

    Essen, 45122

  • Klinikum Frankfurt (Oder) GmbH

    Frankfurt, 15236

  • Universitätsklinikum Frankfurt

    Frankfurt, 60590

  • Universitätsklinikum Halle

    Halle, 06120

  • Medizinische Hochschule Hannover

    Hanover, 30625

  • Universitätsklinikum Heidelberg

    Heidelberg, 69120

  • Universitätsklinikum Jena

    Jena, 07740

  • Gemeinschaftsklinikum Mittelrhein gGmbH

    Koblenz, 56068

  • Universitätsklinikum Leipzig AöR

    Leipzig

  • Universitätsmedizin Mannheim

    Mannheim, 68167

  • Klinikum rechts der Isar der TU München

    München, 81675

  • Universitätsklinikum Münster

    Münster, 48149

  • Klinikum Nürnberg

    Nuremberg, 90419

  • Universitätsmedizin Rostock

    Rostock, 18055

  • Robert-Bosch-Krankenhaus Stuttgart

    Stuttgart, 70376

  • Universitätsklinikum Tübingen

    Tübingen, 72076

  • Universitätsklinikum Ulm

    Ulm, 89081

Other trials in Germany

Austria1 site

  • Ordensklinikum Linz Elisabethinen GmbH

    Linz, 4020

Other trials in Austria

About this study

Official title: 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

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.

Read the detailed description

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.

Conditions
MDS, AML
Being tested
CPX-351, Daunorubicin, Cytarabine, Azacitidine

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CPX-351Drug

    Also called Vyxeos

    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.

  • DaunorubicinDrug

    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.

  • CytarabineDrug

    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.

  • AzacitidineDrug

    Also called Vidaza

    Azacitidine at 75mg/m² for 7 days. Patients should receive a minimum of 2 and up to 6 cycles.

Study groups in NCT04061239
Study groupRoleWhat they receive
CPX-351 ArmExperimentalCPX-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. The treatment includes up to 2 cycles of induction as follows: * 1 x CPX-351 1st induction: daunorubicin 44 mg/m² and cytarabine 100 mg/m² in liposomes on days 1, 3, and 5 * 1 x CPX-351 2nd induction: daunorubicin 44 mg/m² and cytarabine 100 mg/m² in liposomes on days 1 and 3 Each induction cycle will last 28 days. Depending on the type and extent of response as well as toxicity, the patient may continue on to consolidation therapy after induction or be discontinued from the treatment phase and transferred directly to alloHCT, if applicable. CPX-351 consolidation is with daunorubicin 29 mg/m² and cytarabine 65 mg/m² in liposomes on days 1 and 3. For patients < 60 years up to 3 consolidation cycles and for patients ≥ 60 years up to 2 consolidation cycles are allowed.
Receives: Drug: CPX-351
CCR ArmOtherThe conventional care regimens (CCR) arm has 2 options according to the discretion of the investigator: 1. conventional "7+3" cytarabine/daunorubicin chemotherapy regimen 2. treatment with s.c. Azacitidine
Receives: Drug: Daunorubicin; Drug: Cytarabine; Drug: Azacitidine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: 2 years

Secondary outcomes (5)

  1. Response rate

    To compare best and overall response rate of CPX-351 vs. CCR according to AML-ELN and MDS-IWG criteria

    Measured: 2 years

  2. Toxicity Assessment

    To compare the safety and tolerability of CPX-351 vs. CCR measured by NCI CTCAE v5.0

    Measured: 2 years

  3. Proportion of patients proceeding to alloHCT

    To compare the effects of CPX-351 vs. CCR on the proportion of patients proceeding to alloHCT

    Measured: 2 years

  4. 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

    Measured: 2 years

  5. 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

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Aug 2019
  2. Study start19 Aug 2019
  3. Primary completion7 Aug 2026
  4. Study completion7 Aug 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
150 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04061239
Sponsor's protocol ID
PALOMA
Acronym
PALOMA
Status last verified
Sep 2026
First submitted
15 Aug 2019
Last updated
10 Sep 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing CPX-351, Daunorubicin and Cytarabine in people with MDS and AML, run by GWT-TUD GmbH at 27 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 17 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 27 sites in Germany, Austria. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

2-year EFS in both arms (time frame: 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04061239.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2026.

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