Study Comparing CPX-351 and Conventional Drug Combination for Patients with High-Risk Myelodysplastic Syndromes and Oligoblastic Acute Myeloid Leukemia Before Stem Cell Transplant

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What is this study about?

This clinical trial is focused on studying treatments for patients with higher risk Myelodysplastic Syndromes (MDS) and oligoblastic Acute Myeloid Leukemia (AML). These are types of blood disorders where the bone marrow does not produce enough healthy blood cells. The study will compare a new treatment called CPX-351, also known as Vyxeos Liposomal, with conventional care strategies. CPX-351 is a combination of two chemotherapy drugs, cytarabine and daunorubicin, delivered in a special form to improve their effectiveness. The study also involves other medications like azacitidine, which is another type of chemotherapy.

The purpose of the study is to compare the effectiveness of CPX-351 with standard treatments before patients undergo a procedure called allogeneic stem cell transplantation. This procedure involves replacing diseased bone marrow with healthy stem cells from a donor. The study will follow participants over a period of time to see how well they respond to the treatments and how long they remain free of disease events. Some participants will receive CPX-351, while others will receive standard treatments, which may include medications like cytarabine, daunorubicin hydrochloride, and azacitidine. A placebo may also be used in some cases.

Participants in the study will receive their assigned treatment and be monitored regularly by healthcare professionals. The study aims to understand which treatment option provides better outcomes for patients, including survival rates and quality of life. The results will help determine the best approach to treating higher risk MDS and oligoblastic AML before stem cell transplantation. The study is expected to continue until March 2026, with regular assessments to track the progress and health of the participants.

1 initial assessment

Upon joining the clinical trial, you will undergo an initial assessment to confirm eligibility. This includes a review of your medical history, a physical examination, and laboratory tests to ensure you meet the study criteria.

2 treatment phase

You will receive the study medication, Vyxeos Liposomal, which contains the active substances cytarabine and daunorubicin hydrochloride. This medication is administered through an intravenous infusion.

The dosage and frequency of administration will be determined by the study protocol and your healthcare provider. The treatment phase is designed to compare the effectiveness of this medication with conventional care strategies.

3 monitoring and follow-up

Throughout the trial, regular monitoring will be conducted to assess your response to the treatment and to check for any side effects. This includes blood tests, imaging studies, and other necessary evaluations.

You will be required to attend scheduled follow-up visits to ensure your safety and to collect data for the study.

4 end of treatment evaluation

At the end of the treatment phase, a comprehensive evaluation will be conducted to assess the overall response to the therapy. This includes a final set of tests and assessments similar to those conducted during the initial assessment.

5 long-term follow-up

After completing the treatment phase, you will enter a long-term follow-up period. This involves periodic check-ups to monitor your health status and to gather information on the long-term effects of the treatment.

The follow-up period is crucial for understanding the overall impact of the therapy on your condition.

Who Can Join the Study?

Adult patients, aged 18 to 75 years.
Patients must agree to use a medically acceptable method of contraception:
- Female patients of childbearing potential must use contraception for at least 2 months before the first dose of the study drug and continue throughout the study and for 6 months after the last dose.
- Male patients must refrain from sperm donation and use contraception throughout the study and for 6 months after the last dose.
Diagnosis of high-risk MDS (myelodysplastic syndromes) or oligoblastic AML (acute myeloid leukemia) with less than 29% of immature blood cells (blasts) in the bone marrow.
Bone marrow blasts must be 5% or more.
IPSS (International Prognostic Scoring System) score must be intermediate or high.
Plan to undergo alloHCT (allogeneic hematopoietic cell transplantation) within the next 6 months.
ECOG (Eastern Cooperative Oncology Group) performance status of 0 or 1, indicating full activity or some symptoms but nearly fully ambulatory.
Signed informed consent to participate in the study.
Laboratory values must meet all of the following:
- Serum creatinine less than 2.0 mg/dL (a measure of kidney function).
- Serum total bilirubin less than 2.0 mg/dL (a measure of liver function).
- Serum alanine aminotransferase or aspartate aminotransferase less than 3 times the upper limit of normal (measures of liver enzymes).
Cardiac ejection fraction (LVEF) of 50% or more, as measured by echocardiography (a test that checks how well the heart is pumping).

Who Cannot Join the Study?

Patients who have not received treatment for their condition.
Patients with a higher risk of a condition called MDS (a type of blood disorder).
Patients with oligoblastic AML (a type of blood cancer with a low number of immature blood cells).
Patients who are eligible and planning to undergo a procedure called allogeneic HCT (a type of stem cell transplant from a donor) within the next 6 months.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Medizinische Hochschule Hannover	Hanover	Germany
University Hospital Jena KöR	Jena	Germany
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany
Rostock University Medical Center	Rostock	Germany
Technische Universitaet Dresden	Dresden	Germany

Other Sites

Site Name	City	Country
Klinikum Nuernberg	Nürnberg	Germany
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)	Munich	Germany
Robert Bosch Krankenhaus GmbH	Stuttgart	Germany
Universitaetsklinikum Aachen AöR	Aachen	Germany
HELIOS Klinikum Berlin-Buch GmbH	Berlin	Germany
Klinikum Frankfurt (Oder) GmbH	Frankfurt (oder)	Germany
Universitaetsklinikum Tuebingen AöR	Tuebingen	Germany
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Klinikum Chemnitz gGmbH	Chemnitz	Germany
Universitaetsklinikum Mannheim GmbH	Mannheim	Germany
Rheinische Friedrich-Wilhelms-Universitaet Bonn	Bonn	Germany
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH	Ulm	Germany
Universitaetsklinikum Leipzig AöR	Leipzig	Germany
Uwcuptdfthnwthtbgxuyw Ejtsl Aqe	Essen	Germany
Ojklyquwzijrrx Lxel Gavy	Linz	Austria
Ueibnqwfwulhmunkrhlkk Aolstehq	Augsburg	Germany
Gmqzdlceazqvlbwsnhwpj Mgogijqwdnq gzjkq	Koblenz	Germany
Uyaraydvyq Htgskrwu Crimbgl	Cologne	Germany
Uusubxuiqiacngfzetvvb Dkrqvstjwzl Aka	Duesseldorf	Germany
Gtbpgk Unxcctplxc Fnlxoaulw	Frankfurt	Germany
Mnqejqxszbyfeerofxdqypanfa Hemuuhrssxeyeyya	Halle (Saale)	Germany

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	01.04.2019
Germany	Not recruiting	01.04.2019

Trial locations

Investigated drugs:

CPX-351 is a special type of chemotherapy medication that is designed to treat certain blood cancers. It is made up of two drugs, daunorubicin and cytarabine, which are combined in a way that helps them work better together. This combination is enclosed in tiny fat-like particles called liposomes, which help the medication stay in the body longer and target cancer cells more effectively. In this trial, CPX-351 is being tested to see if it can improve the chances of survival without cancer events for patients with higher risk myelodysplastic syndromes (MDS) or oligoblastic acute myeloid leukemia (AML) before they undergo a stem cell transplant.

CCR, or Conventional Care Regimen, refers to the standard treatments that are usually given to patients with higher risk MDS or oligoblastic AML. These treatments can include a variety of chemotherapy drugs that are commonly used to manage these types of blood cancers. The goal of using CCR in this trial is to compare its effectiveness against the new treatment, CPX-351, to see which one provides better outcomes for patients before they receive a stem cell transplant.

Investigated diseases:

Acute myeloid leukaemia

Myelodysplastic Syndromes (MDS) – Myelodysplastic Syndromes are a group of disorders caused by poorly formed or dysfunctional blood cells. These syndromes occur when something goes wrong in the bone marrow, where blood cells are produced. Over time, the bone marrow becomes less effective at producing healthy blood cells, leading to anemia, infections, or bleeding. The disease can progress slowly or quickly, and in some cases, it may transform into acute myeloid leukemia. Patients with higher risk MDS have a greater chance of this progression. The condition is more common in older adults.

Acute Myeloid Leukemia (AML) – Acute Myeloid Leukemia is a type of cancer that starts in the blood-forming cells of the bone marrow and quickly moves into the blood. It is characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with the production of normal blood cells. Oligoblastic AML refers to a subtype with a lower percentage of blast cells in the bone marrow. The disease progresses rapidly and requires immediate medical attention. Symptoms may include fatigue, fever, frequent infections, and easy bruising or bleeding. AML is more common in adults than in children.

Trial ID:

2024-515375-35-00

Protocol code:

PALOMA

NCT ID:

NCT04061239

Trial Phase:

Therapeutic exploratory (Phase II)

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Study Comparing CPX-351 and Conventional Drug Combination for Patients with High-Risk Myelodysplastic Syndromes and Oligoblastic Acute Myeloid Leukemia Before Stem Cell Transplant

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?