Study of ASTX727 and Donor Lymphocyte Infusions for Patients with High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia After Stem Cell Transplant

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What is this study about?

This clinical trial is focused on studying two serious blood-related diseases: Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML). These conditions affect the blood and bone marrow, leading to issues with blood cell production. The treatment being tested in this study is a combination of a medication called ASTX727 and a procedure known as donor lymphocyte infusions (DLI). ASTX727 is a tablet that contains two active substances, decitabine and cedazuridine, which work together to help control the growth of abnormal blood cells. The purpose of the study is to see if this combination can help patients achieve a longer period without disease after undergoing a stem cell transplant.

Participants in the study will receive the ASTX727 medication orally, meaning they will take it by mouth in the form of a tablet. Alongside this, they will also receive donor lymphocyte infusions, which involve giving the patient white blood cells from a donor to help boost their immune system. The study aims to observe the effects of this treatment over a period of time, specifically looking at the patients’ health one year after their stem cell transplant. The goal is to achieve a disease-free survival rate of 35% at 12 months post-transplant, which is higher than the expected rate of 12% without this treatment.

Throughout the study, researchers will monitor the overall survival of participants and identify any factors that might influence the success of the treatment. The study is designed to provide valuable information on whether this combination of ASTX727 and donor lymphocyte infusions can improve outcomes for patients with very high-risk MDS or AML. The trial is expected to continue until March 2025, allowing researchers to gather comprehensive data on the effectiveness and safety of the treatment.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria, including age between 18 and 70 years and diagnosis of high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML).

A suitable donor for stem cell transplantation is identified, and participants agree to use effective contraception during the study and for a specified period afterward.

2 initial treatment phase

Participants undergo an allogeneic stem cell transplantation, a procedure where healthy stem cells from a donor are transferred to the participant to replace damaged or diseased bone marrow.

This phase aims to prepare the body for the subsequent treatment with ASTX727 and donor lymphocyte infusions.

3 medication administration

Participants begin taking ASTX727, which is a combination of two active substances: decitabine and cedazuridine, in the form of a tablet.

The medication is taken orally, following a specific dosage and schedule as determined by the study protocol. The exact dosage and frequency are provided by the healthcare team.

4 donor lymphocyte infusions

Participants receive donor lymphocyte infusions (DLI) after the stem cell transplantation. This involves infusing additional immune cells from the donor to help fight any remaining cancer cells.

The timing and frequency of these infusions are determined by the healthcare team based on individual response and study guidelines.

5 monitoring and follow-up

Regular follow-up visits are scheduled to monitor the participant’s health, response to treatment, and any side effects.

The primary goal is to achieve a disease-free survival rate of 35% at 12 months post-transplant. Participants are closely observed for overall survival and other health outcomes at 1 and 2 years.

Who Can Join the Study?

Patients must be between the ages of 18 and 70 years.
Patients should have one of the following conditions: high-risk myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), or acute myeloid leukemia (AML).
For MDS, patients should have a poor or very poor score on the Revised International Prognostic Scoring System (IPSS).
For AML, patients should have an adverse risk according to the European LeukemiaNet (ELN) guidelines.
Patients with unfavorable genetics, which means having certain genetic abnormalities or mutations, are eligible.
Patients with AML that developed after MDS or myeloproliferative neoplasms (MPN), or AML that relapsed less than 2 years after the first complete remission, are eligible.
AML patients should have received chemotherapy.
For MDS, the percentage of immature blood cells (marrow blasts) should be less than 20% before the transplant. For AML, it should be less than 10% after chemotherapy.
The disease should not be actively growing or spreading (non-proliferative disease).
A suitable donor must be available, either fully matched or partially matched in terms of human leukocyte antigen (HLA).
Women under 50 years and men must agree to use effective birth control methods during the study and for at least 3 months after the last dose of study medication.

Who Cannot Join the Study?

Patients with a high risk of myelodysplastic syndrome or acute myeloid leukemia cannot participate. These are types of blood disorders.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
University Hospital Of Clermont-Ferrand	Clermont Ferrand	France
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Oncopole Claudius Regaud	Toulouse	France
CHU Grenoble Alpes	La Tronche	France

Other Sites

Site Name	City	Country
Centre Henri Becquerel	Rouen	France
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Centre Hospitalier Universitaire De Nantes	Nantes	France
Centre Hospitalier Universitaire Amiens Picardie	Amiens	France
Centre Hospitalier Lyon Sud	Pierre Benite	France
Hopital Beaujon	Clichy	France
Cjsaxu Hcojxunfgwh Ep Uyziyfyauawsl Du Lyqzulc	Limoges	France
Cbvo Dm Nxeaj	Vandoeuvre Les Nancy	France
Cdvnhu Hnxzxosodoq Reurzvmc Ddpcpwmhvdgpyh	Angers	France
Ikpzcctz dd Cluihanbcban Hiokchjumtm Ufenkqhtpkvhs dn Scjrc Encnoqi (nmmwhgc	Saint Priest En Jarez	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	22.06.2021

Trial locations

Investigated drugs:

ASTX727 is a combination of two drugs that work together to treat certain types of blood cancers. One part of this combination helps to stop cancer cells from growing and multiplying, while the other part helps the first drug to work better by preventing the body from breaking it down too quickly. This combination is used in the trial to help patients who have undergone a stem cell transplant, aiming to keep the cancer from coming back.

Donor Lymphocyte Infusions (DLI) is a therapy that involves giving a patient white blood cells from a donor after they have received a stem cell transplant. These white blood cells are important for fighting infections and can also help attack any remaining cancer cells in the body. In this trial, DLI is used to boost the patient’s immune system and help prevent the cancer from returning after the transplant.

Myelodysplastic Syndrome – Myelodysplastic Syndrome is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs 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 symptoms like fatigue, infections, and easy bruising. The disease can remain stable for years or progress to more severe forms. In some cases, it may evolve into acute myeloid leukemia. The progression varies greatly among individuals.

Acute Myeloid Leukemia – 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. Symptoms can include fatigue, fever, frequent infections, and easy bruising or bleeding. The disease progresses rapidly and requires prompt attention. It can spread to other parts of the body, including the lymph nodes, liver, and spleen. The progression and symptoms can vary widely among patients.

Trial ID:

2024-515353-24-00

Protocol code:

GFM-DACORAL-DLI

NCT ID:

NCT04857645

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of ASTX727 and Donor Lymphocyte Infusions for Patients with High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia After Stem Cell Transplant

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?