Study on the Safety and Effectiveness of Azacitidine and Venetoclax for Untreated Higher-Risk Myelodysplastic Syndromes Not Eligible for Transplant

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What is this study about?

This clinical trial is focused on studying a group of blood disorders known as higher-risk myelodysplastic syndromes (MDS). These are conditions where the bone marrow does not produce enough healthy blood cells. The study is specifically for patients who have not received previous treatment and are not eligible for a type of treatment called allogenic transplantation, which involves receiving stem cells from a donor. The trial will test the safety and initial effectiveness of a combination of two medications: Oral Azacitidine (also known by its code name CC-486 or ONUREG) and Venetoclax (also known as VENCLYXTO or by its code name ABT-199).

The purpose of the study is to find the best dose and treatment duration for these medications when used together. Participants will take these medications in tablet form by mouth. The study will monitor how the body responds to the treatment and any side effects that may occur. The trial will also look at how well the treatment works in improving the condition of the blood and bone marrow over time.

Throughout the study, participants will have regular check-ups, including blood tests and other assessments, to track their progress and ensure their safety. The study aims to provide valuable information on how these medications can be used to treat higher-risk myelodysplastic syndromes effectively.

1 initial visit

Upon joining the study, an initial visit is scheduled. During this visit, a detailed explanation of the study procedures and requirements is provided. An informed consent form is signed to confirm understanding and agreement to participate.

A series of assessments are conducted, including blood tests and a bone marrow aspirate, to establish baseline health status and eligibility for the study.

2 medication administration

The study involves taking two medications: azacitidine and venetoclax. Azacitidine is provided in tablet form, with dosages of 200 mg and 300 mg, taken orally. Venetoclax is also taken orally as a film-coated tablet.

The specific dosage and frequency of administration are determined by the study team based on individual health assessments. The treatment duration is adjusted to find the optimal dose and treatment length.

3 regular monitoring

Throughout the study, regular visits are required for monitoring health status and response to the medication. This includes blood tests and other assessments to track progress and any side effects.

Participants are required to be available for these assessments and to communicate any changes in health or side effects experienced during the study.

4 end of cycle evaluation

At the end of each treatment cycle, an evaluation is conducted to assess the response to the medication. This includes measuring overall response and any dose-limiting toxicity.

The results of these evaluations help determine the continuation or adjustment of the treatment plan.

5 completion of study

Upon completion of the study, a final assessment is conducted to evaluate the overall health status and response to the treatment.

Participants may be required to undergo additional follow-up assessments to monitor long-term effects and outcomes.

Who Can Join the Study?

Must understand and voluntarily sign an informed consent form before starting any study procedures.
Must be able to communicate with the study team and follow the study requirements, including regular blood tests and bone marrow tests.
Females of childbearing potential must agree to take pregnancy tests before and during the study and use effective birth control methods or abstain from sex during the study and for a period after the study ends.
Male participants must agree to use effective birth control methods or abstain from sex during the study and for 3 months after the study ends. They should also learn about sperm preservation before starting treatment.
Must be at least 18 years old.
Must have a diagnosis of myelodysplastic syndromes (MDS) with specific characteristics as confirmed by a doctor.
Must not have received prior treatment for MDS with certain types of chemotherapy or experimental drugs.
Must not be eligible for certain intensive treatments due to personal health factors.
Must have an ECOG performance status of 0, 1, or 2, which means being fully active or having some limitations but able to care for oneself.
Must have a white blood cell count of 10 G/L or less. Treatment to lower this count is allowed before starting the study.
Must have adequate liver function, as shown by specific blood test results.
Must have adequate kidney function, as shown by a specific calculation of kidney performance.

Who Cannot Join the Study?

Patients who have already received treatment for their condition.
Patients who are eligible for a type of treatment called allogenic transplantation, which is a procedure where a person receives blood-forming stem cells from a genetically similar, but not identical, donor.
Patients who are not within the specified age range for the study.
Patients who are not part of the specified clinical trial groups.
Patients who are not considered part of the vulnerable population selected for this study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
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 Hospitalier Universitaire De Nimes	Nimes	France
Centre Hospitalier Universitaire De Poitiers	Poitiers	France
Centre Henri Becquerel	Rouen	France
Centre Hospitalier Le Mans	Le Mans	France
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Centre Hospitalier Universitaire De Nantes	Nantes	France
Centre Hospitalier Universitaire De Nice	Nice	France
Centre Hospitalier Universitaire Amiens Picardie	Amiens	France
Centre Hospitalier Lyon Sud	Pierre Benite	France
Hopital Beaujon	Clichy	France
Groupement Des Hopitaux De L’Institut Catholique De Lille	lomme	France
Centre Hospitalier Intercommunal De Mont De Marsan Et Du Pays Des Sources	Mont-de-Marsan	France
Cdhbpt Hudnjdiejxl Vunextm	Valence	France
Hybosbi Pvtmi Svnelcl	Cesson Sevigne	France
Cbi dbqutffldsilxw	Epagny Metz Tessy	France
Cdrvxn Hngvznmjvod Ey Ucwsbddacxfws Dv Lewydlb	Limoges	France
Cyjwaz Hlzjbizuvii Rauxsusf Ucvwxyynmglpl Db Tpdkf	Tours	France
Chtz Df Ncnsg	Vandoeuvre Les Nancy	France
Cfbmzz Heaijktuths Roazwclc Douzzgzvzmwveo	Angers	France
Izoncekv Plwigzxdzkgjkcn Cizrqz Cutgws	Marseille	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	06.12.2023

Trial locations

Investigated drugs:

Oral Azacitidine (ONUREG®) is a medication used in this clinical trial to help treat higher-risk myelodysplastic syndromes, which are a group of disorders caused by poorly formed or dysfunctional blood cells. This medication is taken by mouth and works by interfering with the growth of cancer cells, which may slow down or stop their spread. The goal of using Oral Azacitidine in this trial is to find the best dose and treatment duration when used together with another medication.

Venetoclax (VENCLYXTO®) is another medication used in this trial. It is designed to help treat certain types of blood cancers by targeting and blocking a specific protein that cancer cells need to survive. By doing this, Venetoclax can help kill cancer cells and reduce the number of abnormal blood cells. In this study, Venetoclax is combined with Oral Azacitidine to see how well they work together in treating higher-risk myelodysplastic syndromes.

Higher-risk myelodysplastic syndromes – Myelodysplastic syndromes (MDS) are a group of disorders caused by poorly formed or dysfunctional blood cells. In higher-risk MDS, the bone marrow produces immature blood cells that do not mature properly, leading to a shortage of healthy blood cells. This condition can cause symptoms such as fatigue, infections, and bleeding due to low levels of red blood cells, white blood cells, and platelets. Over time, higher-risk MDS can progress, with an increased likelihood of transforming into acute myeloid leukemia (AML). The progression involves worsening blood cell counts and increased bone marrow blasts. The disease is characterized by its impact on blood cell production and the potential for significant complications.

Trial ID:

2024-514876-41-00

Protocol code:

GFM-ONUVEN-MDS

NCT ID:

NCT05782127

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on the Safety and Effectiveness of Azacitidine and Venetoclax for Untreated Higher-Risk Myelodysplastic Syndromes Not Eligible for Transplant

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?