Study on the Safety and Effectiveness of Azacitidine and Venetoclax for Patients with Higher-Risk Chronic Myelomonocytic Leukemia (CMML)

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What is this study about?

This clinical trial is focused on studying a blood cancer called Chronic Myelomonocytic Leukemia (CMML), specifically in patients who are at a higher risk. The study is investigating the safety and effectiveness of a combination treatment using two medications: Venetoclax and Vidaza (Azacitidine). Venetoclax is taken as a tablet, while Vidaza is given as an injection. The purpose of the study is to determine how safe this combination is for patients and how well it works in treating the disease.

Participants in the study will receive the combination of Venetoclax and Vidaza over a period of time. The study is divided into different phases, starting with a safety run-in phase to monitor any side effects. Following this, the main phase of the study will assess how the treatment affects the disease, including the overall response rate, which measures how the disease responds to the treatment. The study will also look at other outcomes, such as how long the response lasts and the overall survival of the participants.

The trial aims to provide valuable information about the potential benefits and risks of using Venetoclax and Vidaza together for treating higher-risk CMML. This information could help improve treatment options for patients with this type of leukemia. Participants will be closely monitored throughout the study to ensure their safety and to gather data on the effectiveness of the treatment.

1 initiation of treatment

Upon joining the study, the treatment begins with the administration of azacitidine and venetoclax.

Azacitidine is provided as a suspension for injection and is administered subcutaneously. The dosage and frequency are determined by the study protocol.

Venetoclax is taken orally in the form of film-coated tablets. The dosage and frequency are also specified in the study protocol.

2 safety run-in phase

The initial phase of the study focuses on assessing the safety of the combination of azacitidine and venetoclax.

This phase involves monitoring for any dose-limiting toxicities that may occur within the first two cycles of treatment.

3 phase II treatment

Following the safety run-in phase, the study progresses to the phase II portion.

The primary goal is to evaluate the overall response rate after 3 and 6 cycles of treatment. This includes assessing complete remission, partial remission, marrow response, and clinical benefit.

4 response evaluation

Throughout the study, the response to treatment is evaluated using specific criteria.

The criteria include modified guidelines for assessing response in myelodysplastic syndromes and myeloproliferative neoplasms.

5 monitoring and follow-up

Regular monitoring is conducted to assess the safety profile of the treatment, including both hematological and non-hematological effects.

Additional endpoints such as overall survival, progression-free survival, and event-free survival are also evaluated.

Who Can Join the Study?

  • Must be 18 years or older.
  • Must have a CMML diagnosis according to the WHO 2016 criteria. CMML stands for Chronic Myelomonocytic Leukemia, a type of blood cancer.
  • Must be at intermediate-2 or high risk according to the CMML Prognostic Scoring System (CPSS). This system helps determine the severity of the condition.
  • Must not have had any prior treatment with HMAs. HMAs are a type of medication used to treat certain blood disorders. However, prior treatment with Erythropoiesis Stimulating Agents (ESA) is allowed if there has been a break of more than 15 days from these agents. Also, prior treatment with hydroxurea (HY) for less than 6 weeks is acceptable.
  • Must have an ECOG Performance status of 0-2. This is a scale used to assess how a disease affects a patient’s daily living abilities, with 0 being fully active and 2 being able to do some activities but unable to work.
  • Must have adequate organ function:
    • Total bilirubin less than 2 times the upper limit of normal (ULN). Bilirubin is a substance made by the liver.
    • ALT and AST less than 3 times ULN. These are enzymes that help measure liver function.
    • Creatinine clearance greater than 30 mL/min. This measures how well the kidneys are working.
  • Must have signed an Informed Consent Form, which means agreeing to participate in the study after understanding all the details.
  • If relevant, must have a negative pregnancy test and use adequate contraception. This applies to both male and female participants to prevent pregnancy during the study.
  • Must be affiliated with a health insurance system.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Institut Gustave Roussy Villejuif France
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Centre Hospitalier Universitaire De Lille Lille France
Oncopole Claudius Regaud Toulouse France
CHU Grenoble Alpes La Tronche France

Other Sites

Site Name City Country Status
Centre Hospitalier Universitaire De Poitiers Poitiers France
Centre Henri Becquerel Rouen France
Centre Hospitalier Universitaire De Montpellier Montpellier France
Centre Hospitalier Universitaire De Nantes Nantes France
Hopital NOVO Pontoise France
Centre Hospitalier Universitaire De Nice Nice France
Centre Hospitalier Universitaire Amiens Picardie Amiens France
Centre Hospitalier Universitaire De Rennes Rennes France
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
L’Hopital Prive Du Confluent Nantes France
Centre Hospitalier Intercommunal De Mont De Marsan Et Du Pays Des Sources Mont-de-Marsan France
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Clhahv Hoxgiehfhlh Rbhpdkiw Uoomgbwwzmdfb Dj Tbovj Tours France
Cvyeoc Hnfikxtvvno Rhhcsxgo Dhqrwhumtugzfo Angers France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Not recruiting
04.10.2023

Trial locations

Investigated drugs:

Azacitidine is a medication used to treat certain types of blood disorders. In this trial, it is being used to help manage higher-risk Chronic Myelomonocytic Leukemia (CMML). Azacitidine works by interfering with the growth of cancer cells, which can help slow down the progression of the disease.

Venetoclax is a medication that targets and blocks a specific protein in cancer cells, which can lead to the death of these cells. In this trial, venetoclax is combined with azacitidine to evaluate its safety and effectiveness in treating patients with higher-risk CMML. The combination aims to improve the overall response to treatment.

Investigated diseases:

Chronic Myelomonocytic Leukemia (CMML) – Chronic Myelomonocytic Leukemia is a type of cancer that starts in blood-forming cells of the bone marrow and invades the blood. It is characterized by an increase in monocytes, a type of white blood cell, in the blood. CMML is considered a type of myelodysplastic/myeloproliferative disease, which means it has features of both myelodysplastic syndromes and myeloproliferative neoplasms. The disease progresses as the abnormal cells accumulate in the bone marrow, leading to a decrease in the production of normal blood cells. Over time, this can result in symptoms such as fatigue, anemia, and increased risk of infections. In some cases, CMML can transform into acute myeloid leukemia, a more aggressive form of leukemia.

Trial ID:
2024-514878-53-00
Protocol code:
AVENHIR
NCT ID:
NCT05768711
Trial Phase:
Therapeutic exploratory (Phase II)

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