Assistance Publique Hopitaux De Paris
Paris, France
Not recruitingRare disease
Study on the Effectiveness of Enasidenib Mesilate in Patients with Myelodysplastic Syndrome with IDH2 Mutation
Verified siteInvestigational
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What is this trial about?
A plain-language summary of the goals, design and what participants do
This clinical trial is focused on studying a blood disorder called myelodysplastic syndrome (MDS), which is characterized by the bone marrow not producing enough healthy blood cells. The study specifically targets patients with a mutation in a gene known as IDH2. The treatment being tested is a medication called Enasidenib Mesilate, also known by its code name AG-221. This medication is taken orally in the form of a film-coated tablet. Another medication, Vidaza, which is a powder for suspension for injection, is also involved in the study.
The purpose of the study is to evaluate how well AG-221 works in treating patients with myelodysplastic syndrome who have the IDH2 mutation. The study will also assess the safety and tolerability of the treatment. Participants will receive the medication over a period of up to 24 months. During this time, they will have regular check-ups to monitor their response to the treatment and any side effects they may experience.
Throughout the study, the response to the treatment will be measured by looking at changes in the participants' blood cell counts and overall health. The study aims to determine the effectiveness of AG-221 in improving the condition of patients with myelodysplastic syndrome and to gather information on any potential risks associated with the treatment. Participants will be closely monitored to ensure their safety and well-being throughout the trial.
The research process
The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.
Who can join the trial?
14 criteria
- Have a condition called myelodysplastic syndrome with a specific genetic change known as IDH2 mutation.
- Be at least 18 years old.
- Understand and voluntarily sign a consent form to participate in the study.
- Be able to attend all scheduled visits and follow the study's requirements.
- Have an ECOG performance status of 0 to 2, which is a scale used to assess how well a patient can perform daily activities.
- For women who can have children, agree to take a pregnancy test before starting the study and use effective birth control methods during the study and for a specified time after the last dose of the study drug.
- For men, agree to use a condom during sexual activity with a woman who can have children while on treatment and for 3 months after the treatment ends. Also, learn about sperm preservation options before starting treatment.
- Have normal kidney function, which means certain blood test results are within a specific range.
- Have normal liver function, which means certain blood test results are within a specific range.
- Have a normal heart function, with a cardiac ejection fraction greater than 40%, which measures how well the heart pumps blood.
- Not be resistant to platelet transfusions, which are treatments to increase blood platelets.
- Belong to one of the following categories:
- Higher risk MDS without response to a drug called azacitidine after at least 6 cycles, or relapsing after a response but without significant progression.
- Untreated higher risk MDS without life-threatening low blood cell counts or recent severe infections.
- Lower risk MDS with resistance or loss of response to previous treatments and a need for regular blood transfusions.
Who cannot join the trial?
5 criteria
- Patients who do not have a **myelodysplastic syndrome** with an **IDH2 mutation** cannot participate. **Myelodysplastic syndrome** is a group of disorders caused by poorly formed or dysfunctional blood cells. An **IDH2 mutation** is a specific change in a gene that can affect cell growth.
- Patients who are not within the specified age range for the study cannot participate. The study includes certain age groups, so if you are outside these groups, you may not be eligible.
- Patients who are not part of the specified clinical trial groups cannot participate. This means the study is looking for specific types of participants based on certain characteristics.
- Patients who are not able to follow the study procedures or who have conditions that might interfere with the study results cannot participate.
- Patients who are part of a vulnerable population, which means they might need special protection or care, cannot participate.
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Verified sites
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Verified Verified CHU Grenoble Alpes
La Tronche, FranceVerified Centre Henri Becquerel
Rouen, France
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Investigated drugs
AG-221 is a medication being studied for its effects on patients with a specific type of blood disorder called IDH2 mutated myelodysplastic syndrome. This medication works by inhibiting the activity of the IDH2 enzyme, which is believed to play a role in the development of this disorder. The study aims to evaluate how well patients respond to this treatment and to assess its safety and tolerability.
What is already known about the treatment
AG-221 – This medication is administered orally and is currently being studied in clinical trials for its effectiveness in treating myelodysplastic syndrome with IDH2 mutations. It is not yet widely available in medical practice, as it is still under investigation. The main therapeutic indication for AG-221 is to improve the response rate in patients with this specific genetic mutation. At the molecular level, AG-221 works by inhibiting the IDH2 enzyme, which is involved in the abnormal production of cancer cells. It is classified pharmacologically as an IDH2 inhibitor.
Investigated diseases
Myelodysplastic Syndrome with IDH2 Mutated – This is a type of blood disorder where the bone marrow does not produce enough healthy blood cells. It is characterized by the presence of a specific genetic mutation known as IDH2. Over time, the bone marrow becomes less effective at producing blood cells, leading to symptoms such as fatigue, infections, and easy bruising or bleeding. The disease can progress slowly, but in some cases, it may evolve into a more aggressive form of blood cancer. The mutation in the IDH2 gene affects the normal function of cells, contributing to the disease's progression. Monitoring and understanding the changes in blood cell production are crucial in managing this condition.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-513858-29-01Protocol codeAG221CLMDSGFM13217Estimated enrolment68 patientsSponsorGroupe Francophone Des Myelodysplasies
sourced from the EU Clinical Trials Register and site verification
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