Study on the Effectiveness of Enasidenib Mesilate in Patients with Myelodysplastic Syndrome with IDH2 Mutation

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What is this study about?

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.

1 initial treatment phase

The treatment begins with the administration of enasidenib mesilate in the form of film-coated tablets. The dosage is either 50 mg or 100 mg, taken orally. The specific dosage will be determined by the healthcare provider based on individual patient needs.

In addition to enasidenib mesilate, azacitidine is administered as a subcutaneous injection. The dosage is 25 mg/ml, prepared as a suspension for injection. The frequency and duration of administration will be specified by the healthcare provider.

2 monitoring and assessment

Throughout the trial, regular monitoring is conducted to assess the response to treatment. This includes evaluating the overall hematological response at 3 and 6 months, which involves checking for complete response (CR), partial response (PR), or stable disease with hematological improvement (HI).

Safety assessments are also performed, particularly for patients in cohort C, to determine the tolerability of the treatment using established criteria for evaluating non-hematological toxicities.

3 follow-up evaluations

Secondary evaluations include measuring the duration of response, time to progression, and overall survival. These assessments help in understanding the long-term effects of the treatment.

Additional analyses may involve examining cytogenetic and molecular responses, as well as identifying prognostic factors that could influence treatment outcomes.

4 completion of trial

The trial is expected to conclude by March 25, 2026. Upon completion, final assessments will be conducted to gather comprehensive data on the efficacy and safety of the treatment regimen.

Who Can Join the Study?

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 Study?

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.

Where you can join this trial?

Verified and Recommended Sites

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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 De Versailles	Le Chesnay-Rocquencourt	France
Centre Hospitalier Victor Dupouy	Argenteuil	France
Centre Hospitalier Universitaire De Nimes	Nimes	France
Centre Henri Becquerel	Rouen	France
Centre Hospitalier Le Mans	Le Mans	France
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Centre Hospitalier De La Cote Basque	Bayonne	France
Centre Hospitalier Universitaire De Nantes	Nantes	France
Centre Hospitalier Universitaire De Nice	Nice	France
Groupe Hospitalier De La Region De Mulhouse Et Sud Alsace	Mulhouse	France
Centre Hospitalier Lyon Sud	Pierre Benite	France
Hopital Beaujon	Clichy	France
Centre Hospitalier Universitaire De Caen Normandie	Caen	France
Universitaetsklinikum Leipzig AöR	Leipzig	Germany
Arogppknzf Pvjkjohy Hmvjehmk Db Pybmy	Paris	France
Ckbdju Hmvzxbzrpco Rjrqfrve Urmsjyirkceka Di Tdqou	Tours	France
Ctfkdd Hqraqymjauu Rwytpbqr Dfpwimklbaufcp	Angers	France
Ipsqjkjn dl Czkdocrmezav Hybczvtywav Uxoowilxsgfzz di Sdsav Engflba (lcyrhxw	Saint Priest En Jarez	France
Iwnpgzvv Pqheqrzpuhdallp Chjbhi Chldiz	Marseille	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	18.02.2019
Germany	Not recruiting	18.02.2019

Trial locations

Investigated drugs:

Enasidenib Mesilate

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.

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 ID:

2024-513858-29-01

Protocol code:

AG221CLMDSGFM13217

NCT ID:

NCT03503409

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Effectiveness of Enasidenib Mesilate in Patients with Myelodysplastic Syndrome with IDH2 Mutation

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?