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Study on Ivosidenib for Patients with Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome with IDH1 Mutation After Stem Cell Transplant

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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 two blood-related diseases: Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndromes (HR-MDS). These conditions affect the blood and bone marrow, leading to abnormal blood cell production. The study will use a medication called Ivosidenib, also known by its code name AG-120. Ivosidenib is a type of treatment that targets a specific enzyme mutation known as IDH1, which is found in some patients with these diseases.

The purpose of the study is to evaluate how effective Ivosidenib is in improving the time patients remain free from events like disease relapse or death. Participants in the study will receive Ivosidenib as a maintenance therapy for up to 24 months after undergoing a procedure called allogeneic stem cell transplantation (alloSCT), which involves receiving healthy stem cells from a donor. The study will compare the outcomes of patients taking Ivosidenib with those who do not receive this treatment.

Throughout the study, participants will be monitored for various outcomes, including their overall survival and the rate at which they remain free from disease events. The study will also track any side effects or complications that may arise from the treatment. This research aims to provide valuable insights into the potential benefits of Ivosidenib for patients with AML or HR-MDS who have the IDH1 mutation.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, you will be required to sign an informed consent form. This document confirms your understanding of the study and your willingness to participate.
You must meet certain criteria, such as being 18 years or older, having a negative pregnancy test if applicable, and agreeing to follow study procedures.
Step 2
Initial assessment
An initial assessment will be conducted to confirm your eligibility. This includes verifying your medical condition, which should be acute myeloid leukemia (AML) or high-risk myelodysplastic neoplasia (HR-MDS) with an IDH1 mutation.
Your health status will be evaluated, including organ function and performance status, to ensure you meet the study requirements.
Step 3
Medication administration
You will begin taking the investigational drug, ivosidenib, in the form of Tibsovo 250 mg film-coated tablets. This medication is taken orally.
The medication will be administered as a maintenance therapy for a duration of 24 months following your allogeneic stem cell transplantation (alloSCT).
Step 4
Regular follow-up visits
You will have regular follow-up visits to monitor your health and the effectiveness of the treatment. These visits will include assessments to check for any side effects or changes in your condition.
Your progress will be evaluated through various tests, including blood tests and possibly bone marrow aspirations, to ensure the treatment is working as intended.
Step 5
End of study participation
At the end of the 24-month treatment period, your participation in the study will conclude. A final assessment will be conducted to evaluate your overall health and the outcomes of the treatment.
You will be informed about the results of the study and any further steps that may be necessary for your health care.

Who can join the trial?

13 criteria

Age must be 18 years or older.
Women must have a negative pregnancy test.
Men must agree to use protection during sex and not donate sperm from the time they sign the consent form until one month after the last dose of the study drug.
Must be willing and able to follow all study procedures.
Must have signed an informed consent form, meaning you understand and agree to participate in the study.
Must have an ECOG performance status of 0 to 2, which is a scale used to assess how well you can perform daily activities.
Must have acute myeloid leukemia (AML) or myelodysplastic neoplasia (MDS) with an IDH1 mutation.
Must have received an allogeneic stem cell transplant (alloSCT) within the past 100 days.
Must have a documented complete remission (CR), complete remission with incomplete blood count recovery (CRi), complete remission with partial hematologic recovery (CRh), or morphologic leukemia-free state (MLFS) after alloSCT within 28 days before joining the study, confirmed by a bone marrow test.
Must have adequate organ function, which includes:
Serum creatinine clearance greater than 30 mL/min, which is a measure of kidney function.
Serum total bilirubin less than or equal to 2 times the upper limit of normal, unless due to Gilbert’s disease, which is a mild liver condition.
Left ventricular ejection fraction (LVEF) of 35% or higher, which is a measure of heart function.

Who cannot join the trial?

4 criteria

Patients who do not have acute myeloid leukemia (AML) or myelodysplastic neoplasia (MDS) cannot participate.
Patients who do not have an IDH1 mutation cannot participate. An IDH1 mutation is a specific change in a gene that can affect how cells grow.
Patients who have not undergone allo SCT cannot participate. Allo SCT stands for allogeneic stem cell transplant, which is a procedure where a patient receives stem cells from a donor.
Patients who are not able to commit to a 24-month treatment period cannot participate.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Center For Pediatric And Adolescent Medicine Of The Johannes Gutenberg University Mainz
Mainz, Germany
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Goethe University Frankfurt
Frankfurt, Germany
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Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)
Munich, Germany

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Investigated drugs

Ivosidenib is a medication being studied for its potential to help patients with certain types of blood cancers, specifically Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Neoplasia (HR-MDS), who have a specific genetic mutation called IDH1. This medication is being tested to see if it can improve the time patients remain free from cancer-related events after they have received a stem cell transplant. The idea is that by taking Ivosidenib for a period of 24 months after the transplant, it might help keep the cancer from coming back or getting worse.

Ivosidenib

What is already known about the treatment

Ivosidenib – Ivosidenib is administered orally in the form of a tablet and is currently being studied for its effectiveness in maintaining remission in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic neoplasia (MDS) who have undergone a stem cell transplant. This medication is particularly focused on patients with a specific genetic mutation known as IDH1. Ivosidenib works by inhibiting the abnormal enzyme produced by the IDH1 mutation, which helps to stop the growth of cancer cells. It is classified as a targeted therapy, aiming to improve event-free survival in these patients.

Investigated diseases

Acute Myeloid Leukemia (AML)
Acute Myeloid Leukemia is a type of cancer that affects the blood and bone marrow. 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. The disease progresses quickly and can lead to symptoms such as fatigue, fever, and an increased risk of infections. As the abnormal cells multiply, they can spread to other parts of the body, including the liver, spleen, and lymph nodes. The progression of AML can vary, but it typically involves a rapid increase in the number of immature blood cells, known as blasts. These blasts can crowd out normal cells, leading to anemia, bleeding problems, and increased susceptibility to infections.
Myelodysplastic Neoplasia (MDS)
Myelodysplastic Neoplasia, also known as Myelodysplastic Syndromes, is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when the bone marrow does not produce enough healthy blood cells, leading to a shortage of red blood cells, white blood cells, and platelets. The disease can progress slowly or quickly, depending on the specific type and severity. Symptoms may include fatigue, shortness of breath, easy bruising or bleeding, and frequent infections. Over time, MDS can transform into acute myeloid leukemia, a more aggressive form of blood cancer. The progression involves an increase in the number of immature blood cells, which can further impair normal blood cell production.