Study on Molidustat and Ivosidenib for Adults with Relapsed or Refractory IDH1-Mutated Acute Myeloid Leukemia or Myelodysplastic Syndrome

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What is this study about?

This clinical trial is focused on studying two types of blood cancers: Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS). These conditions are characterized by the abnormal growth of blood cells in the bone marrow. The study is specifically for patients whose cancer has returned or has not responded to previous treatments and who have a specific genetic change called an IDH1 mutation. The trial will test a combination of two medications: Molidustat Sodium, known by the code name MUSREDO, and Ivosidenib, also referred to as Tibsovo or by its code name AG-120. These medications are taken orally in tablet form.

The purpose of the study is to see if the combination of these two drugs can effectively target and inhibit the cancer cells in patients with the IDH1 mutation. The study will be conducted in two phases. The first phase will determine the best dose of Molidustat Sodium to use in combination with Ivosidenib. The second phase will assess how well this combination works in achieving complete remission, which means the disappearance of all signs of cancer in response to treatment.

Participants in the study will take the medications as prescribed and will be monitored for their response to the treatment over several months. The study will also look at the safety of the treatment and any side effects that may occur. The goal is to find a new effective treatment option for patients with these specific types of blood cancer who have limited options available. A placebo may be used in some parts of the study to compare the effects of the treatment. The study is expected to continue until early 2026.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria, including age, medical history, and ability to take oral medication.

Informed consent is required, ensuring understanding of the study’s purpose and procedures.

2 initial assessment

An initial assessment is conducted to evaluate health status and confirm the presence of the IDH1 mutation.

Baseline measurements are taken, including blood tests and other necessary evaluations.

3 phase Ia treatment

The first phase involves determining the appropriate dose of molidustat in combination with ivosidenib.

Treatment is administered orally, with doses adjusted based on observed side effects over a period of up to 42 days.

4 phase IIb treatment

In the second phase, the effectiveness of the combination treatment is assessed.

The goal is to achieve complete remission, with treatment continuing for up to 6 months.

5 monitoring and follow-up

Regular monitoring is conducted to track progress and manage any side effects.

Follow-up visits include blood tests and assessments to evaluate treatment response and overall health.

6 end of treatment

Upon completion of the treatment period, a final evaluation is performed.

Long-term follow-up may be required to monitor health outcomes and any lasting effects of the treatment.

Who Can Join the Study?

Must be 18 years or older.
Male patients and their female partners who can have children must use very effective birth control methods, along with a barrier method, from the start of the study and for 4 months and 1 week after the last dose of the study drug.
Male patients must not donate sperm from the start of the study and for 4 months and 1 week after the last dose of the study drug.
Must agree not to participate in another study while receiving treatment in this study.
Must be able to swallow pills and have no known issues with absorbing medication. Must have organs that work well enough for the study treatment, as judged by the study doctor.
Must have a diagnosis of relapsed or refractory AML (a type of blood cancer) or MDS/AML (another type of blood cancer) that has come back or not responded to treatment, and must not be eligible for intensive chemotherapy or a bone marrow transplant, or must choose not to have standard treatment.
Must have a specific genetic change called IDH1 mutation.
Must have an ECOG performance status of 0 to 2, which means the patient is fully active or has some limitations but can still take care of themselves.
Must have liver function within certain limits, unless the study doctor approves otherwise due to specific conditions.
Must have kidney function with a creatinine clearance of at least 30 mL/min, which is a measure of how well the kidneys are working.
Must be able to understand and willing to sign a form agreeing to participate in the study.
Female patients must either be unable to have children or agree not to become pregnant during the study and for 6 months after the last dose of the study drug. They must also have a negative pregnancy test before starting the study and use very effective birth control methods if they are sexually active.
Female patients must agree not to breastfeed from the start of the study and for 2 months after the last dose of the study drug.
Female patients must not donate eggs from the start of the study and for 6 months after the last dose of the study drug.

Who Cannot Join the Study?

Patients who have not experienced a return or worsening of their condition after previous treatment.
Patients who are eligible for intensive chemotherapy, which is a strong treatment using drugs to kill cancer cells.
Patients who can undergo alloHCT, which is a type of bone marrow transplant from a donor.
Patients who do not have the specific genetic change called IDH1 mutation in their cancer cells.
Patients who are not diagnosed with AML (Acute Myeloid Leukemia) or MDS/AML (Myelodysplastic Syndromes/Acute Myeloid Leukemia), which are types of blood cancer.
Patients who are not adults, meaning they are under 18 years old.
Patients who belong to a vulnerable population, which means they might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Medizinische Hochschule Hannover	Hanover	Germany
Universitaetsmedizin Goettingen	Goettingen	Germany
Center For Pediatric And Adolescent Medicine Of The Johannes Gutenberg University Mainz	Mainz	Germany

Other Sites

Site Name	City	Country
Klinikum Oldenburg AöR	Oldenburg In Holstein	Germany
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)	Munich	Germany
Staedtisches Klinikum Braunschweig gGmbH	Brunswick	Germany
Klinikum der Stadt Ludwigshafen am Rhein gGmbH	Ludwigshafen Am Rhein	Germany
Gesundheit Nord gGmbH Klinikverbund Bremen	Bremen	Germany
Universitaetsklinikum Tuebingen AöR	Tuebingen	Germany
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH	Ulm	Germany
Uoieecxxnp Muhiucz Cabphl Hjjfsaujzqfxjvclm	Hamburg	Germany
Usmxhxopympgmssipwvej Mxwuujrg Anb	Munster	Germany
Meftszhujdowmzccsghoqputdh Hhdxdzrfnzguupnb	Halle (Saale)	Germany

Trial status

Country	Status	Recruitment Start
Germany	Not yet recruiting	10.07.2023

Trial locations

Investigated drugs:

Ivosidenib

Molidustat is a medication being studied for its potential to help treat certain types of blood cancers. It works by inhibiting a specific enzyme, which may help to control the growth of cancer cells. In this trial, it is being tested to find the best dose and to see how effective it is when used with another medication.

Ivosidenib is a medication used to treat certain types of blood cancers that have a specific genetic mutation called IDH1. It works by targeting and blocking the abnormal protein produced by the mutated gene, which can help to stop the growth of cancer cells. In this trial, it is being used in combination with molidustat to see if the two medications together can improve treatment outcomes for patients.

Investigated diseases:

Myelodysplastic syndrome

Acute Myeloid Leukemia (AML) – Acute Myeloid Leukemia is a type of cancer that affects the blood and bone marrow, characterized by the rapid growth of abnormal white blood cells. These abnormal cells accumulate in the bone marrow, interfering with the production of normal blood cells. As the disease progresses, it can lead to symptoms such as fatigue, frequent infections, and easy bruising or bleeding. AML can occur at any age but is more common in adults. The disease is classified based on genetic mutations, such as IDH1 mutations, which can influence its behavior and response to treatment. Relapsed or refractory AML refers to cases where the disease returns after treatment or does not respond to initial therapy.

Myelodysplastic Syndromes (MDS) – Myelodysplastic Syndromes are a group of disorders caused by poorly formed or dysfunctional blood cells. These syndromes occur when the bone marrow does not produce enough healthy blood cells, leading to symptoms like anemia, frequent infections, and bleeding issues. MDS can vary in severity and may progress to acute myeloid leukemia in some cases. The condition is more common in older adults and can be associated with genetic mutations, such as those affecting the IDH1 gene. The progression of MDS can be slow, but it may become more aggressive over time. Relapsed or refractory MDS refers to cases where the condition returns after treatment or does not respond to initial therapy.

Trial ID:

2024-514051-15-00

Protocol code:

MOLIVO-1/AMLSG33-22

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study on Molidustat and Ivosidenib for Adults with Relapsed or Refractory IDH1-Mutated Acute Myeloid Leukemia or Myelodysplastic Syndrome

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?