Study on Ivosidenib and Azacitidine for Adults with Myelodysplastic Syndromes and IDH1 Mutation

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What is this study about?

This clinical trial is focused on studying a condition known as myelodysplastic syndromes (MDS), which affects the blood and bone marrow. In this study, two treatments are being tested: ivosidenib, also known by its code name AG-120/S95031, and azacitidine, marketed as Vidaza. Both treatments are being used as monotherapies, meaning they are given alone without combining with other treatments. The purpose of the study is to evaluate the effectiveness of these treatments in patients who have not previously received a type of medication called hypomethylating agents and have a specific genetic change known as an IDH1 mutation.

Participants in the study will be randomly assigned to receive either ivosidenib or azacitidine. Ivosidenib is taken orally as a film-coated tablet, while azacitidine is administered as a powder mixed into a liquid for injection. The study will last for a period of up to 48 weeks, during which the effects of the treatments will be monitored. Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of the actual treatments.

The study aims to observe how well the treatments work in achieving remission, which means reducing or eliminating signs of the disease. It will also look at other outcomes such as overall survival, quality of life, and the rate at which the disease progresses to a more severe form called acute myeloid leukemia (AML). Participants’ health and response to the treatments will be closely monitored throughout the study to gather comprehensive data on the safety and effectiveness of ivosidenib and azacitidine in treating myelodysplastic syndromes with an IDH1 mutation.

1 joining the study

Upon joining the study, you will be randomly assigned to one of two groups. One group will receive ivosidenib and the other will receive azacitidine. This assignment is random, meaning it is like flipping a coin to decide which group you will be in.

2 receiving ivosidenib

If you are assigned to the ivosidenib group, you will take a 250 mg film-coated tablet by mouth. This medication is taken once daily. The duration of this treatment will be determined by the study team based on your response to the medication.

3 receiving azacitidine

If you are assigned to the azacitidine group, you will receive the medication as an injection. This can be administered either through a vein (intravenous) or under the skin (subcutaneous). The dosage is 25 mg/ml, and the frequency and duration will be determined by the study team based on your response to the medication.

4 regular check-ups

Throughout the study, you will have regular check-ups. These visits are important to monitor your health and the effects of the medication. The study team will conduct various tests and assessments during these visits.

5 end of study participation

Your participation in the study will continue until the study ends or until the study team decides it is best for you to stop. The estimated end date for the study is December 1, 2028. At the end of your participation, you will have a final visit to assess your health and discuss any further steps.

Who Can Join the Study?

Must have a diagnosis of HMA naive IDH1 R132 mutated MDS. This means the patient has not been treated with hypomethylating agents (HMA) and has a specific genetic mutation called IDH1 R132 in their myelodysplastic syndrome (MDS).
The diagnosis must be confirmed according to the World Health Organization (WHO) criteria.
For patients with moderate high, high, and very high-risk MDS, they are eligible regardless of blood counts, with blast counts (immature blood cells) between 0-19%.
For patients with low and moderate low-risk MDS, they must have low blood cell counts related to MDS, such as:
- Platelets less than 100,000 per microliter (a measure of blood clotting cells).
- Absolute neutrophil count (ANC) less than 1,000 per cubic millimeter (a measure of a type of white blood cell).
- Hemoglobin (Hgb) less than 10 grams per deciliter (a measure of red blood cells).
For low and moderate low-risk MDS, they must have a blast count between 5-19%.
For low and moderate low-risk MDS, they must be eligible for HMA therapy. Very low-risk participants are not eligible.
The IDH1 R132 mutation must be confirmed either locally or centrally, with specific types of mutations (C/G/H/L/S).
Participants can be of any gender.
Participants must be within the age range of adults and older adults.
Participants from vulnerable populations are eligible.

Who Cannot Join the Study?

Patients who have previously been treated with a type of medication called a hypomethylating agent (HMA) cannot participate. These are drugs used to treat certain blood disorders.
Patients with a different type of blood disorder than the one being studied cannot participate.
Patients who are not within the specified age range for the study cannot participate.
Patients who are not able to give their own consent or are considered part of a vulnerable population cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Technische Universitaet Dresden	Dresden	Germany
Medizinische Hochschule Hannover	Hanover	Germany
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Universitaetsmedizin Goettingen	Goettingen	Germany
Hospital Universitario De Salamanca	Salamanca	Spain
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Universitaet Leipzig	Leipzig	Germany
Oncopole Claudius Regaud	Toulouse	France
Hospital Universitario De Navarra	Pamplona	Spain
IRCCS Humanitas Research Hospital	Rozzano	Italy
Policlinico “Tor Vergata”, Università degli Studi di Roma TOR VERGATA	Rome	Italy

Other Sites

Site Name	City	Country
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)	Munich	Germany
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Marien Hospital Duesseldorf GmbH	Duesseldorf	Germany
Universita’ Politecnica Delle Marche	Ancona	Italy
Centre Hospitalier Universitaire De Nantes	Nantes	France
Centre Hospitalier Universitaire De Nice	Nice	France
Hopital Beaujon	Clichy	France
Usroozhavvvi Mgznupa Cvmhgkw Ghewdepue	Groningen	The Netherlands
Atuhpwpfy Ucq	Amsterdam	The Netherlands
Aqacvvm Owgwbyghgxl Uwhivpwcaxfgz Cjcfahvzrpom Dvsba Speozd E Dqpml Siacqqz Db Tgcrja	Turin	Italy
Cdpo Dd Nqlek	Vandoeuvre Les Nancy	France
Amaotlw Ugatz Sxrrupqfs Lcjzhx Dk Bmnsppi	Bologna	Italy
Iansbodc Ctzafs Dxzmvqpbolryhwzst	L'hospitalet De Llobregat	Spain
Hutnimgn Vpit dtfwphgf	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
France	Recruiting	30.12.2024
Germany	Recruiting	30.12.2024
Italy	Recruiting	30.12.2024
Spain	Recruiting	30.12.2024
The Netherlands	Recruiting	30.12.2024

Trial locations

Investigated drugs:

Ivosidenib is a medication used in this clinical trial to treat patients with a specific type of blood disorder called myelodysplastic syndromes (MDS). This disorder is characterized by the bone marrow not producing enough healthy blood cells. Ivosidenib works by targeting and blocking a specific enzyme that is mutated in some patients with MDS. By blocking this enzyme, the medication helps to stop the growth of abnormal cells and allows the bone marrow to produce more healthy blood cells.

Azacitidine is another medication used in this trial for treating myelodysplastic syndromes (MDS). It is a type of chemotherapy that works by interfering with the growth of cancer cells, which are abnormal cells that grow uncontrollably. Azacitidine helps to slow down or stop the growth of these cells, allowing the bone marrow to produce more normal blood cells. This can help improve symptoms and quality of life for patients with MDS.

Investigated diseases:

Myelodysplastic syndrome

Myelodysplastic Syndromes (MDS) – Myelodysplastic Syndromes are a group of disorders caused by poorly formed or dysfunctional blood cells. These disorders occur when something goes wrong in the bone marrow, where blood cells are produced. Over time, the bone marrow becomes less effective at producing healthy blood cells, leading to anemia, infections, or bleeding. The progression of MDS can vary, with some cases remaining stable for years, while others may progress to more severe forms of blood disorders. The condition is characterized by a shortage of one or more types of blood cells, which can lead to symptoms like fatigue, shortness of breath, and increased risk of infections. MDS can sometimes transform into acute myeloid leukemia, a more aggressive type of blood cancer.

Trial ID:

2023-510155-37-00

Protocol code:

S095031-178

NCT ID:

NCT06465953

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Ivosidenib and Azacitidine for Adults with Myelodysplastic Syndromes and IDH1 Mutation

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?