RecruitingPHASE3INTERVENTIONAL

Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation

This research study is for people with a specific type of blood disorder called myelodysplastic syndromes (MDS). Specifically, it's for those who have a gene change called an IDH1 mutation and haven't received a common group of treatments known as hypomethylating agents (often called HMAs). The study aims to compare two different medicines: ivosidenib and azacitidine. Participants will receive one of these medicines to see how well they work and if they are safe. If you join, you'll have regular hospital visits for blood tests, check-ups, and other assessments. The goal is to find better ways to treat this condition.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Institut de Recherches Internationales Servier

Enrolment target

Start

03 Dec 2024

Estimated completion

01 Dec 2028

Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS)Myelodysplastic Syndromes (MDS)

What is this study about?

This study is about a type of blood cancer called Myelodysplastic Syndromes (MDS). In MDS, your body doesn't make enough healthy blood cells. This particular study focuses on people with MDS who have a specific change in their genes, called an IDH1 mutation. This mutation can affect how your blood cells grow and mature. Understanding and targeting this specific gene change is important because it could lead to more effective and personalised treatments.

The study wants to compare two different medications: ivosidenib and azacitidine. Ivosidenib is a newer drug designed to target the IDH1 mutation. Azacitidine is a commonly used treatment for MDS. The researchers want to find out if ivosidenib is a better treatment option than azacitidine for people with this specific IDH1 mutation who haven't tried certain other treatments (like HMAs) yet.

By carefully comparing these two treatments, doctors hope to learn which one is safer and more effective at controlling MDS for people with the IDH1 mutation. This could help improve the lives of many people living with this challenging condition.

Key takeaways

This study is for people with a specific type of MDS with an IDH1 gene change.
It compares two medicines, ivosidenib and azacitidine, to see which works best.
Participants must not have had certain strong MDS treatments before.
You'll have regular hospital visits, blood tests, and check-ups.
You'll be randomly assigned to one of the two study medicines.
The aim is to find better treatments for MDS with the IDH1 mutation.

Who may be eligible?

This study is looking for people who have been diagnosed with Myelodysplastic Syndromes (MDS) and have a specific gene change known as an IDH1 mutation. It's important that you haven't received certain types of strong anti-cancer treatments for your MDS before. Some other treatments, like growth factors or lenalidomide, might be allowed depending on your situation.

Your specific MDS diagnosis will also matter. If your MDS is considered moderate high, high, or very high-risk, you might be suitable. If it's low or moderate low-risk, you would need to have certain low blood cell counts (like low platelets, white blood cells, or haemoglobin) and a specific percentage of immature blood cells (called blasts) in your bone marrow.

People with very low-risk MDS or those with more than 20% blasts in their bone marrow are usually not suitable for this study. The study is open to both men and women aged 18 years and older.

Quick self-check

I have been diagnosed with Myelodysplastic Syndromes (MDS).
My doctors have told me I have an IDH1 gene mutation.
I have not previously received strong anti-cancer treatments known as 'hypomethylating agents' for my MDS.
I am 18 years old or older.
My doctors consider my MDS to be moderate high, high, or very high-risk, OR I have low/moderate low-risk MDS with specific low blood cell counts and a certain level of 'blasts' in my bone marrow.

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you will be randomly assigned to receive one of two medicines: either ivosidenib or azacitidine. Ivosidenib is taken every day throughout a 28-day treatment cycle. Azacitidine is given for the first 7 days of each 28-day cycle. You won't know which treatment you are getting, and neither will your doctors.

You'll have frequent hospital visits, especially at the start. During the first month, you'll visit weekly (on days 1, 8, 15, and 22). After that, you'll visit on Day 1 of each 28-day cycle. These visits will involve various tests and checks, including physical exams, blood and urine tests, heart checks (like an ECHO and ECG), and sometimes a bone marrow test. You'll also complete questionnaires. After your treatment finishes, there will be a safety check-up, and doctors will continue to follow your health to see how you are doing overall.

Potential risks and benefits

Taking part in a clinical trial might offer potential benefits, such as access to new medications that aren't widely available yet, and closer monitoring of your health. However, there are also potential risks. The medicines might cause side effects, and some tests can be uncomfortable. It's important to remember that the study medicine may not help everyone, and in some cases, it might not work as well as standard treatments. Your health and safety are carefully monitored throughout the study. You're free to withdraw from the study at any time, for any reason, without affecting your regular medical care.

Locations (62)

Presbyterian / St. Luke'S Medical Center
Denver, United States· Not yet recruiting
University of Chicago, Duchossois Center for Advanced Medicine (DCAM)
Chicago, United States· Recruiting
Massachusetts General Hospital
Boston, United States· Recruiting
MSKCC
New York, United States· Recruiting
Unc Lineberger Comprehensive Cancer Center
Chapel Hill, United States· Recruiting
Ohio State University Comprehensive Cancer Center
Columbus, United States· Recruiting
Oncology Associates of Oregon
Eugene, United States· Recruiting
University of Texas UT Southwestern Comprehensive Cancer Center
Dallas, United States· Not yet recruiting
MD Anderson Cancer Centre
Houston, United States· Recruiting
Royal Adelaide Hospital
Adelaide, Australia· Not yet recruiting
Monash Health
Clayton, Australia· Recruiting
Northern Health
Epping, Australia· Recruiting

+50 more sites — see the official record for the full list.

Common questions

What is an IDH1 mutation?

It's a specific change in one of your genes that can affect how your blood cells develop. This study is for people who have this particular gene change.

What does 'HMA naive' mean?

It means you haven't been treated with a specific group of medicines called hypomethylating agents (HMAs) for your MDS before.

Will I know which medicine I'm getting?

No, you will be randomly assigned to receive either ivosidenib or azacitidine, and neither you nor your doctors involved in the study will know which one you are getting. This helps make the study fair.

What is a bone marrow aspirate?

It's a procedure where a small sample of your bone marrow (the spongy tissue inside some bones) is taken, usually from your hip, to check your blood cells.

How long will I be in the study?

The treatment phase involves regular cycles. After your last dose, you'll have a safety follow-up visit, and your health will continue to be monitored long-term to assess overall survival, so the total duration will vary.

How to find out more

Institut de Recherches Internationales Servier (I.R.I.S.), Clinical Studies Department

scientificinformation@servier.com +33 1 55 72 60 00 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.