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Active not recruitingPHASE3INTERVENTIONAL

Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation

This study is for adults who have recently been diagnosed with a type of blood cancer called Acute Myeloid Leukaemia (AML) and have a specific genetic change in their cancer cells, called an IDH1 mutation. It's designed for people who aren't suitable for very strong chemotherapy treatments. The study is testing a new drug called AG-120, given along with a common AML treatment called azacitidine. Some participants will receive AG-120 plus azacitidine, while others will receive a dummy pill (placebo) plus azacitidine. Doctors will be looking to see if the new combination can help people live longer without their disease getting worse, and whether it leads to more remissions. Around 200 people will take part globally.

At a glance

Status
Active not recruiting
Phase
PHASE3
Sponsor
Institut de Recherches Internationales Servier
Enrolment target
146
Start
26 Jun 2017
Estimated completion
30 Jun 2026
Newly Diagnosed Acute Myeloid Leukemia (AML)Untreated AMLAML Arising From Myelodysplastic Syndrome (MDS)Leukemia, Myeloid, Acute

Results

Results from this study

Posted March 2023

Results have been published for this study.

Primary outcome
Event-Free Survival (EFS)
EFS was defined as the time from randomization until treatment failure, relapse from remission, or death from any cause, whichever occurs first. Treatment failure was defined as failure to achieve complete remission (CR) by Week 24. CR: Bone marrow blasts \<5% and no Auer rods; absence of extramedullary disease; Absolute neutrophil count (ANC) ≥1.0 × 10\^9 per litre (10\^9/L) (1000 per microlitre \[1000/μL\]); platelet count ≥100 × 10\^9/L (100,000/μL); independence of red blood cell transfusions. Participants who had an EFS event (relapse or death) after, 2 or more missing disease assessments were censored at the last adequate disease assessment documenting no relapse before the missing assessments. The reported data represents the Kaplan-Meier median value.
Full results on the registry

What is this study about?

This study is for adults who have recently been told they have a type of blood cancer called Acute Myeloid Leukaemia, often shortened to AML. This particular study is for people whose AML has a specific genetic change, known as an IDH1 mutation. This mutation helps doctors understand more about your cancer and can sometimes guide treatment choices.

The main goal of this research is to see if adding a new drug, called AG-120 (also known as ivosidenib), to a standard AML treatment called azacitidine, works better than azacitidine alone. The study is specifically for people who are not able to have very intensive chemotherapy, perhaps because of their age or other health conditions. Researchers want to find out if the new drug combination can help people live longer without their cancer progressing, achieve remission more often, and improve overall survival.

By taking part in this study, you would be helping doctors learn if AG-120 could be a valuable new treatment option for people like you with IDH1-mutated AML. This type of research is vital for finding better ways to treat cancer and improve patients' lives in the future.

Key takeaways

  • This study is for newly diagnosed AML with a specific IDH1 gene change.
  • It's for adults not suitable for very strong chemotherapy.
  • Compares a new drug (AG-120) plus azacitidine to azacitidine alone.
  • Aims to find better ways to treat this type of AML.
  • Participation involves regular clinic visits and health assessments.

Who may be eligible?

This study is looking for adults aged 18 or older who have been newly diagnosed with Acute Myeloid Leukaemia (AML) and have a specific genetic change called an IDH1 mutation. It's important that you haven't received any prior treatment for your AML, apart from a medication called hydroxyurea if needed. You should also be someone for whom intensive (very strong) chemotherapy is not considered the best option, perhaps due to your age (75 or older), general fitness, or other health problems like severe heart or lung conditions, or kidney issues.

To be considered, your AML must be present in your bone marrow, and you should meet certain health checks, including having normal liver and kidney function. You'll also need to be willing to provide blood and bone marrow samples regularly throughout the study. If you're a woman who could become pregnant, you'll need to have a negative pregnancy test and agree to use effective birth control during the study. Men will also need to use effective birth control.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

  1. Are you 18 years old or older?
  2. Have you been newly diagnosed with AML and have an IDH1 gene mutation?
  3. Are you not a candidate for very intense chemotherapy?
  4. Have you had no prior AML treatment (besides hydroxyurea)?
  5. Are you willing to have regular blood and bone marrow tests?
Answer every question to see your result.

What does participation involve?

If you decide to join this study, your doctor will first check if you meet all the necessary requirements based on your medical history and test results. If you are suitable, you will then be randomly assigned to one of two groups, like flipping a coin. One group will receive the new drug, AG-120, along with azacitidine. The other group will receive a dummy pill (placebo) along with azacitidine. Neither you nor your study doctor will know which treatment you are receiving until the end of the study.

Both AG-120 (or the dummy pill) and azacitidine are given as medicines you take. Azacitidine can be given as an injection under the skin or into a vein. You will have regular visits to the clinic for check-ups, blood tests, and possibly bone marrow biopsies to see how you are responding to treatment. You will also be asked to complete questionnaires about your quality of life. The treatments will continue as long as they are helping you and you are not experiencing significant side effects. The total time you participate in the study will depend on how your treatment is working.

Potential risks and benefits

There is a chance that you might benefit from participating in this study if the new drug combination (AG-120 and azacitidine) proves to be more effective than azacitidine alone. However, there's also a possibility that the treatment may not work for you, or you might experience side effects from the study drugs. Possible side effects could include problems with your heart, lungs, kidneys, or liver, as well as general symptoms like feeling sick or tired. Your study team will explain all known side effects in detail. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (90)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Norton Cancer Institute - Suburban
    Verified postcode
    Louisville, United States
  • Massachusetts General Hospital
    Verified postcode
    Boston, United States
  • Royal Prince Alfred Hospital
    Verified postcode
    Camperdown, Australia
  • Royal Adelaide Hospital
    Verified postcode
    Adelaide, Australia
  • Flinders Medical Centre
    Verified postcode
    Bedford Park, Australia
  • Salzburger Landeskliniken
    Verified postcode
    Salzburg, Austria
  • Krankenhaus Hietzing mit Neurologischem Zentrum Rosenhugel
    Verified postcode
    Vienna, Austria
  • Unicamp Universidade Estadual de Campinas
    Verified postcode
    Campinas, Brazil
  • Hospital Amaral Carvalho
    Verified postcode
    Jaú, Brazil
  • Instituto Nacional de Cancer
    Verified postcode
    Rio de Janeiro, Brazil
  • Hospital Sirio Libanes
    Verified postcode
    São Paulo, Brazil
  • Hospital Sao Jose
    Verified postcode
    São Paulo, Brazil

Common questions

What is an IDH1 mutation?

It's a specific change in one of the genes within your cancer cells that doctors look for. It helps them understand your AML better.

What is azacitidine?

Azacitidine is a common medicine used to treat AML, especially for people who can't have aggressive chemotherapy.

What does 'non-intensive therapy' mean?

It means the treatment isn't as strong as very aggressive chemotherapy and might be more suitable for people who are older or have other health issues.

Will I know if I'm getting the new drug or the dummy pill?

No, neither you nor your doctor will know which treatment you are receiving. This is called 'double-blind' and helps ensure fair results.

How long will I be in the study?

You will stay in the study as long as the treatment is helping you and you are not having serious side effects. Your doctor will discuss this with you.

How to find out more

Official trial record

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

Discussion

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