Active not recruitingPHASE2INTERVENTIONAL

Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With an IDH2 Mutation

This study is for children and young people aged 2 to 21 who have Acute Myeloid Leukemia (AML), a type of blood cancer. It's for those whose cancer has returned after treatment (relapsed) or has been difficult to treat with standard chemotherapy (refractory). Participants must also have a specific genetic change, called an IDH2 mutation, in their leukaemia cells. The study aims to see how safe the drug enasidenib is and how well it works. Enasidenib is thought to work by blocking the mutated IDH2 protein, which helps the cancer cells grow. By doing this, it might help stop the leukaemia from getting worse.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Children's Oncology Group

Enrolment target

Start

14 Aug 2023

Estimated completion

03 Oct 2026

Recurrent Acute Myeloid Leukemia Refractory Acute Myeloid Leukemia

What is this study about?

This research study is looking at a new medicine called enasidenib for children and young people (aged 2 to 21 years old) who have a type of blood cancer called Acute Myeloid Leukemia (AML). This study is for those whose AML has either come back after previous treatments or has been very hard to treat with chemotherapy.

Crucially, this study is only for patients whose AML cells have a specific genetic change, known as an IDH2 mutation. This mutation is like a tiny error in the instructions within the cancer cells. The drug enasidenib is designed to target and block this specific mutated IDH2 protein. By doing so, doctors hope it might stop the cancer cells from growing and spreading.

The main goals of the study are to understand the side effects of enasidenib and to see how effective it is in treating AML in this specific group of young patients. Researchers will carefully check how patients respond to the treatment and whether it helps shrink the cancer or put it into remission.

Key takeaways

This study is for children and young people (aged 2-21) with Acute Myeloid Leukemia (AML).
It's for AML that has come back (relapsed) or is hard to treat (refractory).
Your AML must have a specific genetic change called an IDH2 mutation.
The study drug, enasidenib, aims to block this mutation.
Participation involves taking tablets daily and having regular blood and bone marrow tests.

Who may be eligible?

This study is designed for children and young people between the ages of 2 and 21 years old. A very important requirement is that your Acute Myeloid Leukemia (AML) must have a specific genetic change, known as an IDH2 mutation. This mutation needs to be present in your leukaemia cells.

Your AML must also either have come back after previous treatments (relapsed AML) or have been difficult to treat successfully with previous chemotherapy (refractory AML). For those with relapsed AML, you shouldn't have started new leukaemia treatment for that relapse yet. For both types, your bone marrow needs to show more than 5% leukaemia cells.

Before joining, you'll need to be well enough to take part and have recovered from the serious side effects of any previous cancer treatments. Your doctor will carefully check all specific health details to make sure this study is right for you.

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.

Are you between 2 and 21 years old?
Has your AML either come back or been difficult to treat?
Have your doctors confirmed that your AML has an IDH2 genetic mutation?
Are you well enough to participate in a clinical study?
Have you recovered from serious side effects of any previous cancer treatment?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the study drug, enasidenib, as a tablet to take by mouth once a day. This treatment is given in cycles, with each cycle lasting 28 days. You could continue treatment for up to 12 cycles, which is about a year, as long as the medication is helping and you're not experiencing difficult side effects.

Throughout the study, you'll have regular visits for blood tests and bone marrow samples. These are done to check how the treatment is working and to monitor for any side effects. You may also need other tests. These collections help doctors understand how your body reacts to the drug and if the leukaemia is responding. After you finish the study treatment, doctors will continue to check on your health with follow-up appointments for up to one year.

Potential risks and benefits

Taking part in a clinical trial means you might be among the first to benefit from a new treatment for your condition. Enasidenib may help control your AML, especially since it targets a specific genetic change relevant to your cancer. However, like all medicines, enasidenib can cause side effects, some of which could be serious. The research team will carefully monitor you for these and provide support. It's important to remember that there's no guarantee the treatment will work for everyone. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (28)

Children's Hospital of Alabama
Verified postcode
Birmingham, United States
Arkansas Children's Hospital
Verified postcode
Little Rock, United States
Children's Hospital Colorado
Verified postcode
Aurora, United States
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
Verified postcode
Denver, United States
Alfred I duPont Hospital for Children
Verified postcode
Wilmington, United States
MedStar Georgetown University Hospital
Verified postcode
Washington D.C., United States
Children's National Medical Center
Verified postcode
Washington D.C., United States
UF Health Cancer Institute - Gainesville
Verified postcode
Gainesville, United States
Nicklaus Children's Hospital
Verified postcode
Miami, United States
Lurie Children's Hospital-Chicago
Verified postcode
Chicago, United States
Riley Hospital for Children
Verified postcode
Indianapolis, United States
Norton Children's Hospital
Verified postcode
Louisville, United States

Common questions

What is Acute Myeloid Leukemia (AML)?

AML is a type of cancer that starts in the blood-forming cells of the bone marrow. These cells are supposed to develop into different types of blood cells, but in AML, they become abnormal leukaemia cells.

What does 'relapsed or refractory' mean?

Relapsed means the cancer has come back after treatment. Refractory means the cancer did not respond to initial treatments or has been very difficult to treat.

What is an IDH2 mutation?

An IDH2 mutation is a specific genetic change or 'error' found in the leukaemia cells of some people with AML. This study specifically targets this particular change.

How does enasidenib work?

Enasidenib is designed to block the mutated IDH2 protein. By doing this, it aims to stop the growth of the leukaemia cells that rely on this mutated protein.

Is this study safe for children and young people?

This is a Phase 2 study, which means it has already been tested to some extent and researchers are looking more closely at its safety and effectiveness in this age group. Your safety will be carefully monitored throughout the study.