Active not recruitingPHASE2INTERVENTIONAL

A Study of Revumenib in Combination With Chemotherapy for Patients Diagnosed With Relapsed or Refractory Leukemia

This trial is for babies and young children (1 month to under 6 years old) who have a type of blood cancer called leukaemia. This leukaemia has either returned after treatment or isn't getting better with current treatments. Specifically, it's for those with a particular change in their genes called KMT2A-rearranged leukaemia. We want to see if combining a new drug called revumenib with standard chemotherapy is safe and effective. Revumenib is a tablet medicine designed to target these specific gene changes. This study aims to find the right dose and see if this combination improves their chances of getting better.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Children's Oncology Group

Enrolment target

Start

08 Jan 2024

Estimated completion

31 Dec 2027

Recurrent Acute Leukemia of Ambiguous Lineage Recurrent Acute Lymphoblastic Leukemia Recurrent Acute Myeloid Leukemia Due to Lineage Switch From Acute Leukemia of Ambiguous Lineage Recurrent Acute Myeloid Leukemia Due to Lineage Switch From B Acute Lymphoblastic Leukemia, KMT2A-Rearranged Recurrent Acute Myeloid Leukemia Due to Lineage Switch From Mixed Phenotype Acute Leukemia Recurrent Mixed Phenotype Acute Leukemia Refractory Acute Leukemia of Ambiguous Lineage Refractory Acute Lymphoblastic Leukemia Refractory Acute Myeloid Leukemia Due to Lineage Switch From Acute Leukemia of Ambiguous Lineage Refractory Acute Myeloid Leukemia Due to Lineage Switch From B Acute Lymphoblastic Leukemia, KMT2A-Rearranged Refractory Acute Myeloid Leukemia Due to Lineage Switch From Mixed Phenotype Acute Leukemia Refractory Mixed Phenotype Acute Leukemia

What is this study about?

This study is about a type of blood cancer called leukaemia, which affects babies and young children. Leukaemia happens when the body makes too many faulty white blood cells. These faulty cells, called 'blasts,' fill up the bone marrow (the soft centre of your bones) and can stop healthy blood cells from being made. They can also spread to other parts of the body.

For some children, their leukaemia has a specific change in a gene called KMT2A. This change can make the leukaemia harder to treat and more likely to come back. This study is for children whose KMT2A-rearranged leukaemia has either returned after previous treatment (called 'relapsed') or isn't responding to current treatments (called 'refractory').

The researchers are testing a new medicine called revumenib. This is a tablet designed to specifically target the cancer cells with the KMT2A gene change. They want to combine revumenib with standard chemotherapy treatments (like vincristine and prednisone) which work in different ways to kill cancer cells. The main goals are to find out the best and safest dose of revumenib to use with chemotherapy, and to see if this combination helps treat their leukaemia more effectively.

Key takeaways

This study is for young children (1 month to under 6 years) with a specific type of leukaemia.
It tests a new drug, revumenib, combined with chemotherapy.
The leukaemia must be KMT2A-rearranged and either relapsed or refractory.
The aim is to find a safe and effective treatment to improve outcomes.
Regular hospital visits and tests would be part of participation.

Who may be eligible?

To join this study, children need to be between 1 month and under 6 years old. They must have been diagnosed with leukaemia before their second birthday. Their leukaemia also needs to have a specific genetic change called KMT2A-rearranged.

The leukaemia must either have come back after treatment (relapsed) or not be responding to current treatments (refractory). This includes different types of leukaemia like ALL (Acute Lymphoblastic Leukaemia), ALAL (Acute Leukaemia of Ambiguous Lineage), or MPAL (Mixed Phenotype Acute Leukaemia). If their leukaemia has changed into AML (Acute Myeloid Leukaemia) but started as one of the KMT2A-rearranged types, they might also be able to join.

Before enrolling, specialist doctors will confirm the KMT2A gene change by checking a sample of the child’s bone marrow. This makes sure the treatment is appropriate for them.

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.

Is my child between 1 month and under 6 years old?
Was my child diagnosed with leukaemia before their second birthday?
Does my child's leukaemia have the KMT2A gene change?
Has my child's leukaemia come back or isn't responding to treatment?
The medical team would need to confirm these details with specific tests.

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve regular visits to the hospital. Your child would receive the new drug, revumenib, which is given as an oral medicine (a tablet or liquid by mouth), in combination with chemotherapy drugs, which are usually given through a drip (IV).

Throughout the study, there would be various tests to check how your child is doing and how the treatment is working. These might include blood tests, bone marrow checks (where a small sample of marrow is taken), heart scans (like echocardiograms or ECGs), and occasionally a lumbar puncture (where a small amount of fluid is taken from around the spine). Doctors would also keep a close eye on any side effects. The exact number of visits and tests would depend on your child's response to treatment, but the team will explain the full schedule.

Potential risks and benefits

Every medical treatment comes with potential benefits and risks. For your child, a potential benefit of joining this study is getting a new type of treatment (revumenib) that specifically targets their leukaemia, which might be more effective than current options, especially if their leukaemia has proven difficult to treat. However, there are also potential risks; combining new drugs with chemotherapy can lead to unexpected side effects, or known side effects could be more severe. The medical team will closely monitor your child and do their best to manage any side effects. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (84)

Children's Hospital of Alabama
Verified postcode
Birmingham, United States
Arkansas Children's Hospital
Verified postcode
Little Rock, United States
Kaiser Permanente Downey Medical Center
Verified postcode
Downey, United States
Loma Linda University Medical Center
Verified postcode
Loma Linda, United States
UCSF Benioff Children's Hospital Oakland
Verified postcode
Oakland, United States
Kaiser Permanente-Oakland
Verified postcode
Oakland, United States
UCSF Medical Center-Mission Bay
Verified postcode
San Francisco, United States
Children's Hospital Colorado
Verified postcode
Aurora, United States
Connecticut Children's Medical Center
Verified postcode
Hartford, United States
Alfred I duPont Hospital for Children
Verified postcode
Wilmington, United States
Children's National Medical Center
Verified postcode
Washington D.C., United States
Golisano Children's Hospital of Southwest Florida
Verified postcode
Fort Myers, United States

Common questions

What is KMT2A-rearranged leukaemia?

It's a type of leukaemia where a specific gene, KMT2A, has changed. This change can make the leukaemia harder to treat.

What is revumenib?

Revumenib is a new medicine given as a tablet. It's designed to specifically target the leukaemia cells that have the KMT2A gene change.

What does 'relapsed or refractory' mean?

'Relapsed' means the leukaemia has come back after treatment. 'Refractory' means it's not responding well to current treatments.

Is my child guaranteed to get the new drug?

Yes, if your child is eligible for this study, they will receive revumenib in combination with chemotherapy as part of the treatment plan being tested.

What are the common side effects of chemotherapy?

Chemotherapy can cause side effects like feeling sick, tiredness, hair loss, and a higher risk of infections. Your doctor can explain these in more detail.