Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Bosutinib in Pediatric Patients With Newly Diagnosed Chronic Phase or Resistant/Intolerant Ph + Chronic Myeloid Leukemia

This study is looking at a medicine called Bosutinib for children and teenagers aged 1 to 17 who have a type of blood cancer called Philadelphia chromosome-positive Chronic Myeloid Leukemia (CML). This includes those newly diagnosed and those whose CML hasn't responded well to previous treatments or who couldn't tolerate them. The main goals are to figure out the best dose of Bosutinib for children, see how safe it is, and check if it helps treat their CML. Researchers will also learn how the body handles the medicine. The study will involve hospitals internationally, and it's designed to gather important information to help children with this condition.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Children's Oncology Group

Enrolment target

Start

06 Jul 2020

Estimated completion

01 Jul 2028

Philadelphia Chromosome Positive CML Accelerated Phase Chronic Myelogenous Leukemia Blastic Phase Chronic Myelogenous Leukemia Chronic Phase Chronic Myelogenous Leukemia

What is this study about?

This research study is about a medicine called Bosutinib, which is being tested for children and teenagers between 1 and 17 years old who have Chronic Myeloid Leukemia (CML). CML is a type of blood cancer, and 'Philadelphia chromosome-positive' means it has a specific genetic change. The study involves two main groups: those recently diagnosed with CML, and those whose CML has either not responded well to other treatments (called 'resistant') or who couldn't continue other treatments because of side effects (called 'intolerant').

The study has two phases. In the first phase, doctors will carefully increase the dose of Bosutinib to find the safest and most effective amount for children, similar to the dose that works for adults. They will also look at how children's bodies react to the medicine. The second phase will then use this recommended dose to see how well it works and how safe it is for a larger group of children with CML.

Finding new and effective treatments for children with CML is very important. This study helps move us closer to understanding if Bosutinib can be a good treatment option for this specific group of patients, particularly those who might not have responded well to other medicines in the past. The goal is to improve the health and well-being of young people living with CML.

Key takeaways

This study is for children and teenagers (1-17) with Philadelphia chromosome-positive CML.
It is testing a medicine called Bosutinib.
It includes those newly diagnosed, or those who didn't respond to previous treatments, or couldn't tolerate them.
The study aims to find the right dose, safety, and effectiveness of Bosutinib in young people.
Regular hospital visits and tests will be part of taking part.

Who may be eligible?

To join the first part of this study, participants must be between 1 and 17 years old. They need to have Philadelphia chromosome-positive CML that has either not responded well to at least one previous medicine (called a TKI) or they couldn't tolerate that previous medicine due to difficult side effects. We need to be sure of the CML diagnosis using specific blood and bone marrow tests.

If you're unable to take other CML medicines because of side effects, you might still be able to join, even if those medicines were working well. The doctors will decide if your side effects were significant enough to consider you for this study.

Later in the study, participants will also include those who have recently been diagnosed with CML and haven't had other treatments yet, as well as those with more advanced stages of CML (called accelerated or blast phase), provided they also have the Philadelphia chromosome and resistance or intolerance to previous treatments.

Quick self-check

Are you between 1 and 17 years old?
Do you have Philadelphia chromosome-positive CML?
Has your CML not responded well to previous treatments, or did you have significant side effects from them?
Are you willing to have regular blood tests and bone marrow checks?

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, the study will involve regular visits to the hospital. Doctors will perform tests, including blood tests and bone marrow checks, to monitor your CML and how you're responding to the Bosutinib. You'll take Bosutinib medicine by mouth once a day.

The study will carefully track any side effects you might experience and how the medicine works in your body. The overall length of time you'll be in the study will depend on how you respond to the treatment and your individual situation, but it will involve ongoing check-ups. The doctors and nurses will explain the full schedule of visits and assessments if you are considered for the study.

Potential risks and benefits

Taking part in any study has potential benefits and risks. The potential benefit is that Bosutinib might help treat your CML, especially if other treatments haven't worked or caused too many side effects. However, there's no guarantee it will work for everyone, and like all medicines, Bosutinib can have side effects, which the medical team will monitor closely. You have the right to withdraw from the study at any time, for any reason, without affecting your future medical care.

Locations (59)

Children's Hospital of Alabama
Birmingham, United States
Phoenix Childrens Hospital
Phoenix, United States
Arkansas Children's Hospital
Little Rock, United States
Kaiser Permanente Downey Medical Center
Downey, United States
Loma Linda University Medical Center
Loma Linda, United States
Kaiser Permanene-Oakland
Oakland, United States
Children's Hospital of Orange County
Orange, United States
Lucile Packard Children's Hospital Stanford University
Palo Alto, United States
Alfred I duPont Hospital for Children
Wilmington, United States
Golisano Children's Hospital of Southwest Florida
Fort Myers, United States
University of Florida Health Science Center - Gainesville
Gainesville, United States
Nemours Children's Hospital
Orlando, United States

+47 more sites — see the official record for the full list.

Common questions

What is CML?

CML stands for Chronic Myeloid Leukemia, a type of cancer that starts in the blood-forming cells of the bone marrow. 'Philadelphia chromosome-positive' means there's a specific genetic change in the cancer cells.

What is Bosutinib?

Bosutinib is a medicine being studied as a treatment for CML. It's a type of drug called a 'tyrosine kinase inhibitor' (TKI), which works by blocking signals that help cancer cells grow.

What does 'resistant' or 'intolerant' mean?

If your CML is 'resistant,' it means previous treatments haven't worked well enough. If you are 'intolerant,' it means you had severe side effects from previous CML medicines and couldn't continue taking them.

Will I get a placebo (dummy medicine)?

No, this study does not use a placebo. All participants will receive Bosutinib, the study medication.

How long will I be in the study?

The study will involve ongoing treatment and check-ups. The exact duration for you will depend on your response to the treatment and what your doctor advises.