RecruitingPHASE1, PHASE2INTERVENTIONAL

Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia

This study is investigating a new medicine called asciminib for children and teenagers aged 1 to 17 who have a blood cancer called Chronic Myeloid Leukemia (CML). This particular type of CML has a specific genetic marker called 'Philadelphia chromosome positive'. The study focuses on patients who have already been treated with other similar medicines (TKIs) but these haven't worked well enough or caused too many side effects. The main goals are to find the safest and most effective dose of asciminib for children of different ages, and to understand how their bodies process the medicine. Early results suggest this medicine works similarly in children and adults with CML, which means it might be a good option for younger patients too.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Novartis Pharmaceuticals

Enrolment target

Start

27 Dec 2021

Estimated completion

01 Nov 2031

Myeloid Leukemia, Philadelphia Positive

What is this study about?

This study is looking into a medicine called asciminib for children and teenagers with a type of blood cancer known as Chronic Myeloid Leukemia (CML). Specifically, it's for those whose CML has a particular genetic change called the 'Philadelphia chromosome positive'. The study includes young people aged between 1 and under 18 years old.

The main aim is to figure out the best dose of asciminib for different age groups in children and teenagers, and to make sure it's safe. It's especially for those who have already tried other similar CML medicines (called TKIs) but found they didn't work well enough or caused too many problems. Scientists believe that CML works in a similar way in children as it does in adults. This means if the medicine works for adults, it might also work for children, but it's important to find the right amount for their growing bodies.

Researchers are using two main groups: one group will receive a special liquid version of the medicine designed for children, and another group (older teenagers) might start with the adult tablet version. They will carefully check how the medicine is absorbed, how long it stays in the body, and its effects, to make sure children get the same benefits as adults with a safe dose.

Key takeaways

The study is for children and teenagers (1-17 years old) with a specific type of CML.
It's for those whose previous CML treatments (TKIs) were not effective or had too many side effects.
The main goals are to find the right dose of asciminib for different ages and check its safety.
Participants will receive the study medicine and have regular medical check-ups and tests.
The research aims to help both current and future children with CML.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To be able to join this study, children and teenagers generally need to be between 1 and under 18 years old. If they are getting the special children's liquid medicine, they can be as young as 1. If they are older teenagers (14 to under 18) and weigh at least 40 kg (about 6 stone 4 pounds), they might start on the adult tablet version.

All participants must have a specific type of CML that is Philadelphia chromosome positive and have already tried at least one other CML medicine (TKI) which didn't work well or caused side effects. They must be generally well enough to take part, with good organ function (like kidneys, liver, heart), and their blood test results need to be within certain ranges. There must also be clear evidence of their CML at a genetic level.

Some reasons a child or teenager might not be able to join include having a specific CML gene change called T315I, or if their CML has worsened to a more advanced stage after getting better previously. The study team will review all medical history to determine if someone is suitable.

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 your child between 1 and under 18 years old?
Does your child have 'Philadelphia chromosome positive' CML?
Has your child already tried at least one other CML medicine (TKI) that didn't work well or caused problems?
Does your child generally feel well enough and have good organ function (heart, liver, kidneys)?
Does your child not have a specific CML gene change called T315I?
Has your child's CML not progressed to a more advanced stage in the past?

Answer every question to see your result.

What does participation involve?

If you join this study, your child will receive the study medicine, asciminib, either as a special liquid formulation for younger children or as an adult tablet for older teenagers. The dose will be carefully determined by the study doctors based on age and weight, and your child will take it by mouth, usually twice a day. Food intake around the medicine might be important for some groups.

Participation will involve regular hospital visits for check-ups, blood tests, and possibly bone marrow tests, to monitor how your child's body handles the medicine and how their CML is responding. This is also to check for any side effects. These visits will be more frequent initially and then become less frequent over time. While the exact duration of treatment can vary, the study will involve multiple stages, with careful monitoring throughout.

The study aims to discover the best and safest dosing strategy for children. This means that for some participants, especially in the early parts of the study, the dose might be adjusted up or down based on how their body reacts and to ensure they receive a safe and effective amount.

Potential risks and benefits

Taking part in any study involves potential benefits, risks, and responsibilities. A potential benefit of this study is that your child might receive a new medicine that could help manage their CML, especially if other treatments haven't worked well. This research could also help future children with CML by providing important information about effective treatments. However, like all medicines, asciminib can have side effects, which the study team will explain in detail. Some common side effects in adult studies have included tiredness, upset stomach, and changes in blood test results. There might also be unknown risks, as this is a new medicine for children. You are free to withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (39)

Indiana UH Riley H for CIU
Verified postcode
Indianapolis, United States· Recruiting
Dana Farber Cancer Institute
Verified postcode
Boston, United States· Recruiting
University of Mississippi Medical Center
Verified postcode
Jackson, United States· Recruiting
Columbia University Medical Center New York Presbyterian
Verified postcode
New York, United States· Recruiting
Cinn Children Hosp Medical Center
Verified postcode
Cincinnati, United States· Recruiting
Childrens Hospital of Philadelphia
Verified postcode
Philadelphia, United States· Recruiting
Uni Of Texas MD Anderson Cancer Ctr
Verified postcode
Houston, United States· Recruiting
University Of Utah
Verified postcode
Salt Lake City, United States· Recruiting
Novartis Investigative Site
Verified postcode
Hangzhou, China· Recruiting
Novartis Investigative Site
Verified postcode
Beijing, China· Recruiting
Novartis Investigative Site
Verified postcode
Shanghai, China· Recruiting
Novartis Investigative Site
Verified postcode
Tianjin, China· Recruiting

Common questions

What is CML?

CML stands for Chronic Myeloid Leukemia. It's a type of cancer that affects the blood and bone marrow, where blood cells are made.

What is asciminib?

Asciminib is a medicine being tested to treat CML. It works by targeting a specific protein involved in the growth of CML cells.

Why are they studying this medicine in children?

Researchers want to find out if asciminib can help children and teenagers with CML, especially if other medicines haven't worked, and to determine the safest dose for young people.

Will my child have to take tests?

Yes, your child will have regular check-ups, blood tests, and possibly bone marrow tests to see how they are doing and if the medicine is working.

What if we change our mind?

You can decide to withdraw your child from the study at any time, for any reason, and it won't affect their regular medical care.

How to find out more

Novartis Pharmaceuticals

novartis.email@novartis.com 1-888-669-6682 Official trial record

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