Active not recruitingPHASE3INTERVENTIONAL

A Study of Oral Asciminib Versus Other TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP

This study is testing a new medicine called asciminib for adults recently diagnosed with chronic myeloid leukaemia (CML). CML is a type of blood cancer linked to a specific change in chromosomes, called the Philadelphia chromosome. Researchers want to compare asciminib against other common CML medicines like imatinib, nilotinib, bosutinib, or dasatinib. The main goal is to find out if asciminib is more effective for these patients. Participants will be randomly assigned to either asciminib or one of the other standard treatments. The study also includes periods to see if patients can stop treatment successfully and what happens if their cancer returns after stopping. This is a big study to help improve treatments for CML.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

405

Start

06 Oct 2021

Estimated completion

18 Jan 2031

Chronic Myeloid Leukemia (CML) Philadelphia Chromosome Positive

Results

Results from this study

Posted March 2025

Results have been published for this study.

Primary outcome

Percentage of Participants With Major Molecular Response (MMR) at Week 48 - Ascimimib vs. Investigator Selected TKI

Molecular response is assessed using BCR-ABL1 transcript levels measured by realtime quantitative polymerase chain reaction. MMR is defined as a ratio BCR-ABL/ABL ≤0.1% on the international scale (ie, at least 3 log reduction from a standardized baseline value).

Full results on the registry

What is this study about?

Imagine you've just been diagnosed with a type of blood cancer called Chronic Myeloid Leukaemia, or CML. This means there's a problem with some of your blood cells, often due to a specific change in your chromosomes called the Philadelphia chromosome. Doctors already have medicines called TKIs (Tyrosine Kinase Inhibitors) that work well for CML. This study is like a careful test to see if a newer medicine, asciminib, might be an even better first treatment for people newly diagnosed with CML.

The study aims to compare asciminib against other commonly used CML medicines like imatinib, nilotinib, dasatinib, or bosutinib. You won't get to choose which one you receive, as this is decided randomly, a bit like flipping a coin. This helps make sure the study results are fair and reliable. The main goal is to understand if asciminib is more effective at treating CML than the existing treatments. This is important because finding better first-line treatments can make a big difference in a patient's journey.

This study is designed in three main parts over several years. First, everyone receives their assigned treatment. For some people who respond very well, there might be a chance to carefully stop treatment to see if their body can keep the cancer under control without daily medicine. If the cancer does come back after stopping, there's a plan to restart treatment. This careful approach helps researchers learn not just about new medicines, but also about the possibility of taking treatment breaks, which can improve quality of life.

Key takeaways

Tests a new CML medicine, asciminib, against current standard treatments.
For adults newly diagnosed with a specific type of CML (Philadelphia chromosome positive).
You'll be randomly assigned to receive either asciminib or one of the other CML drugs.
Involves regular medical check-ups and blood tests.
Some participants might try stopping treatment later if very successful.
Your health will be carefully monitored throughout the study.

Who may be eligible?

To join this study, you need to be an adult, at least 18 years old, and have been diagnosed with a specific type of CML (Philadelphia chromosome positive) fairly recently, within the last three months. Your CML must be in what doctors call the 'chronic phase,' which means it's not too advanced, with specific levels of certain cells in your blood and bone marrow.

You'll also need to be generally in good health, meaning you can carry out most of your daily activities without too much trouble. Your doctors will check that your kidneys, liver, and other body functions are working well through blood tests. Specific blood levels for things like potassium, calcium, and magnesium also need to be within a healthy range, or corrected with supplements if needed.

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 18 years old or older?
Have you been diagnosed with CML (Philadelphia chromosome positive) in the last three months?
Is your CML in the 'chronic phase'?
Are you generally in good health and able to manage daily activities?
Do your recent blood tests show healthy kidney and liver function, and balanced minerals?
Have you not received any previous treatment for your CML?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be assigned, by chance, to one of two groups. One group will take the new medicine, asciminib, as a tablet once a day, usually before food. The other group will take one of the standard CML medicines (imatinib, nilotinib, dasatinib, or bosutinib) as a tablet, typically once or twice a day, often with food. The exact duration of your treatment will depend on how you respond and what the study team decides.

You'll have regular hospital visits for check-ups, blood tests, and scans to monitor your health and how well the treatment is working. If you respond very well, you might be able to try stopping treatment later on. If you do stop and your CML comes back, you'll restart treatment. The study involves long-term follow-up, which means keeping in touch with the study team even after you've finished the main treatment, to see how you're doing over time.

Potential risks and benefits

Every medical treatment has potential benefits and potential risks. The possible benefit of joining this study is that you might receive a new treatment, asciminib, which could be more effective than current standard options for CML. You'll also receive careful monitoring and support from a dedicated medical team. However, all medicines can cause side effects. Asciminib and the other CML drugs can have various side effects, which your doctor will discuss with you. These can range from mild to more serious, and there's no guarantee that any treatment will work perfectly for everyone. Remember, your participation is voluntary, and you can withdraw from the study at any time without affecting your future medical care.

Locations (110)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Rocky Mountain Cancer Centers
Verified postcode
Denver, United States
Florida Cancer Specialists
Verified postcode
Fort Myers, United States
Florida Cancer Specialists Pan
Verified postcode
Tallahassee, United States
University of Kentucky
Verified postcode
Lexington, United States
Uni of Massachusetts Medical Center
Verified postcode
Worcester, United States
Wake Forest University Baptist Medical Center
Verified postcode
Winston-Salem, United States
Williamette Cancer Center
Verified postcode
Eugene, United States
Oregon Health Sciences University
Verified postcode
Portland, United States
Avera Cancer
Verified postcode
Sioux Falls, United States
Chattanooga Onc And Hem Assoc PC
Verified postcode
Chattanooga, United States
Texas Oncology
Verified postcode
Amarillo, United States
Texas Oncology-Baylor USO
Verified postcode
Dallas, United States

Common questions

What is CML?

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

What is a TKI?

TKI stands for Tyrosine Kinase Inhibitor. These are a type of medicine that specifically targets the abnormal cells in CML, helping to control the disease.

Will I definitely get the new medicine?

No, it's decided by chance, like a lottery. You'll either get asciminib or one of the standard CML medicines.

Can I stop treatment if I feel better?

For some people who respond very well, there might be a chance to carefully stop treatment later in the study, but this is under strict medical guidance.

How long will the study last for me?

The treatment period might last for several years, followed by long-term monitoring, depending on how you respond. Your doctor will provide more details.