Active not recruitingPHASE3INTERVENTIONAL

A Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP)

This Phase 3 study investigated two oral treatments, asciminib and nilotinib, for adults aged 18 and over who were recently diagnosed with a specific type of blood cancer called Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML). The main goal was to see which drug was better tolerated, meaning caused fewer side effects or less severe ones. Participants were randomly assigned to receive either asciminib or nilotinib. The study had several stages, including an initial treatment phase and an optional phase where patients might stop treatment if their cancer was well controlled, to see if they could remain cancer-free without medication. Key eligibility included a new diagnosis of CML in its chronic phase and good general health.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

568

Start

21 Nov 2022

Estimated completion

07 Jul 2031

Philadelphia Chromosome-Positive Chronic Myeloid Leukemia

Results

Results from this study

Posted May 2026

Results have been published for this study.

Primary outcome

Time to Discontinuation of Study Treatment Due to Adverse Event (TTDAE) (From Interim Analysis)

TTDAE is defined as the interval from the date of first study treatment administration to the date of discontinuation of study treatment due to an adverse event (AE). The comparison between the asciminib and nilotinib treatment arms is performed using a cause-specific hazard model, in which discontinuations due to AE are considered the event of interest and discontinuations for reasons other than AE are treated as competing risks. The number of participants who discontinued study treatment due to AE within the specified timeframe is presented in the results table. The formal comparison between treatment arms is performed using a cause-specific hazard model, and the corresponding hazard ratio, confidence interval, and p-value are provided in the Statistical Analysis section. The TTDAE endpoint is event-driven by counting how many participants have experienced treatment discontinuations due to AE.

Full results on the registry

What is this study about?

This research study is looking at two different medicines, asciminib and nilotinib, for people who have recently been diagnosed with a type of blood cancer called Philadelphia chromosome-positive chronic myeloid leukemia (CML). You might hear it called Ph+ CML. This type of CML means there's a specific change in your chromosomes that leads to the cancer.

The main aim of the study is to compare how well people tolerate each of these medicines. "Tolerability" basically means how easy it is for patients to take the drug without experiencing too many bothersome side effects. Doctors want to find treatments that are not only effective but also allow people to maintain a good quality of life. Understanding tolerability helps doctors decide which treatment might be best for different patients.

Participants in the study were randomly assigned to receive either asciminib or nilotinib. This is like flipping a coin to ensure a fair comparison between the two drugs. The study also had longer-term phases. For some patients whose cancer was well-controlled, there was an optional period where they might try to stop their medication altogether to see if their CML remained in remission without continuous treatment. This is a very important part of CML research, as reducing or stopping lifelong medication could significantly improve quality of life for many.

Key takeaways

Researched two oral drugs (asciminib, nilotinib) for newly diagnosed CML.
Main goal was to compare how well patients tolerated each drug.
Included an optional phase to try stopping treatment if cancer was controlled.
Participants were adults, 18+, with a specific type of CML.
Study aimed to find treatments that are effective and well-tolerated.

Who may be eligible?

To be considered for this study, you would generally need to be an adult, 18 years or older, with a new diagnosis of Philadelphia chromosome-positive chronic myeloid leukemia (CML) in its chronic phase. This means your CML diagnosis should have been made within the last three months, and specific tests would confirm the type and stage of your cancer, showing it's not advanced.

Your general health would also be assessed. You would need to have certain blood counts and organ functions (like liver and kidney) within a healthy range. Researchers also looked for a specific genetic marker (BCR::ABL1 transcript) in your blood, which is characteristic of this type of CML and helps them monitor the disease. Your ability to carry out daily activities would also be checked, ensuring you are well enough to participate.

Essentially, the study was for newly diagnosed adults with a specific, less aggressive form of CML (chronic phase) who were otherwise in reasonably good health. This helps ensure that the study results are clear and that participants can safely take part.

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 an adult aged 18 or over?
Have you been diagnosed with Philadelphia chromosome-positive CML within the last 3 months?
Is your CML in its 'chronic phase' (not advanced)?
Are your liver, kidney, and blood tests generally within a healthy range?
Are you generally able to carry out your daily activities without much difficulty?

Answer every question to see your result.

What does participation involve?

If you had joined this study, you would first have given your informed consent, meaning you understood and agreed to take part. After that, you would have been randomly assigned to receive one of the two study medications, either asciminib or nilotinib, both taken by mouth.

The study had three main stages: a treatment phase, where you would regularly take the assigned medication and have check-ups. For those whose cancer was well-controlled, there was an optional 'treatment-free remission' phase, where you might have stopped medication for up to two years. During this time, you'd still have regular monitoring. If your cancer showed signs of returning, or if you and your doctor decided, you would then enter a 'treatment re-initiation' phase and restart medication. The total duration of participation would vary depending on which phases you entered.

Potential risks and benefits

Participating in a clinical trial like this offers potential benefits, such as access to new medications that aren't widely available or more intensive monitoring of your health. However, there are also potential risks, as any medication can cause side effects. Researchers carefully monitor participants to manage any side effects, but some could be serious or unexpected. It's also possible that the study treatment might not be effective for your specific condition. Throughout the study, participants have the right to withdraw at any time, for any reason, without affecting their future medical care.

Locations (120)

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
Illinois Cancer Care
Verified postcode
Peoria, United States
Regions Hospital
Verified postcode
Saint Paul, United States
Messino Cancer Centers
Verified postcode
Asheville, United States
Oncology Hematology Care Inc
Verified postcode
Cincinnati, United States
Williamette Cancer Center
Verified postcode
Eugene, United States
Texas Oncology P A
Verified postcode
Bedford, United States
Texas Oncology PA Bedford
Verified postcode
Bedford, United States
Virginia Oncology Associates
Verified postcode
Norfolk, United States
Novartis Investigative Site
Unverified
CABA, Argentina
Novartis Investigative Site
Verified postcode
Buenos Aires, Argentina
Novartis Investigative Site
Verified postcode
Buenos Aires, Argentina

Common questions

What is Philadelphia chromosome-positive CML?

It's a type of blood cancer where your body makes too many white blood cells. It's linked to a specific change in your chromosomes called the Philadelphia chromosome.

What does "tolerability" mean in this study?

Tolerability refers to how well patients can take a medication. It's about monitoring how many side effects occur, how severe they are, and if people need to stop treatment because of them.

Why were people randomly assigned to treatments?

Random assignment (like flipping a coin) helps ensure that the groups receiving each treatment are as similar as possible, making the comparison between the medicines fair and reliable.

Could I stop treatment if my cancer was under control?

Yes, if your cancer was well-controlled and met specific criteria, you could enter an optional phase to try stopping medication while being closely monitored.

What if I wanted to leave the study early?

You always have the right to withdraw from a clinical study at any point, and doing so would not affect your standard medical care in the future.