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Study of Asciminib treatment for patients with Chronic Myeloid Leukemia who have T315I mutation and cannot take or tolerate ponatinib

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on patients with Chronic Myeloid Leukemia (CML) who have a specific genetic change called T315I mutation. The study includes patients whose disease is either in the chronic phase or accelerated phase. These patients have either not responded well to a medication called ponatinib, cannot tolerate it, or are not eligible to receive it.

The treatment being tested is a medication called asciminib (also known as Scemblix or ABL001). This medication comes in the form of film-coated tablets that are taken by mouth. The study aims to determine how well patients respond to asciminib after 12 months of treatment.

During this open-label study, all participants will receive asciminib tablets. The treatment may continue for up to 48 months, with regular check-ups to monitor how well the medication is working and to watch for any side effects. The amount of medication can be adjusted based on how each patient responds to the treatment.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Your eligibility for the study will be evaluated based on specific medical criteria, including confirmation of Chronic Myeloid Leukemia (CML) diagnosis and presence of T315I mutation
Medical history review will be performed to confirm previous treatments and response to them
Step 2
Treatment initiation
You will receive Scemblix (asciminib) 40 mg film-coated tablets for oral use
The medication needs to be taken daily according to the prescribed schedule
Step 3
3-month evaluation
Blood tests will be performed to measure the level of disease markers (BCR::ABL1)
Assessment of any side effects or health changes
Step 4
6-month evaluation
Follow-up blood tests to monitor disease markers
Continued assessment of treatment response and any side effects
Step 5
9-month evaluation
Regular monitoring of disease markers through blood tests
Ongoing assessment of treatment effectiveness and safety
Step 6
12-month evaluation
Major evaluation point to assess treatment response
Comprehensive review of disease markers and overall health status
Step 7
Long-term follow-up
Continued monitoring at months 18 and 24
Regular assessment of disease status and treatment effectiveness
Monitoring of long-term health outcomes and any side effects

Who can join the trial?

11 criteria

Must sign an informed consent form before participating in the study
Must be at least 18 years old, male or female
Must have been diagnosed with CML-CP (Chronic Myeloid Leukemia in Chronic Phase) or CML-AP (Accelerated Phase) with confirmed T315I mutation after at least one previous treatment
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For CML-CP patients must meet all these blood criteria:
- Less than 15% blast cells in blood and bone marrow
- Less than 30% combined blasts and promyelocytes in blood and bone marrow
- Less than 20% basophils in blood
- Platelet count of at least 100,000 per cubic millimeter
- No spread of leukemia outside blood and bone marrow (except enlarged liver or spleen)
- Must not have previously received treatment with asciminib or similar medications
- Must have either not responded well to (failed) or been unable to tolerate (intolerant) a medication called ponatinib, or be unsuitable for ponatinib treatment
- Must have evidence of specific genetic markers (BCR::ABL1 transcripts) that can be measured through laboratory testing

Who cannot join the trial?

15 criteria

History of severe allergic reactions to similar medications
Pregnancy or breastfeeding
Uncontrolled heart conditions including heart failure, irregular heartbeat, or recent heart attack
Severe liver problems or abnormal liver function tests
Severe kidney problems requiring dialysis
Active, uncontrolled infections
Other types of cancer that require treatment
Mental health conditions that could affect ability to follow study requirements
Taking certain medications that could interact with the study drug
Participation in other clinical trials within the past 30 days
Blood disorders not related to CML (Chronic Myeloid Leukemia)
Uncontrolled high blood pressure
History of stroke or bleeding in the brain
Digestive system problems that could affect drug absorption
Known HIV, hepatitis B, or hepatitis C infection

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Assistance Publique Hopitaux De Paris
Paris, France
Responsive
CHRU De Nancy
Vandoeuvre Les Nancy, France
Verified
Centre Hospitalier Universitaire De Lille
Lille, France

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Investigated drugs

Asciminib
is a medication used to treat patients with chronic myeloid leukemia (CML). It works by targeting specific genetic mutations in leukemia cells, particularly in patients who have developed a specific mutation called T315I. This medication is designed for patients who cannot take or have not responded well to another medication called ponatinib.
Ponatinib
is a reference medication in this trial, though not actively used. It is a type of cancer medication that is typically used to treat chronic myeloid leukemia, but some patients may be resistant to it, unable to tolerate it, or not eligible to receive it.

Asciminib Hydrochloride

What is already known about the treatment

Asciminib
A targeted therapy medication administered orally that is specifically designed to treat Chronic Myeloid Leukemia (CML) in patients with the T315I mutation who cannot use or tolerate other treatments like ponatinib. This drug works by selectively binding to the ABL1 component of the BCR-ABL1 protein in a unique way, making it effective even when other treatments fail due to resistance. As a kinase inhibitor, it blocks specific cellular signals that drive leukemia cell growth, and it represents a significant advancement in precision medicine for leukemia treatment with ongoing clinical trials showing promising results.
Ponatinib
An oral medication belonging to the tyrosine kinase inhibitor class that is used to treat patients with Chronic Myeloid Leukemia, particularly those with the T315I mutation. This drug works by blocking several protein kinases involved in cancer cell growth and survival, including the BCR-ABL1 protein that drives leukemia development. While effective against resistant forms of CML, ponatinib requires careful monitoring due to its cardiovascular risk profile, and it is typically reserved for patients who have limited treatment options or have not responded to other therapies.

Investigated diseases

Chronic Myeloid Leukemia (CML) - A type of blood cancer that begins in the bone marrow, causing the body to produce too many white blood cells. The disease typically progresses through three phases: chronic phase (CP), accelerated phase (AP), and blast phase. In the chronic phase, there are increased numbers of white blood cells, but they still function normally. The accelerated phase shows more immature blood cells and more severe symptoms. The T315I mutation is a specific genetic change that makes some CML cases resistant to certain treatments. Patients with this mutation have a distinct form of CML that requires specialized medical attention.