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Study of ponatinib and azacitidine combination treatment in patients with accelerated phase or blast crisis chronic myelogenous leukemia

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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 treating patients with Chronic Myelogenous Leukemia that has progressed to an advanced stage called accelerated phase or myeloid blast crisis. The study evaluates a combination treatment using two medications: ponatinib (taken as tablets by mouth) and azacitidine (given as an injection under the skin).

The purpose of this research is to determine how long patients survive when treated with this drug combination over a two-year period. The treatment plan involves taking ponatinib tablets daily and receiving azacitidine injections for 7 days during each treatment cycle. The maximum daily dose of ponatinib is 45 mg, while azacitidine is given at 75 mg per square meter of body surface.

During the study, patients will be monitored for their response to treatment and any side effects they may experience. Doctors will regularly check blood samples and perform other medical tests to evaluate how well the treatment is working. The study will track various measures of treatment success, including changes in blood cell counts and the presence of cancer cells.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial medical assessment
A doctor will perform medical tests to confirm your eligibility for the study, including blood tests to check your liver and kidney function
You will need to undergo an electrocardiogram (ECG) test to check your heart rhythm
If you are a woman who can become pregnant, you will need to take a pregnancy test
Step 2
Starting treatment
You will receive two medications as part of your treatment:
Azacitidine will be given as an injection under your skin
Ponatinib will be given as tablets (15 mg or 30 mg) that you take by mouth
Step 3
Regular monitoring
Your doctor will regularly check your blood counts and monitor your response to treatment
You will have tests to check your liver and kidney function throughout the study
The medical team will monitor you for any side effects
Your bone marrow may be examined to check how well the treatment is working
Step 4
Long-term follow-up
The study will continue for up to 2 years
Your overall survival and response to treatment will be tracked during this period
The medical team will document any changes in your condition
If your disease improves, you may be evaluated for a possible bone marrow transplant

Who can join the trial?

12 criteria

Must be at least 18 years old
Must agree to use effective contraception throughout the study if able to have children
Must have recovered from previous cancer treatments (side effects must be mild or gone)
Must have signed informed consent form
Must have Philadelphia chromosome positive CML in blast crisis with at least 20% blast cells in bone marrow or blood (blast cells are immature blood cells)
Must have an ECOG performance status of 0-3 (a measure of daily living abilities)
Must have adequate kidney function (serum creatinine no more than 1.5 times the normal limit)
check
Must have adequate liver function, measured by:
- Bilirubin levels no more than 1.5 times normal
- Liver enzymes (ALT and AST) no more than 2.5 times normal, or 5 times if related to disease
- Must have normal pancreas function (normal lipase and amylase levels)
- Must have normal heart rhythm on ECG (QTcF interval no more than 450ms for males or 470ms for females)
- Women who can become pregnant must have a negative pregnancy test before starting

Who cannot join the trial?

14 criteria

Age under 18 years old
Pregnant or breastfeeding women
Previous participation in another clinical trial within the last 30 days
Known allergy or hypersensitivity to ponatinib or 5-azacitidine (the study medications)
Severe liver problems (liver failure)
Severe kidney problems (kidney failure)
Uncontrolled heart conditions
Active, uncontrolled infections
Other types of cancer that require treatment
Mental conditions that could interfere with following study procedures
Unable to give informed consent
History of non-compliance with medical treatments
Life expectancy less than 3 months
Any condition that, in the opinion of the study doctor, would make participation unsafe

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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
Verified
Besancon University Hospital Center
Besançon, France
Responsive
CHRU De Nancy
Vandoeuvre Les Nancy, France

Trial locations

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Investigated drugs

Ponatinib is a targeted therapy medication used to treat certain types of leukemia. It works by blocking specific proteins that cause cancer cells to grow and multiply. This medication is taken orally and helps control the growth of cancer cells in patients with chronic myelogenous leukemia. 5-Azacitidine is a chemotherapy medication that affects how cells grow and divide. It is commonly used to treat blood disorders and certain types of blood cancers. This medication works by helping bone marrow produce normal blood cells and by killing abnormal cells in the blood. It is typically given as an injection under the skin or through an intravenous line. The combination of these two medications is being studied to treat patients with chronic myelogenous leukemia that has progressed to more advanced stages (accelerated phase or blast crisis).

What is already known about the treatment

Ponatinib
An oral medication used in the treatment of chronic myelogenous leukemia (CML), particularly in its accelerated phase or blast crisis. This targeted therapy works by blocking specific proteins (tyrosine kinases) that are responsible for the abnormal growth of cancer cells, effectively slowing down or stopping the progression of leukemia. The drug belongs to the class of tyrosine kinase inhibitors and is typically administered as tablets taken once daily.
5-Azacitidine
A chemotherapy medication administered via subcutaneous injection or intravenous infusion, used in treating various blood cancers including chronic myelogenous leukemia. This drug works by interfering with DNA production in cancer cells and helps restore normal blood cell production in the bone marrow. It belongs to the class of medications called hypomethylating agents and is typically given in treatment cycles with specific days of administration followed by rest periods.

Investigated diseases

Chronic Myelogenous Leukemia – A type of blood cancer that begins in the bone marrow and affects blood cell production. The disease develops when bone marrow cells that produce white blood cells begin to grow and function abnormally. It typically progresses through three phases: chronic phase, accelerated phase, and blast crisis phase. In the accelerated phase, there is an increase in abnormal blood cells, and the disease becomes more aggressive. The blast crisis phase is the most advanced stage, where immature blood cells (blasts) multiply rapidly in the bone marrow and blood. During these advanced phases, the bone marrow produces excessive amounts of abnormal white blood cells that don't mature properly.