Study of Ponatinib for Patients with Resistant Chronic Myeloid Leukemia

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What is this study about?

This clinical trial is focused on studying a type of blood cancer called Chronic Myeloid Leukemia (CML), specifically in its chronic phase. The study is investigating the effectiveness and safety of a medication called Ponatinib, which is also known by the brand name Iclusig. Ponatinib is a type of treatment known as a kinase inhibitor, which works by blocking certain proteins that help cancer cells grow. The trial involves patients who have a specific genetic marker, known as the T315I mutation, or those who have not responded well to previous treatments with other kinase inhibitors.

The purpose of the study is to evaluate how well Ponatinib works when given in different doses: 45 mg, 30 mg, and 15 mg daily. Participants will take the medication in the form of film-coated tablets, which are taken orally. The study will monitor the participants over a period to see how their condition responds to the treatment. The goal is to see if the medication can reduce the levels of a specific protein associated with CML, known as BCR-ABL1, to a very low level within 12 months. This protein is a marker used to measure the presence of cancer cells in the body.

Throughout the study, the safety of Ponatinib will also be assessed by observing any side effects or adverse reactions that participants may experience. The trial aims to provide valuable information on the best dosage of Ponatinib for treating patients with resistant CML, helping to improve treatment options for those affected by this condition.

1 Joining the study

Upon joining the study, the patient will be assigned to one of three starting doses of ponatinib: 45 mg, 30 mg, or 15 mg. This medication is taken orally in the form of film-coated tablets.

The purpose of the study is to evaluate the effectiveness and safety of these doses in patients with chronic phase chronic myeloid leukemia (CP-CML) who are resistant to previous treatments or have a specific genetic mutation known as T315I.

2 Medication administration

The patient will take the assigned dose of ponatinib daily. The medication is administered orally, meaning it is taken by mouth.

The study will monitor the patient’s response to the medication over a period of 12 months, with the goal of achieving a specific reduction in the BCR-ABL1IS level, which is a marker used to measure the effectiveness of the treatment.

3 Monitoring and assessments

Throughout the study, the patient will attend scheduled visits for monitoring and assessments. These visits will include various tests to evaluate the patient’s response to the treatment and to monitor for any side effects.

The primary goal is to achieve a BCR-ABL1IS level of less than or equal to 1% at 12 months. Secondary goals include measuring molecular and cytogenetic response rates, as well as monitoring the safety and tolerability of the treatment.

4 Completion of the study

The study is expected to conclude by June 30, 2025. At the end of the study, the patient’s overall response to the treatment will be evaluated, including any changes in their condition and any side effects experienced.

The results will help determine the most effective and safe dose of ponatinib for patients with CP-CML who are resistant to previous treatments or have the T315I mutation.

Who Can Join the Study?

Have Chronic Phase Chronic Myeloid Leukemia (CP-CML) and have received at least two prior treatments with a type of medication called TKI (Tyrosine Kinase Inhibitor) and shown resistance to treatment, or have a documented history of a specific genetic change called T315I mutation after receiving any number of prior TKI treatments.
Be at least 18 years old.
Have a performance status of 0, 1, or 2 on a scale used to assess how a disease affects a patient’s daily living abilities, known as the Eastern Cooperative Oncology Group (ECOG) performance status.
Have adequate kidney function, which means certain blood tests show that the kidneys are working well enough.
Have adequate liver function, which means certain blood tests show that the liver is working well enough.
Have normal pancreatic status, which means certain blood tests show that the pancreas is working well enough.
Have a normal QT interval on an electrocardiogram (ECG), which is a test that checks the heart’s electrical activity.
For females who can have children, have a negative pregnancy test before joining the study.
Agree to use a highly effective form of birth control with sexual partners from the start of the study through at least 4 months after the end of treatment, for both male and female patients who can have children.
Provide written informed consent, which means agreeing to participate in the study after being fully informed about it.
Be willing and able to follow the scheduled visits and study procedures.
Have recovered from any side effects related to previous cancer treatments to a certain level, as defined by a standard called NCI CTCAE v 4.0 grade 1 or lower.

Who Cannot Join the Study?

Patients who have a different type of leukemia other than Chronic Phase Chronic Myeloid Leukemia (CP-CML) cannot participate. CP-CML is a specific stage of a blood cancer called chronic myeloid leukemia.
Patients who are not resistant to previous TKI therapy cannot participate. TKI therapy refers to a treatment using drugs called tyrosine kinase inhibitors, which are used to treat certain types of cancer.
Patients who do not have the T315I mutation cannot participate. This mutation is a specific change in the cancer cells that can affect how the disease responds to treatment.
Patients who are not within the specified age range cannot participate. The age range is typically defined by the study and includes certain age groups.
Patients who are not part of the specified clinical trial group cannot participate. This refers to the specific group of patients the study is focusing on.
Patients who are not male or female cannot participate. The study includes both male and female participants.
Patients who are not considered part of a vulnerable population cannot participate. Vulnerable populations may include groups like children, pregnant women, or others who need special protection in research.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Uniwersytecki Szpital Kliniczny Im Jana Mikulicza Radeckiego We Wroclawiu	Wroclaw	Poland

Other Sites

Site Name	City	Country	Status
Wkiwudafjgk Wxvoqmvdfohqodajvisv Ceswelz Ogbtwygok I Tgqokoclbfxtk Id Magmiqobdod W Ldrdb	Lodz	Poland

Trial status

Country	Status	Recruitment Start
France	Not recruiting	09.10.2015
Poland	Not recruiting	09.10.2015

Trial locations

Investigated drugs:

Ponatinib

Ponatinib is a medication used in this trial to treat patients with chronic phase chronic myeloid leukemia (CP-CML) who have not responded to previous treatments or have a specific genetic mutation known as T315I. The trial aims to understand how effective and safe ponatinib is when given in different starting doses. Ponatinib works by blocking certain proteins that help cancer cells grow, which may help to control the disease.

Chronic Phase Chronic Myeloid Leukemia – Chronic Phase Chronic Myeloid Leukemia (CP-CML) is a type of cancer that affects the blood and bone marrow. It is characterized by the overproduction of immature white blood cells, known as myeloid cells. In the chronic phase, the disease progresses slowly, and patients may not experience significant symptoms initially. Over time, the accumulation of these cells can lead to symptoms such as fatigue, weight loss, and an enlarged spleen. The disease is associated with a specific genetic abnormality known as the Philadelphia chromosome, which results in the production of an abnormal protein that promotes cancer cell growth. If left untreated, CP-CML can progress to more aggressive phases, known as the accelerated phase and blast phase.

Trial ID:

2024-514516-27-00

Protocol code:

AP24534-14-203

NCT ID:

NCT02467270

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of Ponatinib for Patients with Resistant Chronic Myeloid Leukemia

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