Study on Ponatinib for Achieving Treatment-Free Remission in Patients with 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 treatment being tested is a medication called Ponatinib, which is taken as a film-coated tablet. Ponatinib is a type of drug known as a tyrosine kinase inhibitor, which works by blocking certain proteins that help cancer cells grow.

The purpose of the study is to assess patients who have achieved a specific level of response, known as MR4, after two years of treatment with Ponatinib. MR4 is a term used to describe a deep molecular response, indicating a very low level of cancer cells in the body. The study will follow participants over a period of 24 months to monitor their response to the treatment. During this time, researchers will evaluate the molecular status of the disease, the safety and tolerability of Ponatinib, and any side effects that may occur. Additionally, the study will look at the quality of life of participants while on the treatment.

Throughout the study, researchers will also identify factors that may be associated with achieving MR4 or better, such as risk scores and the duration of previous treatments. The study aims to provide insights into the potential for treatment-free remission, where patients may be able to stop treatment while maintaining their response. Overall survival and progression-free survival will also be evaluated to understand the long-term benefits of Ponatinib therapy.

1 initiation of treatment

Upon joining the study, the patient begins treatment with ponatinib, which is administered in the form of Iclusig 15 mg film-coated tablets.

The medication is taken orally, following the prescribed dosage and schedule provided by the healthcare team.

2 treatment phase

The patient continues to take ponatinib for a duration of 24 months.

During this period, the patient’s response to the treatment is monitored, focusing on achieving a molecular response known as MR4.

3 evaluation of molecular status

Throughout the treatment, the patient’s molecular status is evaluated at scheduled visits.

The evaluations include checking for different levels of response, such as no MMR (major molecular response), MMR, MR4, and MR4.5.

4 monitoring safety and quality of life

The safety profile and tolerability of ponatinib are assessed regularly to identify any adverse events.

The patient’s health-related quality of life is also monitored to ensure well-being during the treatment.

5 identification of factors for response

Clinical and biological factors associated with achieving MR4 or better are identified.

Factors such as risk scores, gender, duration of previous treatments, and molecular level at study entry are considered.

6 evaluation of survival outcomes

The study evaluates overall survival and progression-free survival as part of the treatment outcomes.

The potential for a treatment-free remission (TFR) approach is also assessed.

Who Can Join the Study?

  • Must be a female or male who is 18 years of age or older.
  • Must have chronic myeloid leukemia (CML) in the chronic phase. This is a type of blood cancer that is in a specific stage.
  • Must have a BCR-ABL IS level between 0.5-0.01. This is a specific measurement from a blood test called PCR that checks for certain genetic markers.
  • Must not have achieved MR4 or a stable MR4 after at least 3 years of treatment with certain medications like nilotinib, dasatinib, or bosutinib. MR4 is a specific level of response to treatment, indicating a very low level of cancer cells.
  • Must have Philadelphia chromosome and/or BCR-ABL fusion gene positive CML. These are specific genetic changes found in the cancer cells.
  • Must have had an eye examination, including a check of the back of the eye (fundoscopy), by an eye doctor within 8 weeks before starting the treatment.

Who Cannot Join the Study?

  • Patients who have not achieved MR4 after 2 years of treatment with ponatinib. MR4 means a specific level of response to treatment where the disease is very well controlled.
  • Patients who are not in the chronic phase of chronic myeloid leukemia. The chronic phase is an early stage of the disease where symptoms are usually mild.
  • Patients who are not within the specified age range for the study.
  • Patients who belong to a group that is considered vulnerable, meaning they might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Universitaetsklinikum Heidelberg AöR Heidelberg Germany

Other Sites

Site Name City Country Status
Universitaetsklinikum Aachen AöR Aachen Germany
Kasaoynp Bebueobg Gxsp Bayreuth Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Germany Germany
Not recruiting
20.12.2019

Trial locations

Investigated drugs:

Ponatinib is a medication used in this clinical trial to treat patients with chronic myeloid leukemia. The trial aims to assess patients who have achieved a specific level of remission, known as MR4, after two years of treatment with this medication. Ponatinib works by blocking certain proteins that promote the growth of cancer cells, helping to control the disease.

Chronic Myeloid Leukemia in Chronic Phase – This is a type of cancer that affects the blood and bone marrow. It is characterized by the overproduction of white blood cells. In the chronic phase, the disease progresses slowly and may not cause noticeable symptoms initially. Over time, the number of abnormal cells increases, which can lead to fatigue, weight loss, and an enlarged spleen. The chronic phase can last for several years before potentially advancing to more aggressive phases. Monitoring and regular check-ups are essential to track the progression of the disease.

Trial ID:
2024-518971-76-00
Protocol code:
Pontrack01
Trial Phase:
Therapeutic exploratory (Phase II)

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