Study on Ponatinib for Treating Recurrent or Refractory Leukemias and Solid Tumors in Children

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What is this study about?

This clinical trial is focused on studying the effects of a medication called Ponatinib on certain types of cancers in children. The cancers being studied include recurrent or refractory leukemias, which are blood cancers that have returned or do not respond to treatment, and solid tumors, which are abnormal masses of tissue. The purpose of the study is to evaluate the safety and effectiveness of Ponatinib in treating these conditions. Ponatinib is a type of medication known as a kinase inhibitor, which works by blocking certain proteins that help cancer cells grow.

The study is divided into two phases. In the first phase, the goal is to find the best dose of Ponatinib for children with advanced blood cancers or solid tumors. In the second phase, the study will look at how well Ponatinib works in children with a specific type of leukemia called Chronic Myeloid Leukemia (CML) and other selected cancers. Participants will take Ponatinib orally, which means they will swallow it in the form of a tablet or capsule. The study will monitor the participants’ health and any side effects they may experience while taking the medication.

Throughout the study, researchers will assess the participants’ response to the treatment by using various medical tests and imaging techniques like MRI or CT scans. The study aims to provide valuable information on the potential benefits and risks of using Ponatinib in treating these challenging cancers in children. The study is expected to continue until 2025, allowing researchers to gather comprehensive data on the long-term effects of the treatment.

1 joining the study

Upon joining the study, the participant will be informed about the trial’s objectives and procedures. Consent will be obtained from a parent or legal guardian, and assent from the participant if appropriate.

2 initial assessment

An initial assessment will be conducted to confirm the diagnosis of recurrent or refractory leukemias, lymphomas, or solid tumors. This may involve tests such as bone marrow aspirate or imaging studies.

3 medication administration

The participant will receive ponatinib, administered orally. The dosage will be determined based on the phase of the study and the participant’s specific condition.

In Phase 1, the dosage will be adjusted to find the maximum tolerated dose. In Phase 2, the dosage will be based on the results from Phase 1.

4 treatment monitoring

The participant’s response to the treatment will be monitored regularly through clinical evaluations, blood tests, and imaging studies.

The frequency and severity of any side effects will be recorded, and adjustments to the treatment may be made as necessary.

5 evaluation of treatment efficacy

The effectiveness of the treatment will be assessed at specific intervals. This may include measuring the reduction in tumor size or changes in blood cell counts.

For participants with chronic myeloid leukemia (CML), the response will be evaluated using specific criteria such as cytogenetic or molecular response.

6 end of treatment

The treatment will continue until the participant completes the study, experiences unacceptable side effects, or the disease progresses.

After the treatment ends, follow-up assessments will be conducted to monitor the participant’s health and any long-term effects of the treatment.

Who Can Join the Study?

Participants must have a confirmed diagnosis of certain types of cancer, such as leukemias, lymphomas, or solid tumors. This means that tests have shown they have one of these cancers.
Participants must have tried standard treatments for their cancer, but these treatments did not work or are not suitable for them.
Participants must be between 1 and 18 years old at the time of joining the study.
Participants must have a certain level of physical ability, measured by a test called the Karnofsky performance status for those 16 and older, or the Lansky Play Scale for those under 16. This means they need to be able to do some daily activities.
Participants must have recovered from any side effects of previous treatments to a certain level, except for hair loss.
Participants must agree to avoid pregnancy or fathering a child during the study and for a period after the study ends. This involves using effective birth control methods.
Participants must have a parent or legal guardian who can understand and sign a consent form, agreeing to the study’s requirements and procedures.

Who Cannot Join the Study?

Patients who have a different type of cancer than the ones being studied, such as recurrent or refractory leukemias, lymphomas, and solid tumors. These are cancers that have come back after treatment or do not respond to treatment.
Patients who are not within the age range specified for the study. The study is for children, so adults cannot participate.
Patients who are not able to take the study medication by mouth, as the treatment is given orally.
Patients who have other serious health conditions that might interfere with the study treatment or make it unsafe for them to participate.
Patients who are pregnant or breastfeeding, as the effects of the study treatment on unborn babies or infants are not known.
Patients who are participating in another clinical trial at the same time, as this could affect the results of the study.
Patients who have had a recent major surgery or are recovering from a major surgery, as this could affect their ability to participate safely.
Patients who have an active infection that requires treatment, as this could interfere with the study treatment.
Patients who have a known allergy to the study medication or any of its ingredients.
Patients who have not recovered from the side effects of previous cancer treatments.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Hospital Universitario Y Politecnico La Fe	Valencia	Spain

Other Sites

Site Name	City	Country
Centre Hospitalier Universitaire De Poitiers	Poitiers	France
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Universitair Ziekenhuis Gent	Gent	Belgium
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Fondazione IRCCS San Gerardo Dei Tintori	Monza	Italy
Azienda Ospedaliera Santobono Pausilipon	Naples	Italy
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Karolinska University Hospital	Solna	Sweden
Prinses Maxima Centrum voor Kinderoncologie B.V.	Utrecht	The Netherlands
Centre Hospitalier Universitaire De Rennes	Rennes	France
Hopital Beaujon	Clichy	France
Universita Degli Studi Di Brescia	Brescia	Italy
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Adhhffz Ocxensbqcxp Uhyhfkafuoilv Cnkfigkudhcp Dblnt Sbrhcm E Dqjfq Sjbmfpb Db Tdkjkw	Turin	Italy
Apdzyyt Urrzs Spyuxnxst Lxcpqp Do Bkkiuxy	Bologna	Italy
Hrhexzjp Vgih dajrqrmi	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	02.12.2019
France	Recruiting	02.12.2019
Italy	Recruiting	02.12.2019
Spain	Recruiting	02.12.2019
Sweden	Recruiting	02.12.2019
The Netherlands	Recruiting	02.12.2019

Trial locations

Investigated drugs:

Ponatinib

Ponatinib is a medication being studied for its safety and effectiveness in treating certain types of leukemia and solid tumors in children. It is taken by mouth once a day. The study aims to find the best dose of Ponatinib for children with these conditions and to see how well it works for those who have not responded to other treatments or have specific genetic mutations.

Recurrent or Refractory Leukemias – These are types of blood cancers that return after treatment or do not respond to standard therapies. Leukemias affect the blood and bone marrow, leading to an overproduction of abnormal white blood cells. As the disease progresses, it can interfere with the body’s ability to produce healthy blood cells. Symptoms may include fatigue, frequent infections, and easy bruising or bleeding. The progression can vary depending on the specific type of leukemia and individual patient factors.

Lymphomas – Lymphomas are cancers of the lymphatic system, which is part of the body’s immune system. They typically begin in lymph nodes or other lymphatic tissues and can spread to other parts of the body. As lymphomas progress, they may cause swollen lymph nodes, fever, night sweats, and weight loss. The disease can be classified into Hodgkin and non-Hodgkin types, each with different progression patterns. The rate of progression can vary widely, with some lymphomas growing slowly and others more rapidly.

Solid Tumors – Solid tumors are abnormal masses of tissue that can occur in various organs or tissues, excluding blood-related cancers. They can be benign (non-cancerous) or malignant (cancerous), with malignant tumors having the potential to invade nearby tissues and spread to other parts of the body. As solid tumors grow, they may cause symptoms related to the affected organ, such as pain, swelling, or dysfunction. The progression of solid tumors depends on the type, location, and aggressiveness of the cancer cells.

Trial ID:

2023-509699-41-00

Protocol code:

INCB 84344-102

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on Ponatinib for Treating Recurrent or Refractory Leukemias and Solid Tumors in Children

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