Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors
This research study is testing a medication called ponatinib in young people aged 1 to 17 who have certain blood cancers (like leukemia or lymphoma) or specific solid tumours. We know that these conditions can be very challenging, especially when standard treatments haven't been successful or aren't available. The main goals are to understand how safe ponatinib is for children, how their bodies handle the medicine, and if it can help to treat their cancer. The study is split into two parts: an early phase to check safety and dosage, and a later phase to see how effective it is in specific groups of patients. We're looking for children whose cancer has returned, become difficult to treat, or where other options are limited.
At a glance
What is this study about?
This study is investigating a new treatment called ponatinib for children and teenagers between 1 and 17 years old who are dealing with certain types of cancer. These include blood cancers like different kinds of leukaemia (such as Acute Myeloid Leukaemia, Acute Lymphoblastic Leukaemia, and Chronic Myeloid Leukaemia) and lymphoma, as well as some solid tumours found elsewhere in the body. We understand that hearing about such conditions can be worrying, and this study aims to explore new possibilities when previous treatments haven't worked as hoped, or when there are no standard treatments available.
The main purpose of this research is two-fold. Firstly, we want to make sure the medicine, ponatinib, is safe for young patients and to understand any side effects it might have. Secondly, we want to see how well it works in fighting the cancer. The study is structured in two parts (Phase 1 and Phase 2), which is common in medical research. Phase 1 focuses on finding the right dose and checking for safety, while Phase 2 looks more closely at how effective the treatment is for particular types of cancer.
By taking part, we hope to gather important information that could lead to new treatment options for young people facing challenging cancer diagnoses. Your well-being and safety are our top priority throughout this research.
Key takeaways
- This study is for young people (1-17 years) with specific cancers.
- It's testing a new medicine called ponatinib.
- It focuses on cancers that are hard to treat or have come back.
- The main goals are to check for safety and how well the medicine works.
- Participation means contributing to learning about new cancer treatments for children.
Who may be eligible?
This study is for children and teenagers aged 1 to 17. To be considered, you would need to have certain types of cancer, including various forms of leukaemia, lymphoma, or a solid tumour. Importantly, these cancers must either not have responded well to previous treatments, have come back, or have no standard treatment options currently available or suitable.
For some types of leukaemia, especially Chronic Myeloid Leukaemia, you might be eligible if your previous treatments haven't worked or you couldn't tolerate them. We'd also need to see if your cancer has specific genetic changes to be sure ponatinib might be helpful. For those with solid tumours or lymphoma, the doctors need to be able to measure the size of your tumour using scans like CT or MRI.
If you've had previous treatments, we'd look to see that your cancer has progressed despite these, or that there are no other effective standard treatments for your situation.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you between 1 and 17 years old?
- Do you have a diagnosis of leukaemia, lymphoma, or a solid tumour?
- Has your cancer been difficult to treat with standard methods, come back, or are there no standard treatments available?
- For some cancers, have you specifically not responded to or tolerated previous targeted treatments?
- For some cancers, do genetic tests show specific changes in your tumour that ponatinib might target?
What does participation involve?
The detailed description of what participation involves (like visits, assessments, medication, follow-up, and total duration) is not provided in the 'DETAILED DESCRIPTION' section of the raw data. To explain this clearly, further information from the full study protocol would be needed. However, generally, taking part in a clinical trial might involve regular hospital visits for check-ups, blood tests, scans, and receiving the study medication. Doctors will closely monitor your health and how you respond to the treatment. The duration can vary widely depending on the study and how you respond.
Potential risks and benefits
Locations (23)
- Ghent University HospitalVerified postcodeGhent, Belgium· Recruiting
- Hopital Robert DebreVerified postcodeParis, France· Recruiting
- Armand Trousseau HospitalVerified postcodeParis, France· Recruiting
- Centre Hospitalier Universitaire de PoitiersVerified postcodePoitiers, France· Recruiting
- Chu de Rennes - Hospital SudVerified postcodeRennes, France· Recruiting
- Aou Policlinico S. Orsola-MalpighiVerified postcodeBologna, Italy· Recruiting
- Asst Degli Spedali Civili Di BresciaVerified postcodeBrescia, Italy· Recruiting
- Ospedale Pediatrico G. GasliniVerified postcodeGenova, Italy· Recruiting
- Comitato Etico Fondazione Irccs Istituto Nazionale Dei Tumori MilanoVerified postcodeMilan, Italy· Recruiting
- University of Milano BicoccaVerified postcodeMonza, Italy· Recruiting
- Aorn Santobono PausiliponVerified postcodeNaples, Italy· Not yet recruiting
- Comitato Di Bioetica Della Fondazione Irccs Policlinico San MatteoVerified postcodePavia, Italy· Recruiting
Common questions
What is ponatinib?
Ponatinib is a medicine being tested in this study to see if it can help treat certain cancers, especially when other treatments haven't worked.
What types of cancer is this study looking at?
This study is for children with certain types of leukaemia (blood cancer), lymphoma, and particular solid tumours.
Who can take part in this study?
Children and teenagers aged 1 to 17 whose cancer has come back, is difficult to treat, or where standard treatments aren't suitable, might be eligible.
Will taking part affect my regular medical care?
No, you can withdraw from the study at any time for any reason, and it will not affect the quality of your usual medical care.
What are the main goals of the study?
The study aims to understand how safe ponatinib is for children and how well it works against their cancer.
How to find out more
Incyte Corporation Call Center (ex-US)
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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