A Study of Repotrectinib in Pediatric and Young Adult Subjects Harboring ALK, ROS1, OR NTRK1-3 Alterations
This research study is testing a new medicine called repotrectinib (also known as TPX-0005) for children and young adults up to 25 years old. It's for those with advanced or spreading cancers like solid tumours (including those in the brain) or certain types of lymphoma. The study aims to see if repotrectinib is safe and can help treat these cancers, especially if they have particular genetic changes called ALK, ROS1, or NTRK. The study has two main parts: Phase 1 focuses on finding the safest and most effective dose for younger children. Phase 2 then looks at how well this dose works to shrink tumours in different groups of patients with these specific genetic changes, particularly ROS1 and NTRK.
At a glance
What is this study about?
This study is looking into a new drug called repotrectinib (you might also hear it called TPX-0005) for children and young adults up to 25 years old. These patients have certain types of cancer that are advanced or have spread, such as solid tumours (which can include those in the brain) or a type of lymphoma called anaplastic large cell lymphoma. Doctors are particularly interested in patients whose cancers have specific genetic changes, known as ALK, ROS1, or NTRK.
The main goals of this research are to figure out if repotrectinib is safe for young people and, if so, what the best dose is to give. Once that's established, the study then aims to see how well the drug works at shrinking tumours that have these specific genetic changes. This is important because these genetic changes can sometimes make cancers harder to treat with standard medicines.
The study is divided into two parts: In Phase 1, the researchers will mainly focus on very young children (under 12) to find the right and safest dose of repotrectinib. Then, in Phase 2, they will give this dose to more children and young adults (up to 25) to see how effective it is in shrinking their tumours. They will look at different groups of patients depending on the specific genetic change in their cancer and if they’ve had similar treatments before.
Key takeaways
- This study is for children and young adults (up to 25) with specific advanced cancers.
- It tests a new drug called repotrectinib for cancers with ALK, ROS1, or NTRK genetic changes.
- The study aims to find a safe dose and see how well the drug shrinks tumours.
- Patients will take repotrectinib as a tablet by mouth.
- Regular hospital visits, blood tests, and scans will be part of the study.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
To join this study, people generally need to be under 25 years old. Importantly, their cancer must have certain genetic changes – specifically, a change in the ROS1 gene or one of the NTRK1, NTRK2, or NTRK3 genes. This would have been found through tests on their cancer tissue. For the first part of the study (Phase 1), patients need to be under 12, while for the second part (Phase 2), patients are typically aged 12 to 25.
Also, a patient’s general health needs to be good enough to take part. This means that if they’ve had previous cancer treatments, any side effects from those treatments (apart from hair loss) should have mostly cleared up. They also need to have what doctors call 'measurable disease,' which means their tumour can be seen and measured to check if the treatment is working. If they have cancer in the brain, it needs to be stable and they shouldn't be needing increasing amounts of certain medicines to control it.
There are also reasons why someone might not be able to join. For example, if their neuroblastoma (a type of cancer) is only found in the bone marrow and can only be checked by bone marrow tests, they might not be eligible.
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.
- Is the patient under 25 years old?
- Does their cancer have a ROS1 or NTRK genetic change?
- Are any severe side effects from previous treatments mostly gone (except hair loss)?
- Did doctors find that the patient has 'measurable disease' (tumour can be seen and measured)?
- Is their overall health, as rated by a doctor, good enough for a study?
What does participation involve?
If you decide to take part in this study, you will be given the study drug, repotrectinib, as a tablet that you take by mouth. The study involves regular visits to the hospital or clinic so doctors can carefully check your health, how you are feeling, and if the medicine is working. These visits will include various tests, such as blood tests, scans (like CT or MRI scans) to measure the tumour, and checks of your overall well-being.
For children under 12, the first part of the study will help doctors find the safest and most effective dose. Adults and young people aged 12 to 25 might go straight into the second part, where the main goal is to see how well the drug works. The exact number of visits and tests will depend on which part of the study you are in and how you respond to the treatment. There will also be follow-up appointments after you finish taking the drug to make sure you are still doing well. The total length of time you'll be involved in the study can vary, but doctors will explain this fully if you're thinking of joining.
Potential risks and benefits
Locations (68)
- Children's Hospital Los AngelesVerified postcodeLos Angeles, United States· Recruiting
- University of California at Los AngelesVerified postcodeLos Angeles, United States· Recruiting
- Children's Hospital Colorado - Anschutz Medical CampusVerified postcodeAurora, United States· Recruiting
- Local Institution - 2105Verified postcodeOrlando, United States· Completed
- Local Institution - 2120Verified postcodeOrlando, United States· Completed
- Children's Healthcare of Atlanta - Egleston HospitalVerified postcodeAtlanta, United States· Recruiting
- Maine Medical CenterVerified postcodeScarborough, United States· Recruiting
- Dana Farber Cancer Institute.Verified postcodeBoston, United States· Recruiting
- Washington University School of Medicine in St. LouisVerified postcodeSt Louis, United States· Recruiting
- Local Institution - 2110Verified postcodeNew Brunswick, United States· Completed
- Local Institution - 2102Verified postcodeNew York, United States· Completed
- Levine Children's Hospital- Pediatric Neuro-OncologyVerified postcodeCharlotte, United States· Recruiting
Common questions
What is repotrectinib?
Repotrectinib is a new experimental drug being tested to treat certain advanced cancers in children and young adults that have specific genetic changes.
What are ALK, ROS1, and NTRK alterations?
These are specific genetic changes or 'alterations' that can happen in cancer cells. Doctors test for them because they can help guide which treatments might work best.
What does 'Phase 1' and 'Phase 2' mean in a study?
Phase 1 is usually the first time a drug is tested in people, focusing on safety and finding the right dose. Phase 2 then looks to see how well that dose works against the cancer.
Can I still join if I've had chemotherapy before?
Yes, having had chemotherapy or other cancer treatments before is generally allowed, as long as any side effects from those treatments have mostly cleared up.
Will I have to stay in the hospital?
You will need to attend regular clinic visits for check-ups and tests, but it's not expected that you will need to stay in hospital for the duration of the study, unless your medical condition requires it.
How to find out more
BMS Study Connect Contact Center www.BMSStudyConnect.com
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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