Active not recruitingPHASE1, PHASE2INTERVENTIONAL

A Study to Test the Safety and Efficacy of the Drug Larotrectinib for the Treatment of Tumors With NTRK-fusion in Children

This study, called "A Study to Test the Safety and Efficacy of the Drug Larotrectinib for the Treatment of Tumors With NTRK-fusion in Children," is looking into a new cancer medication named larotrectinib (also known as Vitrakvi) for children. The research focuses on tumours that have a particular genetic change called an NTRK fusion. Larotrectinib works by blocking the signals from these changed NTRK genes, which can help stop cancer cells from growing. The first part of the study (Phase 1) helped doctors find a safe dose for children and understand how their bodies handle the drug. The second part (Phase 2) is now looking at how well the treatment works for different types of cancer and how long any improvements last.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Bayer

Enrolment target

154

Start

16 Dec 2015

Estimated completion

30 Sep 2026

Solid Tumors Harboring NTRK Fusion

What is this study about?

This research is a clinical trial exploring a new medication called larotrectinib, also known as Vitrakvi. It's specifically for children and young people up to 21 years old who have certain types of cancer. The key thing about the cancers this study is interested in is that they must have a specific change in their genes, called an 'NTRK fusion'. This gene change can cause cancer cells to grow out of control.

Larotrectinib is designed to block the signals from these changed NTRK genes. By interfering with these signals, the drug aims to stop the cancer cells from growing and spreading. The study is divided into two main parts. The first part (Phase 1) was about finding the safest dose of larotrectinib for children, understanding how their bodies process the drug, and seeing if their cancers responded to treatment. This phase has now finished enrolling new patients.

The second part of the study (Phase 2), which is currently ongoing, aims to find out how effective larotrectinib is. Researchers want to understand how many patients with different types of NTRK fusion cancer respond to the treatment, and for how long these responses last. The overall goal is to see if larotrectinib can be a helpful new treatment option for children with these specific types of cancer.

Key takeaways

This study is testing a new cancer medicine called larotrectinib for children.
It's for specific tumours that have an 'NTRK gene fusion'.
The drug aims to block signals from these changed genes to stop cancer growth.
The study assesses how safe the medicine is and how well it treats the cancer.
It's for children up to 21 years old with advanced cancer that lacks standard treatment options.

Who may be eligible?

The study is for children and young people, from birth up to 21 years old. To join, participants need to have a solid tumour (a lump of cancer cells) or a brain tumour that has either come back, got worse after other treatments, or didn't respond to treatments that are usually available. Importantly, there must not be any other standard treatment options that could cure their cancer.

A key requirement is that the cancer must have a specific genetic change called an 'NTRK gene fusion'. This change is carefully checked by doctors. There are a few exceptions: for certain rare cancers like infantile fibrosarcoma, congenital mesoblastic nephroma, or secretory breast cancer, the genetic test might look for slightly different signs if an NTRK fusion isn't found.

In some cases, if a patient has infantile fibrosarcoma (a type of soft tissue cancer in infants) and doctors believe that surgery would cause severe disfigurement or require limb removal, they might also be able to join the study.

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 21 years old or younger?
Does the cancer have an 'NTRK gene fusion' confirmed by a test?
Has the cancer either come back, got worse, or not responded to other treatments?
Are there no other standard or available treatment options that could cure the cancer?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive the medication larotrectinib. The study does not specify exactly how many visits or tests would be involved, but typically in clinical trials, there are regular check-ups with your doctor, including scans and blood tests, to see how you are responding to the treatment and to monitor for any side effects. These assessments would help the doctors understand how the drug is working and adjust your care if needed. The total duration of participation isn't specified here, but clinical trials usually involve ongoing treatment and follow-up as long as the treatment is beneficial and safe. You will be given more detailed information about the schedule if you consider joining.

Potential risks and benefits

Participating in this study might offer the benefit of receiving a new, targeted treatment for your cancer, especially if other standard treatments haven't worked or aren't suitable. This drug is specifically designed to target the NTRK gene changes in your tumour. However, like all medications, larotrectinib can have side effects, which the study doctors will monitor closely. You will be told about all known possible risks before you decide to join. It's very important to remember that joining a clinical trial is always voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (45)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Children's Hospital Los Angeles - Hematology/Oncology
Verified postcode
Los Angeles, United States
UCLA Jonsson Comprehensive Cancer Center
Verified postcode
Los Angeles, United States
Lucille Packard Children's Hospital Stanford - Pediatric Nephrology
Verified postcode
Palo Alto, United States
Nemours Children's Hospital - Florida - Hematology / Oncology
Verified postcode
Orlando, United States
Dana-Farber Cancer Institute
Verified postcode
Boston, United States
Memorial Sloan Kettering Cancer Center New York - Main Campus
Verified postcode
New York, United States
Cincinnati Children's Hospital Medical Center | Division of Nephrology and Hypertension
Verified postcode
Cincinnati, United States
Children's Hospital of Philadelphia - Hematology/Oncology
Verified postcode
Philadelphia, United States
St. Jude Children's Research Hospital
Verified postcode
Memphis, United States
University of Texas Southwestern Medical Center
Verified postcode
Dallas, United States
Seattle Children's Hosptial - Oncology
Verified postcode
Seattle, United States
Sydney Children's Hospital
Verified postcode
Sydney, Australia

Common questions

What is larotrectinib?

Larotrectinib is a new cancer medicine that targets specific gene changes (NTRK fusions) found in some tumours.

What is an NTRK gene fusion?

It's a specific change in a gene within cancer cells that can cause them to grow unusually. This study is for cancers with this particular change.

Who can take part in this study?

Children and young people up to 21 years old who have certain types of advanced cancer with an NTRK gene fusion and no other standard treatment options.

What happens if I join the study?

You would receive the study medicine, larotrectinib, and have regular check-ups, scans, and blood tests to monitor your health and how the treatment is working.

Can I leave the study at any time?

Yes, taking part is completely voluntary. You can decide to leave the study at any point without it affecting your future medical care.