Ongoing Clinical Trials for NTRK Gene Fusion Overexpression
Currently, there are 2 ongoing clinical trials investigating treatments for cancers with NTRK gene fusion. These studies are testing larotrectinib, an oral medication that targets the abnormal proteins produced by NTRK gene fusions, in both adult and pediatric patients with solid tumors. The trials are being conducted across multiple European countries including France, Germany, Spain, Sweden, Denmark, and others.
Clinical trial locations
- Czechia
- Denmark
- France
- Germany
- Ireland
- Italy
- Netherlands
- Portugal
- Spain
- Sweden
Study on How Larotrectinib Works in Adults with Solid Tumors with NTRK Gene Fusion
This trial is investigating the effectiveness of larotrectinib in treating adult patients with various types of solid cancers that have a specific genetic change known as NTRK fusion. Solid cancers are tumors that form in solid organs or tissues, such as the breast, lung, or colon. The NTRK fusion involves the joining of an NTRK gene with another gene, which can lead to uncontrolled cancer growth.
Who can participate:
- Adults aged 18 years or older
- Patients with locally advanced or metastatic cancer confirmed to have an NTRK1, NTRK2, or NTRK3 gene fusion through laboratory testing
- Patients who have received previous standard treatments for their cancer type or have no other satisfactory treatment options available
- At least one measurable tumor that can be assessed using medical imaging
- Good enough organ function, particularly liver and kidneys, as shown by blood tests
- A Performance Status score of 3 or less, meaning patients can still perform most daily activities with some limitations
- Willingness to provide tumor tissue samples before starting treatment
- Ability to use two effective methods of birth control during the study for patients who can have children
Who cannot participate:
- Patients without solid tumors containing NTRK fusion
- Those who fall outside the specified age range
- Patients unable to follow study procedures or with conditions that might interfere with study results
- Members of vulnerable populations who cannot provide proper consent
Study focus: The main goal is to learn how well larotrectinib works in treating these cancers by monitoring tumor response over time. Participants will receive larotrectinib as an oral solution or capsule. The study will look for signs of tumor shrinkage or stabilization through regular check-ups and imaging tests such as MRI or CT scans. Researchers will evaluate the overall response rate, duration of response, and progression-free survival, which means the length of time the cancer does not worsen during and after treatment. Safety will be closely monitored throughout the study, which is expected to continue until October 2025.
Investigational drug: Larotrectinib is an oral medication that targets specific proteins called TRK proteins in cancer cells. These proteins become abnormal due to changes in NTRK genes. By blocking the activity of these TRK proteins, larotrectinib may help stop cancer cell growth or even shrink tumors.
Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion
This clinical trial is studying the effects of larotrectinib in children with certain types of tumors known as solid tumors that have an NTRK fusion. The study is divided into two phases: the first focuses on determining the safety of the medication and identifying any side effects, while the second phase looks at how well tumors respond to treatment.
Who can participate:
- Children from birth to 21 years old with solid tumors or primary central nervous system tumors that have come back, gotten worse, or did not respond to other treatments
- Patients must have documented NTRK gene fusion confirmed by laboratory testing
- Infants with worsening cancer and NTRK fusion who have no other standard treatment options
- Patients with infantile fibrosarcoma who would need disfiguring surgery or limb amputation to remove the tumor completely
- Patients with NTRK-fusion positive benign tumors are also eligible
- Performance score of at least 50 on the Karnofsky scale (for ages 16 and older) or Lansky scale (for younger than 16), which measures ability to perform daily activities
- Adequate blood cell counts, liver function, and kidney function
Who cannot participate:
- Patients without solid tumors containing NTRK fusion
- Those outside the specified age range
- Patients unable to take medication by mouth
- Those with other medical conditions that might interfere with the study
- Pregnant or breastfeeding patients
- Patients currently participating in another clinical trial
- Those who have had certain recent treatments that could affect how the study medication works
Study focus: The primary purpose is to test the safety and effectiveness of larotrectinib in children with these specific tumors. In Phase 1, researchers determine the safety of the medication and the maximum tolerated dose while observing any dose-limiting side effects. In Phase 2, the focus shifts to determining the overall response rate, with an independent radiology review committee evaluating whether tumors shrink completely or partially. Throughout the trial, patients are monitored for any side effects, and regular assessments measure the concentration of larotrectinib in the blood and evaluate overall health and quality of life. The trial is expected to continue until September 2026.
Investigational drug: Larotrectinib is an oral medication taken either as a capsule or liquid solution. It works by inhibiting TRK proteins, which are involved in the growth of cancer cells with NTRK fusions. The study aims to determine how well this medication can help shrink tumors in children whose cancers have these specific genetic changes.
Summary
Both ongoing clinical trials are focused on the same investigational drug, larotrectinib, but target different patient populations. One study focuses on adult patients aged 18 and older, while the other is designed specifically for pediatric patients from birth to 21 years of age. The trials share a common approach of targeting cancers with NTRK gene fusions, which is a specific genetic change that drives tumor growth.
The trials are being conducted across a wide range of European countries, with notable concentration in France, Germany, Spain, Sweden, and Denmark, where both studies are active. The pediatric trial has a broader geographic reach, including additional locations in Czechia, Italy, Netherlands, and Ireland.
Both studies use the same mechanism of action through larotrectinib, which blocks TRK proteins produced by NTRK gene fusions. The adult study is expected to conclude in October 2025, while the pediatric trial will continue until September 2026. These trials represent important research efforts to develop targeted therapies for rare cancers characterized by NTRK fusions, offering potential treatment options for patients who have limited alternatives available.
