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Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion

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What is this study about?

This clinical trial is focused on studying the effects of a medication called larotrectinib in children with certain types of tumors. These tumors are known as solid tumors that have a specific genetic change called NTRK fusion. The study aims to test the safety and effectiveness of larotrectinib in treating these tumors. Larotrectinib is taken by mouth, either as a capsule or an oral solution.

The purpose of the study is to understand how safe larotrectinib is for children and how well it works in shrinking or controlling the tumors. The study is divided into two phases. In the first phase, the focus is on determining the safety of the medication and identifying any side effects. In the second phase, the study looks at how well the tumors respond to the treatment, which means checking if the tumors get smaller or stop growing.

Participants in the study will receive larotrectinib and will be monitored by healthcare professionals to observe any changes in their condition. The study will help gather important information about the potential benefits and risks of using larotrectinib for treating tumors with NTRK fusion in children. This research is important for developing new treatments for these types of tumors and improving the care of children affected by them.

1 Joining the study

Upon joining the study, the patient will be informed about the trial’s purpose, which is to test the safety and effectiveness of the drug larotrectinib for treating tumors with NTRK-fusion in children.

The patient will be assessed to ensure they meet the eligibility criteria, which include having a solid tumor with NTRK fusion and being between birth and 21 years of age.

2 Phase 1: Safety assessment

The primary goal of Phase 1 is to determine the safety of oral larotrectinib in pediatric patients with advanced solid or primary central nervous system tumors.

The patient will receive larotrectinib in the form of a hard capsule or oral solution, taken by mouth.

The dosage will be adjusted to find the maximum tolerated dose and to observe any dose-limiting side effects.

3 Phase 2: Effectiveness assessment

In Phase 2, the focus is on determining the overall response rate to larotrectinib treatment.

The patient’s response will be evaluated by an independent radiology review committee, looking for complete or partial tumor response.

The patient will continue to take larotrectinib orally, with the dosage determined from Phase 1.

4 Monitoring and follow-up

Throughout the trial, the patient will be monitored for any treatment-emergent adverse events.

Regular assessments will be conducted to measure the concentration of larotrectinib in the blood and to evaluate the patient’s overall health and quality of life.

The trial is expected to continue until September 2026, with ongoing monitoring and data collection.

Who Can Join the Study?

  • Patients must have a type of cancer called a solid tumor with a specific genetic change known as an NTRK fusion.
  • For Phase 1 (which is closed to new participants): Patients from birth to 21 years old with a solid tumor or a primary central nervous system (CNS) tumor that has come back, gotten worse, or did not respond to other treatments, and no other standard treatment options are available.
  • Infants with a cancer diagnosis and an NTRK fusion that has worsened or not responded to available treatments, with no other standard treatment options available.
  • Patients with a type of cancer called infantile fibrosarcoma (IFS) who would need surgery that could cause disfigurement or limb loss to remove the tumor completely.
  • For Phase 2: Infants from birth and older with locally advanced or metastatic IFS, or patients with IFS who would need surgery that could cause disfigurement or limb loss, with a specific genetic change called ETV6 rearrangement or NTRK3 rearrangement.
  • Patients from birth to 21 years old with a solid tumor or primary CNS tumor that has come back, gotten worse, or did not respond to other treatments, with a documented NTRK gene fusion.
  • Patients with NTRK-fusion positive benign tumors are also eligible.
  • Patients older than 21 years with a tumor type usually found in children and an NTRK fusion may be considered for the study after discussion with the study team.
  • Patients with primary CNS tumors or cancer that has spread to the brain.
  • Patients must have a performance score of at least 50 on the Karnofsky scale (for those 16 years and older) or the Lansky scale (for those younger than 16 years). These scales measure how well a patient can perform daily activities.
  • Patients must have adequate blood cell counts, which is referred to as adequate hematologic function.
  • Patients must have proper liver and kidney function, known as adequate hepatic and renal function.

Who Cannot Join the Study?

  • Patients who do not have solid tumors with NTRK fusion cannot participate. Solid tumors are abnormal masses of tissue that usually do not contain liquid areas. NTRK fusion is a specific change in the genes that can cause cancer.
  • Patients who are not within the specified age range for the study cannot participate. The study is for certain age groups, so if a patient is too young or too old, they may not be eligible.
  • Patients who are not able to take the study medication orally (by mouth) cannot participate. The medication in this study is taken by mouth, so patients need to be able to swallow it.
  • Patients who have other medical conditions that might interfere with the study cannot participate. This means if a patient has another illness that could affect the study results, they may not be eligible.
  • Patients who are pregnant or breastfeeding cannot participate. This is to ensure the safety of both the mother and the baby.
  • Patients who are participating in another clinical trial cannot participate. Being in more than one study at a time can affect the results of both studies.
  • Patients who have had certain treatments recently may not be able to participate. Some treatments can affect how the study medication works, so patients may need to wait before joining the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Institut Gustave Roussy Villejuif France
Universitaetsklinikum Heidelberg AöR Heidelberg Germany

Other Sites

Site Name City Country Status
Klinikum Der Landeshauptstadt Stuttgart gKAöR Stuttgart Germany
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Karolinska University Hospital Solna Sweden
Prinses Maxima Centrum voor Kinderoncologie B.V. Utrecht The Netherlands
Rigshospitalet Copenhagen Denmark
Fondazione I.R.C.C.S. Istituto Neurologico Besta Milan Italy
Children’s Health Ireland Dublin Ireland
Fptdhgch nshnccssp Mxfyb a Hpswdqy Prague Czechia
Hyzzuopk Vywz dczaxjvt Barcelona Spain
Idwumrxy Couxb Paris France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Czechia Czechia
Not recruiting
15.12.2015
Denmark Denmark
Not recruiting
15.12.2015
France France
Not recruiting
15.12.2015
Germany Germany
Not recruiting
15.12.2015
Ireland Ireland
Not recruiting
15.12.2015
Italy Italy
Not recruiting
15.12.2015
Spain Spain
Not recruiting
15.12.2015
Sweden Sweden
Not recruiting
15.12.2015
The Netherlands The Netherlands
Not recruiting
15.12.2015

Trial locations

Investigated drugs:

Larotrectinib is an oral medication being studied for its safety and effectiveness in treating pediatric patients with advanced solid tumors or primary central nervous system tumors. It works by inhibiting TRK proteins, which are involved in the growth of certain types of cancer cells. The study aims to determine how well this medication can help shrink tumors in children whose cancers have specific genetic changes known as NTRK fusions.

Investigated diseases:

Solid Tumors Harboring NTRK Fusion – These are a group of cancers that occur when a specific genetic change, known as an NTRK fusion, is present in the tumor cells. This fusion involves the joining of an NTRK gene with another gene, leading to uncontrolled cell growth. These tumors can develop in various parts of the body, including the central nervous system. The presence of NTRK fusions can lead to the formation of solid masses that may grow and spread to other areas. The progression of these tumors depends on the location and size of the tumor, as well as the specific characteristics of the NTRK fusion. They are considered rare diseases due to their uncommon genetic profile.

Trial ID:
2022-502668-20-00
Protocol code:
20290
NCT ID:
NCT02637687
Trial Phase:
Human Pharmacology (Phase I) – Other

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