NTRK Gene Fusion - Positive Advanced or Recurrent Solid Tumors, a Rare Cancer Caused by Specific Changes in the Genes

Special Drug Use Investigation for Larotrectinib

Researchers want to learn more about the use of larotrectinib as a real-world treatment for tropomyosin receptor kinase fusion cancer, also called TRK fusion cancer.

In people with TRK fusion cancer, a gene called neurotrophic TRK, (NTRK) joins or "fuses" with another gene. This creates a protein known as a fusion protein, which can cause cancer cells to grow. The study treatment, larotrectinib, is already available for doctors to prescribe to patients with TRK fusion cancer. Larotrectinib works by blocking TRK genes in cancer cells which helps stop the cancer from growing.

In this study, the researchers want to learn more about the safety and effectiveness of larotrectinib in adults and children with advanced or recurrent TRK fusion cancer. This means that their cancer has spread from where it started to other areas of the body, or the cancer has come back after a period of time. To answer this question, the researchers will collect information from patients who are taking larotrectinib as prescribed by their doctors. The researchers will learn what adverse events the patients are having. An adverse event is any medical problem that a patient has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments.

The study will include patients of all ages with TRK fusion cancer.

In this study, there will be no required tests or visits to a study site. Instead, the researchers will collect information from:

• the patients' medical records
• interviews with the patients or their parents or guardians
• the patients' visits to their doctor as part of their usual care The researchers will collect information about the adults for up to about 2 years and about the children for up to about 8 years.

Study Overview

• Status: Recruiting
• Conditions: Advanced or Recurrent Solid Tumor Harboring an NTRK Gene Fusion
• Intervention / Treatment: Drug: Larotrectinib (Vitrakvi, BAY2757556)

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: Bayer Clinical Trials Contact; Phone Number: (+)1-888-84 22937; Email: clinical-trials-contact@bayer.com

Study Locations

• Japan
  • Multiple Locations, Japan
    • Recruiting
    • Many Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult and pediatric (from birth to 18 year old) patients treated with larotrectinib.

Description

Inclusion Criteria:

• Patients who are treated with larotrectinib or decided to be treated with larotrectinib, under routine clinical practice.

Exclusion Criteria:

• Participation in an investigational program with interventions outside of routine clinical practice

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

8

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Gastrointestinal (GI); Participants with GI cancer.	Drug: Larotrectinib (Vitrakvi, BAY2757556); Follow clinical practice
Head and neck (H&N); Participants with H&N cancer.	Drug: Larotrectinib (Vitrakvi, BAY2757556); Follow clinical practice
Lung; Participants with lung cancer.	Drug: Larotrectinib (Vitrakvi, BAY2757556); Follow clinical practice
Soft tissue sarcoma (STS); Participants with STS cancer.	Drug: Larotrectinib (Vitrakvi, BAY2757556); Follow clinical practice
Primary central nervous system (CNS); Participants with CNS cancer.	Drug: Larotrectinib (Vitrakvi, BAY2757556); Follow clinical practice
Melanoma; Participants with Melanoma cancer.	Drug: Larotrectinib (Vitrakvi, BAY2757556); Follow clinical practice
Pediatrics	Drug: Larotrectinib (Vitrakvi, BAY2757556); Follow clinical practice
Others	Drug: Larotrectinib (Vitrakvi, BAY2757556); Follow clinical practice

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Severity of treatment emergent adverse events (TEAEs)	Approximate 8 years
Frequency of TEAEs	Approximate 8 years
Seriousness of TEAEs	Approximate 8 years
Outcome of TEAEs	Approximate 8 years
Causality assessment of TEAEs	Approximate 8 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall response rate (ORR), based on investigator assessment preferably using Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 or Response Assessment in Neuro-Oncology (RANO) as appropriate by local investigator assessment		Approximate 8 years
Disease control rate (DCR)		Approximate 8 years
Duration of response (DOR)		Approximate 8 years
Time to response (TTR)		Approximate 8 years
Progression-free survival (PFS)		Approximate 8 years
Overall survival (OS)		Approximate 8 years
Total dose		Approximate 8 years
Starting and ending dose		Approximate 8 years
Dose modification during treatment		Approximate 8 years
Duration of treatment (DOT)		Approximate 8 years
Baseline characteristics	Including but not limited to: age, NTRK gene, NTRK gene partner, prior therapy (type, number of lines of therapy), other.	Approximate 8 years
Neurological examination (normal/abnormal)		Approximate 8 years
Change in height from baseline by visit		Approximate 8 years
Change in weight from baseline by visit		Approximate 8 years
Developmental milestones abnormalities (normal/abnormal)		Approximate 8 years

Collaborators and Investigators

• Sponsor: Bayer

Study record dates

Study Major Dates

• Study Start (Actual): November 5, 2021
• Primary Completion (Estimated): June 30, 2029
• Study Completion (Estimated): December 31, 2029

Study Registration Dates

• First Submitted: June 20, 2021
• First Submitted That Met QC Criteria: June 29, 2021
• First Posted (Actual): June 30, 2021

Study Record Updates

• Last Update Posted (Actual): April 14, 2026
• Last Update Submitted That Met QC Criteria: April 13, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Recurrence; larotrectinib
• Other Study ID Numbers: 21793

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

IPD Plan Description

Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No