Clinical trial NCT04945330: NTRK Gene Fusion - Positive Advanced or Recurrent Solid Tumors, a Rare Cancer Caused by Specific Changes in the Genes
Official title: Special Drug Use Investigation for Larotrectinib
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Advanced or Recurrent Solid Tumor Harboring an NTRK Gene Fusion
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2021-06-30
Enrollment: 100
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult and pediatric (from birth to 18 year old) patients treated with larotrectinib.

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Severity of treatment emergent adverse events (TEAEs)
timeFrame: Approximate 8 years
measure: Frequency of TEAEs
timeFrame: Approximate 8 years
measure: Seriousness of TEAEs
timeFrame: Approximate 8 years
measure: Outcome of TEAEs
timeFrame: Approximate 8 years
measure: Causality assessment of TEAEs
timeFrame: Approximate 8 years
Secondary outcomes:
measure: 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
timeFrame: Approximate 8 years
measure: Disease control rate (DCR)
timeFrame: Approximate 8 years
measure: Duration of response (DOR)
timeFrame: Approximate 8 years
measure: Time to response (TTR)
timeFrame: Approximate 8 years
measure: Progression-free survival (PFS)
timeFrame: Approximate 8 years
measure: Overall survival (OS)
timeFrame: Approximate 8 years
measure: Total dose
timeFrame: Approximate 8 years
measure: Starting and ending dose
timeFrame: Approximate 8 years
measure: Dose modification during treatment
timeFrame: Approximate 8 years
measure: Duration of treatment (DOT)
timeFrame: Approximate 8 years
measure: Baseline characteristics
timeFrame: Approximate 8 years
measure: Neurological examination (normal/abnormal)
timeFrame: Approximate 8 years
measure: Change in height from baseline by visit
timeFrame: Approximate 8 years
measure: Change in weight from baseline by visit
timeFrame: Approximate 8 years
measure: Developmental milestones abnormalities (normal/abnormal)
timeFrame: Approximate 8 years
Other outcomes:
Locations:
city: Multiple Locations
country: Japan
facility: Many locations
Study officials:
References: