RecruitingObservational

PrOspective Non-interventional Study in Patients With Locally Advanced or Metastatic TRK Fusion Cancer Treated With Larotrectinib

NCT ID: NCT04142437Sponsor: BayerLast updated: 2026-04-14

Summary

In this observational study researcher want to learn more about the effectiveness of drug VITRAKVI (generic name: larotrectinib) and how well the drug is tolerated during routine use in patients with TRK fusion cancer which is locally advanced or spread from the place where it started to other places in the body. TRK fusion cancer is a term used to describe a variety of common and rare cancers that are caused by a change to the NTRK (Neurotrophic Tyrosine Kinase) gene called a fusion. During this fusion, an NTRK gene joins together, or fuses, with a different gene. This joining results in the activation of certain proteins (TRK fusion proteins), which can cause cancer cells to multiply and form a tumor. VITRAKVI is an approved drug that blocks the action of the NTRK gene fusion. This study will enroll adult and paediatric patients suffering from a solid tumor with NTRK gene fusion for whom the decision to treat their disease with VITRAKVI has been made by their treating physicians. During the study, patients' medical information such as treatment information with VITRAKVI, other medication or treatments, changes in disease status and other health signs and symptoms will be collected within the normal medical care by the treating doctor. Participants will be observed over a period from 24 to 60 months.

Arms & interventions

Druglarotrectinib(Vitrakvi, BAY2757556)
In the study, patients treated under local standard of care clinical practice; all decisions in terms of diagnostic procedures, treatments, management of the disease, and resource utilization are fully dependent on mutual agreement between the patient and the attending physician, without interference by the study initiator or study protocol

Outcome measures

Primary

Number of participants with treatment-emergent adverse events (TEAEs)
Time frame: Up to 30 days after last dose
Severity of TEAEs
Time frame: Up to 30 days after last dose
Seriousness of TEAEs
Time frame: Up to 30 days after last dose
Reasonable causal relationship between larotrectinib and an AE
Time frame: Up to 30 days after last dose
Causality of TEAEs
Time frame: Up to 30 days after last dose
Action taken related to larotrectinib treatment
Time frame: Up to 30 days after last dose

Secondary

Objective response rate (ORR)
Time frame: Up to 8 years
Disease control rate (DCR)
Time frame: Up to 8 years
Duration of response (DOR)
Time frame: Up to 8 years
Time to response (TTR)
Time frame: Up to 8 years
Progression-free survival (PFS)
Time frame: Up to 8 years
Overall survival (OS)
Time frame: Up to 8 years
Total dose
Time frame: Up to 8 years
Starting and ending dose
Time frame: Up to 8 years
Dose modification during treatment
Time frame: Up to 8 years
Duration of treatment (DOT)
Time frame: Up to 8 years
ORR by patient subgroup(s)
Time frame: Up to 8 years
DCR by patient subgroup(s)
Time frame: Up to 8 years
DOR by patient subgroup(s)
Time frame: Up to 8 years
TTR by patient subgroup(s)
Time frame: Up to 8 years
PFS by patient subgroup(s)
Time frame: Up to 8 years
OS by patient subgroup(s)
Time frame: Up to 8 years
Number of patients with change in height and weight from baseline by visit, neurological abnormalities (normal/abnormal)
Time frame: Up to 8 years
Number of patients with abnormal developmental milestones
Time frame: Up to 8 years
Number of patients with abnormal Tanner stage
Time frame: Up to 8 years

Eligibility criteria

Sex: AllAge: All agesHealthy volunteers: No

Inclusion Criteria: * Adult and pediatric (from birth to 18-year-old) patients * Patients with locally advanced or metastatic solid tumor harboring an NTRK gene fusion. NTRK (NTRK1, NTRK2, and NTRK3) gene fusions will be identified locally. Acceptable methods of detection of NTRK gene fusion include NGS, fluorescence in situ hybridization (FISH), reverse-transcription polymerase chain reaction (rt-PCR) or any other genomic testing able to detect NTRK gene fusion. If a pan-TRK IHC method is used, this result needs to be accompanied with the results using one of the other methods noted above. * Life expectancy of at least 3 months based on clinical judgement * Decision to treat with larotrectinib made by the treating physician prior to study enrollment * Patients can also be enrolled if the initial visit (larotrectinib start date) occurred within 2 months ±3 days prior to informed consent signed date * Signed informed consent form * For patients under legal age, signed assent by the patient (where applicable) and parental/legal guardian signed informed consent is required Exclusion Criteria: * Any contraindications as listed in the local approved product information * Pregnancy * Participation in an investigational program with interventions outside of routine clinical practice * Prior treatment with larotrectinib or other kinase inhibitor with TRK inhibition * Patients with NTRK gene amplification or NTRK point mutation

Study locations (51)

Banner Desert Medical Center

Mesa, Arizona, 85202

Withdrawn

California Research Inst.

Los Angeles, California, 90027

Recruiting

USC / Norris Comprehensive Cancer Center

Los Angeles, California, 90089

Withdrawn

UCLA - Mattel Children's Hospital

Los Angeles, California, 90095

Recruiting

Hoag Memorial Hospital Presbyterian

Newport Beach, California, 82663

Recruiting

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609

Withdrawn

Stanford Univ Med Ctr. / Lucile Packard Children's Hosp

Palo Alto, California, 94304

Recruiting

Providence Health System - Southern California

Santa Monica, California, 90404

Terminated

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Torrance, California, 90502

Withdrawn

SCL Health

Grand Junction, Colorado, 81501

Withdrawn

Yale University

New Haven, Connecticut, 06520

Recruiting

Mayo Clinic

Jacksonville, Florida, 32224

Withdrawn

University of Miami

Miami, Florida, 33146

Recruiting

Nicklaus Children's Hospital

Miami, Florida, 33155

Recruiting

Nemours Children's Hospital

Orlando, Florida, 32827

Withdrawn

Fort Wayne Medical Oncology Hematology

Fort Wayne, Indiana, 46804

Recruiting

Regional Health Hope Center

Terre Haute, Indiana, 47802

Withdrawn

Cancer Center of Kansas

Wichita, Kansas, 67214

Recruiting

Maine Health

South Portland, Maine, 04106

Recruiting

Univ. of Maryland / Greenebaum Comp. Cancer Ctr.

Baltimore, Maryland, 21201

Withdrawn

Johns Hopkins / Sidney Kimmel Cancer Center

Baltimore, Maryland, 21205

Recruiting

Frederick Health-James M Stockman Cancer Institute

Frederick, Maryland, 21702

Terminated

Tufts / Neely Cancer Center

Boston, Massachusetts, 02111

Recruiting

Boston Children's / Dana Farber

Boston, Massachusetts, 02215

Withdrawn

Detroit Clinical Research Center

Farmington Hills, Michigan, 48334

Recruiting

Sparrow Cancer Center

Lansing, Michigan, 48912

Recruiting

Nevada Cancer Research Foundation

Las Vegas, Nevada, 89169

Recruiting

Atlantic Hem Onc / Morristown Medical Center

Morristown, New Jersey, 07960

Withdrawn

Rutgers Cancer Institute of New Jersey

New Brunswick, New Jersey, 08901

Withdrawn

Great Lakes Cancer Center

Buffalo, New York, 14210

Terminated

Memorial Sloan Kettering Children's Cancer Center

New York, New York, 10065

Recruiting

Staten Island Univ. Hospital (Northwell Health)

Staten Island, New York, 10305

Withdrawn

Levine Cancer Center

Charlotte, North Carolina, 28204

Withdrawn

East Carolina University / Vidant Health

Greenville, North Carolina, 27834

Recruiting

Ohio State Comp. Cancer Ctr. / James Cancer Hospital

Columbus, Ohio, 43210

Withdrawn

Mercy Health Youngstown

Youngstown, Ohio, 44501

Recruiting

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

Recruiting

University of Pennsylvania (Penn Med)

Philadelphia, Pennsylvania, 19104

Withdrawn

Allegheny Health Network

Pittsburgh, Pennsylvania, 15212

Terminated

Medical Univ. of South Carolina

Charleston, South Carolina, 29425

Recruiting

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105

Withdrawn

Vanderbilt University Medical Center

Nashville, Tennessee, 37232

Recruiting

UT Southwestern Medical Center / Children's Health

Dallas, Texas, 75390

Recruiting

MD Anderson Cancer Center

Houston, Texas, 77030

Recruiting

Intermountain Healthcare - Intermountain Medical Center

Murray, Utah, 84107

Withdrawn

Univ. of Utah / Huntsman Cancer Center

Salt Lake City, Utah, 84112

Recruiting

Intermountain Healthcare - Dixie Regional Medical Center

St. George, Utah, 84790

Withdrawn

Seattle Children's

Seattle, Washington, 98105

Recruiting

West Virginia University

Morgantown, West Virginia, 26506

Recruiting

Gundersen Health System

La Crosse, Wisconsin, 54601

Recruiting

SSM Health Cancer Center - Dean Medical Group

Madison, Wisconsin, 53717

Terminated

References

Yang JCH, Brose MS, Castro G, Kim ES, Lassen UN, Leyvraz S, Pappo A, Lopez-Rios F, Reeves JA, Fellous M, Penault-Llorca F, Rudzinski ER, Tabatabai G, Vassal G, Drilon A, Trent J. Rationale and design of ON-TRK: a novel prospective non-interventional study in patients with TRK fusion cancer treated with larotrectinib. BMC Cancer. 2022 Jun 7;22(1):625. doi: 10.1186/s12885-022-09687-x.(PubMed)