A Retro-/Prospective, Non-interventional, Cohort Study in Adult Patients With Locally Advanced or Metastatic Tumors With a Neurotrophic Tyrosine Receptor Kinase (NTRK) Gene Fusion, Treated With Larotrectinib

NCT ID: NCT04814667

Last Updated: 2024-12-20

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 26 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2021-02-23
• Study Completion Date: 2024-09-15

Brief Summary

Larotrectinib, a selective TRK inhibitor has showed marked and durable antitumor activity in patients with NTRK gene-fusion-positive tumors regardless of the tumor type, gene partner and patient's age. Because of this and the lack of alternative therapy in this rare but severe disease, the French National Agency for Medicines and Health Products Safety (ANSM) granted in April 2019, a "cohort" Temporary Authorization for Use (ATU) in the indication:"Larotrectinib is indicated as monotherapy for the treatment of adult and paediatric patients from one month, with locally advanced or metastatic solid tumours with a Neurotrophic Tyrosine Receptor Kinase (NTRK) fusion, refractory to standard treatments or in the absence of appropriate therapeutic alternative." Despite the potential benefit of identifying these fusions, the clinicopathologic features of NTRK fusion-positive tumors which are treated with Larotrectinib, are not well characterized. This study will provide information about the diagnosis and management of patients with locally advanced or metastatic NTRK fusion cancer treated with Larotrectinib under real-world treatment conditions in France, and describes the dosing patterns, safety and effectiveness of this agent.

Detailed Description

Tropomyosin receptor kinases (TRK) are a family of tyrosine kinases that bind neurotrophins, a family of growth factors important to the formation and function of the nervous system. In cancer, the neurotrophic tyrosine kinase receptor (NTRK)1, NTRK2 and NTRK3 genes, which encode for the TRKA, TRKB and TRKC proteins, respectively, are subject to gene-arrangements that lead to kinase domain expression and constitutive downstream pathway activation. In preclinical models, NTRK gene fusions have transformative oncogenic potential, and they appear to be widely distributed across histologically diverse adult and pediatric cancers. Hence, these genetic abnormalities, observed in both children and adults, have recently emerged as targets for cancer therapy.

Larotrectinib, a selective TRK inhibitor has showed marked and durable antitumor activity in patients with NTRK gene-fusion-positive tumors regardless of the tumor type, gene partner and patient's age.

Because of this and the lack of alternative therapy in this rare but severe disease, the French National Agency for Medicines and Health Products Safety (ANSM) granted in April 2019, a "cohort" Temporary Authorization for Use (ATU) in the indication:

"Larotrectinib is indicated as monotherapy for the treatment of adult and paediatric patients from one month, with locally advanced or metastatic solid tumours with a Neurotrophic Tyrosine Receptor Kinase (NTRK) fusion, refractory to standard treatments or in the absence of appropriate therapeutic alternative." Despite the potential benefit of identifying these fusions, the clinicopathologic features of NTRK fusion-positive tumors which are treated with Larotrectinib, are not well characterized.

This study will provide information about the diagnosis and management of patients with locally advanced or metastatic NTRK fusion cancer treated with Larotrectinib under real-world treatment conditions in France, and describes the dosing patterns, safety and effectiveness of this agent.

Conditions

Solid Tumor, Adult; Locally Advanced Solid Tumor; Metastatic Cancer

Keywords

NTRK gene fusion; Larotrectinib; Neurotrophic Tyrosine Receptor Kinase gene fusion; NTRK rearrangements

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• Adult male or female patients
• Histological confirmed diagnosis of advanced/metastatic solid tumor type.
• Patients previously, currently or to be treated with Larotrectinib within the ATU/post-ATU period. Patient must be > 25 years-old at time of larotrectinib start.
• Patients not opposed to collection of personal clinical data

• Minimum Eligible Age: 25 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Bayer

INDUSTRY

Sponsor Role: collaborator

Centre Leon Berard

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Armelle DUFRESNE

Role: PRINCIPAL_INVESTIGATOR

Centre Leon Berard

Locations

CHU Amiens

Amiens, , France

Centre Georges Francois Leclerc

Dijon, , France

Centre Leon Berard

Lyon, , France

Chi Elbeuf Louviers

Saint-Aubin-lès-Elbeuf, , France

Countries

France

Other Identifiers

LAROTRACKING

Identifier Type: -

Identifier Source: org_study_id