A Study Called EPI VITRAKVI to Compare Treatment Results in Patients With Infantile Fibrosarcoma (IFS), a Type of Connective Soft Tissue Cancer, Who Received a Treatment Called Larotrectinib From a Study Called SCOUT With Patient Data From an External Database

Sponsor

Bayer (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT05236257

Collaborator

(none)

Enrollment

Location

4.7

Anticipated Duration (Months)

15.5

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is an observational study in which data from the past of children and young people with a specific cancer, called NTRK gene fusion positive infantile fibrosarcoma (IFS) is studied.

IFS is a rare type of childhood cancer that commonly affects legs and arms. IFS cancers typically have specific changes in their building plans (genes) called NTRK gene fusion. NTRK stands for the specific gene that has been altered, the neurotrophic tyrosine kinase (NTRK) gene.

This change to the building plan leads to the creation of an altered protein known as a TRK fusion protein, which can cause cancer cells to grow and to survive. The specific cancer is therefore also called TRK (tropomyosin receptor kinase) fusion-positive IFS.

The study drug, larotrectinib (also called BAY2757556) works by blocking the altered TRK fusion protein. Larotrectinib is already available in Europe and in many other countries and is approved for doctors to prescribe to patients with NTRK gene fusion cancer which has spread to nearby tissues and/or lymph nodes or to other parts of the body.

In France, HAS (the French authority in charge of evaluating health products and technologies) gave a positive opinion for the reimbursement of larotrectinib but only in the pediatric patients with IFS or another STS harboring a NTRK gene fusion, which is locally advanced or metastatic, and refractory or in relapse mainly due to the lack of comparative evidence.

The main purpose of this study is to collect more data to learn how well larotrectinib works compared with current standard of care chemotherapy in people up to 21 years of age with NTRK gene fusion positive IFS that has spread to nearby tissues and/or lymph nodes (locally advanced) or other parts of the body (metastatic).

To see how well larotrectinib works, researchers will make a comparison between

how long larotrectinib works well and
how long the standard of care works well.

Working well means that the treatments can prevent the following from happening:

need for a new treatment for the cancer
need for radiation therapy for the cancer
need for surgery to treat the cancer, but which causes major damage to body parts
death.

In addition to the above, data about medical problems related to the treatments in both groups and that may have required to stop the treatment will be compared.

The data for the comparison will come from

an ongoing international study called SCOUT which was started in December 2015 (larotrectinib group)
international databases (standard of care chemotherapy group). Data will be from the year 2000 up to the present.

There will be no required visits with a study doctor or required tests in this study.

Condition or Disease	Intervention/Treatment	Phase
Locally Advanced or Metastatic Infantile Fibrosarcoma Harboring an NTRK Gene Fusion Infantile Fibrosarcoma	Drug: Larotrectinib (Vitrakvi, BAY2757556) Drug: Standard of Care

Study Design

Study Type:

Observational

Anticipated Enrollment :

73 participants

Observational Model:

Cohort

Time Perspective:

Retrospective

Official Title:

A Comparison of Clinical Outcomes in Infantile Fibrosarcoma (IFS) Patients Treated With Larotrectinib in the Phase I/II SCOUT Study Versus (an) External Historical Cohort(s)

Actual Study Start Date :

Mar 10, 2022

Anticipated Primary Completion Date :

Jul 31, 2022

Anticipated Study Completion Date :

Jul 31, 2022

Arms and Interventions

Arm	Intervention/Treatment
Larotrectinib Pediatric patients with IFS harboring an NTRK gene fusion who have been enrolled in the SCOUT study.	Drug: Larotrectinib (Vitrakvi, BAY2757556) Pediatric patients with IFS harboring an NTRK gene fusion.
Standard care Pediatric patients with IFS harboring an NTRK gene fusion in the eligible external cohort(s).	Drug: Standard of Care Standard of care for the patients from the eligible external cohorts.

Arm

Intervention/Treatment

Larotrectinib

Pediatric patients with IFS harboring an NTRK gene fusion who have been enrolled in the SCOUT study.

Drug: Larotrectinib (Vitrakvi, BAY2757556)

Pediatric patients with IFS harboring an NTRK gene fusion.

Standard care

Pediatric patients with IFS harboring an NTRK gene fusion in the eligible external cohort(s).

Drug: Standard of Care

Standard of care for the patients from the eligible external cohorts.

Outcome Measures

Primary Outcome Measures

Time to treatment failure [Retrospective data analysis 2000 to July 2021]
Defined as the time (months) from the start of treatment to the date of the following events, whichever comes first: subsequent systemic treatment, radiation therapy, mutilating surgery or death due to any cause.

Secondary Outcome Measures

Time to subsequent systemic treatment [Retrospective data analysis 2000 to July 2021]
Calculated from the start date of larotrectinib treatment for the SCOUT study and start date of chemotherapy (first line) for the external historical control cohort(s) to the start date of a post-treatment systemic anti-cancer therapy, if any.
Time to mutilating surgery including limb amputation [Retrospective data analysis 2000 to July 2021]
Calculated from the start date of larotrectinib treatment for the SCOUT study and start date of chemotherapy (first line) for the external historical control cohort(s) to the date of a post-treatment mutilating surgery including limb amputation, if any.
Time to radiation therapy. [Retrospective data analysis 2000 to July 2021]
Calculated from the start date of larotrectinib treatment for the SCOUT study and start date of chemotherapy (first line) for the external historical control cohort(s) to the start date of a post-treatment radiation therapy, if any.
Time to complete surgical resection [Retrospective data analysis 2000 to July 2021]
Calculated from the start date of larotrectinib treatment for the SCOUT study and start date of chemotherapy (first line) for the external historical control cohort(s) to the date of a post-treatment complete surgical resection (excluding amputation), if any.
Overall Survival (OS) [Retrospective data analysis 2000 to July 2021]
Start date of larotrectinib treatment for the SCOUT study and start date of chemotherapy (first line) for the external historical control cohort(s), Date of death due to any cause, if applicable, or, Last date of patient follow-up for patients alive and lost to follow-up, if applicable.
Number of participants with treatment discontinuation due to treatment-related adverse events [Retrospective data analysis 2000 to July 2021]
Larotrectinib treatment for the SCOUT study and chemotherapy (first line) for the external historical control cohort(s).

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 21 Years

Sexes Eligible for Study:

All

Inclusion Criteria:

Age ≤ 21 years old.
Locally advanced or metastatic Infantile Fibrosarcoma (IFS).
Identification of an NTRK gene fusion by a molecular biology assay.
Patients with available information on clinical, radiological characteristics of their tumor, therapies administered and outcomes.
Patients receiving larotrectinib in the SCOUT trial.
Patients receiving at least chemotherapy drugs in the historical control cohort(s).
No opposition from the patients and/or representatives for data use.

Exclusion Criteria:

Patients treated with TRK inhibitors in the historical control cohort(s).
Patients with documented absence of NTRK gene fusion.
Patients participating in an investigational program with interventions outside of routine clinical practice.