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

NCT ID: NCT05236257

Last Updated: 2024-12-10

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 93 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2022-03-10
• Study Completion Date: 2022-09-13

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.

Conditions

Locally Advanced or Metastatic Infantile Fibrosarcoma Harboring an NTRK Gene Fusion; Infantile Fibrosarcoma

Keywords

Advanced solid tumors; Infantile fibrosarcoma (IFS); Neurotrophic tyrosine receptor kinase (NTRK); Fusion Positive; Tropomyosin Receptor Kinase (TRK) fusion; Larotrectinib; External control

Study Design

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

Study Groups

Larotrectinib

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

Larotrectinib (Vitrakvi, BAY2757556)

Intervention Type: DRUG

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).

Standard of Care

Intervention Type: DRUG

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

Interventions

Larotrectinib (Vitrakvi, BAY2757556)

Pediatric patients with IFS harboring an NTRK gene fusion.

Intervention Type: DRUG

Standard of Care

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

Intervention Type: DRUG

Eligibility Criteria

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.

• Maximum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Bayer

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Multiple Locations

Multiple Locations, , France

Countries

France

References

Carton M, Del Castillo JP, Colin JB, Kurtinecz M, Feuilly M, Pierron G, Arvis P, Khadir SK, Sparber-Sauer M, Orbach D. Larotrectinib versus historical standard of care in patients with infantile fibrosarcoma: protocol of EPI-VITRAKVI. Future Oncol. 2023 Aug;19(24):1645-1653. doi: 10.2217/fon-2023-0114. Epub 2023 May 3.

Reference Type: DERIVED

PMID: 37133249 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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VITRAKVI Patient Information

http://clinicaltrials.bayer.com

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Other Identifiers

21767

Identifier Type: -

Identifier Source: org_study_id