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

NCT05236257 | Completed Results

• 1 other identifier: 21767
• Study Type: observational
• Target: 93
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• Time to Medical Treatment Failure

  Time to medical treatment failure was defined as the time (months) from the start of treatment to the date of the earliest event from: subsequent systemic treatment, radiation therapy, mutilating surgery or death due to any cause.

  up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control

Secondary Outcomes (6)

• Time to Subsequent Systemic Treatment

  up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control

• Time to Mutilating Surgery Including Limb Amputation

  up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control

• Time to First Radiation Therapy

  up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control

• Time to Complete Surgical Resection

  up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control

• Overall Survival

  up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control

Study Arms (2)

Larotrectinib

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

Drug: Larotrectinib (Vitrakvi, BAY2757556)

Standard care

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

Drug: Standard of Care

Interventions

Larotrectinib (Vitrakvi, BAY2757556) DRUG

Pediatric patients with IFS harboring an NTRK gene fusion.

Larotrectinib

Standard of Care DRUG

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

Standard care

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

The study population comprises of all pediatric patients in the SCOUT study and the eligible historical cohort(s) with a diagnosis of locally advanced or metastatic IFS harboring an NTRK gene fusion.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Bayer: lead

Study Sites (1)

Multiple Locations

Multiple Locations, France

Location

Related Publications (1)

• 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.

  PMID: 37133249 DERIVED

Related Links

• VITRAKVI Patient Information
• Click here to find information for studies related to Bayer products. To find this study enter the NCT number or Bayer Study ID in the search field.

MeSH Terms

Interventions

larotrectinib; Standard of Care

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Results Point of Contact

• Title: Therapeutic Area Head
• Organization: Bayer

clinical-trials-contact@bayer.com

(+) 1-888-8422937

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 2, 2022
• First Posted: February 11, 2022
• Study Start: March 10, 2022
• Primary Completion: September 13, 2022
• Study Completion: September 13, 2022
• Last Updated: December 10, 2024
• Results First Posted: December 10, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)