An observational study in Locally Advanced or Metastatic Infantile Fibrosarcoma Harboring an NTRK Gene Fusion and Infantile Fibrosarcoma, sponsored by Bayer. Completed at 1 site in France. Open to participants aged Up to 21 Years. Per ClinicalTrials.gov, last updated 2024-12-10.
Sponsored by Bayer · Observational
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
Working well means that the treatments can prevent the following from happening:
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
There will be no required visits with a study doctor or required tests in this study.
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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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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 in the eligible external cohort(s).
Drug: Standard of Care
Pediatric patients with IFS harboring an NTRK gene fusion.
Standard of care for the patients from the eligible external cohorts.
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.
Time frame: up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control
Time to Subsequent Systemic Treatment
Time to subsequent systemic treatment was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a post-treatment systemic anti-cancer therapy, if any
Time frame: 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
Time to mutilating surgery including limb amputation was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a mutilating surgery (including limb amputation)
Time frame: 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
Time to radiation therapy was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy(for historical control cohorts) till the start date of a radiation therapy, if any
Time frame: 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
Time to complete surgical resection (excluding amputation) was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a complete surgical resection (excluding amputation), if any
Time frame: up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control
Overall Survival
Number of participants with death due to any cause, alive, or lost to follow-up.
Time frame: up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control
Number of Participants With Treatment Discontinuation Due to Treatment Emergent Adverse Events
Larotrectinib treatment for the SCOUT study and chemotherapy (first line) for the external historical control cohort(s).
Time frame: up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control
Study was conducted between 10-Mar-2022 (start retrospective observational study) and 13-Sep-2022 (table listing filles final) using secondary data. Three data sources were used in the study including SCOUT study (18 countries worldwide including France) and external historical control cohorts from the Institut Curie database and the Cooperative Weichteilsarkom Studiengruppe (CWS) database.
| Milestone | Larotrectinib | External Controls |
|---|---|---|
| Started | 51 | 42 |
| Completed | 0 | 0 |
| Not completed | 51 | 42 |
| Withdrew: Ongoing with study treatment | 29 | 1 |
| Withdrew: Discontinued study treatment | 22 | 41 |
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.
| months | Larotrectinib | External Controls |
|---|---|---|
| Unweighted sample | NA (NA to NA) | NA (57.1 to NA) |
| Weighted sample | NA (NA to NA) | 24.0 (3.0 to NA) |
Time to subsequent systemic treatment was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a post-treatment systemic anti-cancer therapy, if any
| months | Larotrectinib | External Controls |
|---|---|---|
| Unweighted Sample | NA (NA to NA) | NA (13.0 to NA) |
| weighted Sample | NA (NA to NA) | 24.0 (24.0 to NA) |
Time to mutilating surgery including limb amputation was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a mutilating surgery (including limb amputation)
| months | Larotrectinib | External Controls |
|---|---|---|
| Unweighted Sample | NA (NA to NA) | NA (NA to NA) |
| Weighted Sample | NA (NA to NA) | NA (NA to NA) |
Time to radiation therapy was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy(for historical control cohorts) till the start date of a radiation therapy, if any
| months | Larotrectinib | External Controls |
|---|---|---|
| Unweighted Sample | NA (NA to NA) | NA (NA to NA) |
| Weighted Sample | NA (NA to NA) | NA (NA to NA) |
Time to complete surgical resection (excluding amputation) was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a complete surgical resection (excluding amputation), if any
| months | Larotrectinib | External Controls |
|---|---|---|
| Unweighted Sample | NA (NA to NA) | NA (NA to NA) |
| Weighted Sample | NA (NA to NA) | 6.1 (5.1 to NA) |
Number of participants with death due to any cause, alive, or lost to follow-up.
| Participants | Larotrectinib | External Controls |
|---|---|---|
| Dead | 1 | 3 |
| Alive | 50 | 39 |
Larotrectinib treatment for the SCOUT study and chemotherapy (first line) for the external historical control cohort(s).
| Participants | Larotrectinib | External Controls |
|---|---|---|
| Number of Participants With Treatment Discontinuation Due to Treatment Emergent Adverse Events | 0 | 1 |
Collected over up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Larotrectinib | 1/51 (2%) | 1/51 (2%) | 0/51 (0%) |
| External Controls | 3/42 (7.1%) | 3/42 (7.1%) | 0/42 (0%) |
| Event | Larotrectinib | External Controls |
|---|---|---|
| DeathGeneral disorders | 1/51 | 3/42 |
| Age, Continuous(years) | Larotrectinib | External Controls | Total |
|---|---|---|---|
| Mean | 2.01 ± 3.49 | 0.73 ± 1.81 | 1.43 ± 2.91 |
| Sex: Female, Male(Participants) | Larotrectinib | External Controls | Total |
|---|---|---|---|
| Female | 21 | 15 | 36 |
| Male | 30 | 27 | 57 |
| Race (NIH/OMB)(Participants) | Larotrectinib | External Controls | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 0 | 0 | 0 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 51 | 42 | 93 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No — Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.
Ran from Mar 2022 to primary completion in Sep 2022, 6 months.
Results were first posted in Dec 2024, 2 years 2 months after primary completion.
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