Locally Advanced or Metastatic Infantile Fibrosarcoma Harboring an NTRK Gene FusionEPI VITRAKVINCT05236257
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
An observational study of Locally Advanced or Metastatic Infantile Fibrosarcoma Harboring an NTRK Gene Fusion and Infantile Fibrosarcoma, run by Bayer at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Locally Advanced or Metastatic Infantile Fibrosarcoma Harboring an NTRK Gene Fusion, Infantile Fibrosarcoma
- What is being tested?
- Larotrectinib (Vitrakvi, BAY2757556)
- How many people
- 93 participants
- Where
- 1 siteFrance
- Study dates
- 10 Mar 2022to 13 Sep 2022
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- Child, Adult
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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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)
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.
Read more
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
- Being tested
- Larotrectinib (Vitrakvi, BAY2757556), Standard of Care
- Keywords
- Advanced solid tumors, Infantile fibrosarcoma (IFS), Neurotrophic tyrosine receptor kinase (NTRK), Fusion Positive, Tropomyosin Receptor Kinase (TRK) fusion, Larotrectinib, External control
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Larotrectinib (Vitrakvi, BAY2757556)Drug
Pediatric patients with IFS harboring an NTRK gene fusion.
Standard of CareDrug
Standard of care for the patients from the eligible external cohorts.
| Study group | Role | What they receive |
|---|---|---|
| Larotrectinib | Pediatric patients with IFS harboring an NTRK gene fusion who have been enrolled in the SCOUT study. Receives: Drug: Larotrectinib (Vitrakvi, BAY2757556) | |
| Standard care | Pediatric patients with IFS harboring an NTRK gene fusion in the eligible external cohort(s). Receives: Drug: Standard of Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
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
Measured: 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)
Measured: 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
Measured: 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
Measured: 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.
Measured: 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).
Measured: up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2022
- Study start10 Mar 2022
- Primary completion13 Sep 2022
- Study completion13 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Locally Advanced or Metastatic Infantile Fibrosarcoma Harboring an NTRK Gene FusionStudies of the same condition, open and under way.
- More trials run by BayerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 93 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05236257
- Sponsor's protocol ID
- 21767
- Acronym
- EPI VITRAKVI
- Status last verified
- Dec 2024
- First submitted
- 2 Feb 2022
- Last updated
- 10 Dec 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- 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.
Publications
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.
PubMed 37133249
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT05236257
- Acronym / sponsor ID
- EPI VITRAKVI / 21767
- 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 type
- Observational
- Recruitment status
- Completed
- Enrolment
- 93 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 10 Mar 2022 (actual)
- Primary completion
- 13 Sep 2022 (actual)
- Study completion
- 13 Sep 2022 (actual)
- First posted
- 11 Feb 2022 (actual)
- Results
- Posted 10 Dec 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Larotrectinib | – | Drug: Larotrectinib (Vitrakvi, BAY2757556)Pediatric patients with IFS harboring an NTRK gene fusion who have been enrolled in the SCOUT study. |
| Standard care | – | Drug: Standard of CarePediatric patients with IFS harboring an NTRK gene fusion in the eligible external cohort(s). |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Larotrectinib (Vitrakvi, BAY2757556)Pediatric patients with IFS harboring an NTRK gene fusion. | Drug | Larotrectinib |
| Standard of CareStandard of care for the patients from the eligible external cohorts. | Drug | Standard care |
Primary outcome
Time to Medical Treatment Failure
Time frame: up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control
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.
6 secondary outcomes
Time to Subsequent Systemic Treatment
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 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 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 frame: up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control
Overall Survival
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
Time frame: up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Dec 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37133249Carton 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.
- VITRAKVI Patient InformationLink provided by the sponsor.
- Individual participant dataSharing planned: 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.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Locally Advanced or Metastatic Infantile Fibrosarcoma Harboring an NTRK Gene Fusion and Infantile Fibrosarcoma, run by Bayer at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Dec 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to Medical Treatment Failure (time frame: up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05236257, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Dec 2024.