Trial Outcomes & Findings for 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 NCT05236257)

NCT ID: NCT05236257

Last Updated: 2024-12-10

Results Overview

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.

• Recruitment status: COMPLETED
• Target enrollment: 93 participants
• Primary outcome timeframe: up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control
• Results posted on: 2024-12-10

Participant Flow

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.

In total, 93 patients were included in this study (IFS population). The larotrectinib arm of the study included 51 patients from the SCOUT study. The single comparator arm of patients that received conventional chemotherapy included in total 42 external control patients, pooled from the Institut Curie database (N=18) and the CWS database (N=24).

Participant milestones

Measure	Larotrectinib Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.	External Controls External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).
Overall Study STARTED	51	42
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	51	42

Reasons for withdrawal

Measure	Larotrectinib Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.	External Controls External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).
Overall Study Ongoing with study treatment	29	1
Overall Study Discontinued study treatment	22	41

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Larotrectinib n=51 Participants Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.	External Controls n=42 Participants External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).	Total n=93 Participants Total of all reporting groups
Age, Continuous	2.01 years STANDARD_DEVIATION 3.49 • n=5 Participants	0.73 years STANDARD_DEVIATION 1.81 • n=7 Participants	1.43 years STANDARD_DEVIATION 2.91 • n=5 Participants
Sex: Female, Male Female	21 Participants n=5 Participants	15 Participants n=7 Participants	36 Participants n=5 Participants
Sex: Female, Male Male	30 Participants n=5 Participants	27 Participants n=7 Participants	57 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	51 Participants n=5 Participants	42 Participants n=7 Participants	93 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Measure	Larotrectinib n=51 Participants Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.	External Controls n=42 Participants External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).
Time to Medical Treatment Failure Unweighted sample	NA months median time to medical treatment failure was not estimable due to censored data	NA months Interval 57.1 to median time to medical treatment failure was not estimable due to censored data
Time to Medical Treatment Failure Weighted sample	NA months median time to medical treatment failure was not estimable due to censored data	24.0 months Interval 3.0 to upper limit of median time to medical treatment failure was not estimable due to censored data

SECONDARY outcome

Timeframe: 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 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

Outcome measures

Measure	Larotrectinib n=51 Participants Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.	External Controls n=42 Participants External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).
Time to Subsequent Systemic Treatment Unweighted Sample	NA months median time to subsequent systemic treatment was not estimable due to censored data	NA months Interval 13.0 to median time to subsequent systemic treatment was not estimable due to censored data
Time to Subsequent Systemic Treatment weighted Sample	NA months median time to subsequent systemic treatment was not estimable due to censored data	24.0 months Interval 24.0 to Upper limit of median time to subsequent systemic treatment was not estimable due to censored data

SECONDARY outcome

Timeframe: 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 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)

Outcome measures

Measure	Larotrectinib n=51 Participants Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.	External Controls n=42 Participants External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).
Time to Mutilating Surgery Including Limb Amputation Unweighted Sample	NA months median time to mutilating surgery including limb amputation was not estimable due to censored data	NA months median time to mutilating surgery including limb amputation was not estimable due to censored data
Time to Mutilating Surgery Including Limb Amputation Weighted Sample	NA months median time to mutilating surgery including limb amputation was not estimable due to censored data	NA months median time to mutilating surgery including limb amputation was not estimable due to censored data

SECONDARY outcome

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

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

Outcome measures

Measure	Larotrectinib n=51 Participants Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.	External Controls n=42 Participants External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).
Time to First Radiation Therapy Unweighted Sample	NA months median time to first radiation therapy was not estimable due to censored data	NA months median time to first radiation therapy was not estimable due to censored data
Time to First Radiation Therapy Weighted Sample	NA months median time to first radiation therapy was not estimable due to censored data	NA months median time to first radiation therapy was not estimable due to censored data

SECONDARY outcome

Timeframe: 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 (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

Outcome measures

Measure	Larotrectinib n=51 Participants Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.	External Controls n=42 Participants External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).
Time to Complete Surgical Resection Unweighted Sample	NA months median time to complete surgical resection was not estimable due to censored data	NA months median time to complete surgical resection was not estimable due to censored data
Time to Complete Surgical Resection Weighted Sample	NA months median time to complete surgical resection was not estimable due to censored data	6.1 months Interval 5.1 to upper limit of median time to complete surgical resection was not estimable due to censored data

SECONDARY outcome

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

Number of participants with death due to any cause, alive, or lost to follow-up.

Outcome measures

Measure	Larotrectinib n=51 Participants Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.	External Controls n=42 Participants External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).
Overall Survival Alive	50 Participants	39 Participants
Overall Survival Dead	1 Participants	3 Participants

SECONDARY outcome

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

Larotrectinib treatment for the SCOUT study and chemotherapy (first line) for the external historical control cohort(s).

Outcome measures

Measure	Larotrectinib n=51 Participants Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.	External Controls n=42 Participants External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).
Number of Participants With Treatment Discontinuation Due to Treatment Emergent Adverse Events	0 Participants	1 Participants

Adverse Events

Larotrectinib

Serious events: 1 serious events

Other events: 0 other events

Deaths: 1 deaths

External Controls

Serious events: 3 serious events

Other events: 0 other events

Deaths: 3 deaths

Serious adverse events

Measure	Larotrectinib n=51 participants at risk Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.	External Controls n=42 participants at risk External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).
General disorders Death	2.0% 1/51 • Number of events 1 • up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control As it was stated in the protocol of the study, this retrospective observational study used secondary data collection from a previous clinical trial (SCOUT study) and from eligible databases used to select external historical control cohorts. Therefore, no new AEs or adverse drug reactions occurred besides the ones already described during the conduct of the initial clinical trial.	7.1% 3/42 • Number of events 3 • up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control As it was stated in the protocol of the study, this retrospective observational study used secondary data collection from a previous clinical trial (SCOUT study) and from eligible databases used to select external historical control cohorts. Therefore, no new AEs or adverse drug reactions occurred besides the ones already described during the conduct of the initial clinical trial.

Other adverse events

Adverse event data not reported

Additional Information

Therapeutic Area Head

Bayer

Phone: (+) 1-888-8422937

Email: clinical-trials-contact@bayer.com

Results disclosure agreements

• Principal investigator is a sponsor employee The disclosure agreement is that the publication is restricted for external partners.
• Publication restrictions are in place

Restriction type: OTHER