Clinical trial NCT04317599: A Retrospective Non Interventional Study on First Line Treatment for Patients With BRAFV600E Mutant Metastatic Colorectal Cancer (mCRC)
Official title: A Retrospective Non Interventional Study on First Line Treatment for Patients With BRAFV600E Mutant Metastatic Colorectal Cancer (mCRC)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: BRAF V600E Mutation Positive
Metastatic Colorectal Cancer
Keywords: first line
retrospective study
mCRC
BRAF V600E
Lead sponsor: Pierre Fabre Medicament
Sponsor class: INDUSTRY
Collaborators: name: Clinact
First posted: 2020-03-23
Enrollment: 274
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients diagnosed with BRAFV600E mutant mCRC (determined by local laboratory result) in the target countries and initiating first line treatment between January 1st, 2016 and December 31st, 2018 (both days inclusive) will be eligible for enrollment into the study.

Brief summary: The presence of a BRAFV600E mutation is considered a marker of poor prognosis in patients with mCRC, and findings from clinical trials have largely remained inconclusive regarding the efficacy of first line treatments for BRAF-mutant mCRC patients. In the absence of targeted/specific treatment for BRAF-mutant mCRC, treatment practices can vary based on local practices and guidelines. There is, therefore, an unmet need to document the current practices for first-line treatment of BRAF-mutant mCRC, and their effectiveness and safety in a real-world setting.

This real-world, multicenter non-interventional study (NIS) will describe the treatment patterns, effectiveness and safety of current treatment regimens in BRAFV600E mutant mCRC patients in Europe, with the aim to put the clinical study findings of the ongoing Phase 2, single-arm, open label trial (ANCHOR) into context of the current treatment landscape excluding investigational therapies. Additionally, the NIS output may be used to support future health technology assessment submissions and publications.

Detailed description: This retrospective, multi-center longitudinal study on BRAFV600E mutant mCRC patients will be conducted in Europe to characterize the first-line treatment patterns. All BRAFV600E mutant patients having initiated a first-line treatment for mCRC between January 1st, 2016 and December 31st, 2018 (both days inclusive) with drugs registered for mCRC in respective country will be eligible to participate. The study will not provide or recommend any treatment or procedure; all decisions regarding treatment are made at the sole discretion of the treating physician in accordance with their usual practices and all eligible patients will be considered for enrollment.

The target countries for patient enrollment will include Germany, France, Italy, United Kingdom, Spain, Belgium, Austria and the Netherlands. Approximately 300 adult patients (≥18 years) from a mix of academic and non-academic sites (up to 65 sites) will be enrolled.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of histologically or cytologically confirmed CRC that is metastatic and unresectable
* Presence of BRAFV600E mutation in tumor tissue, as confirmed by a local assay
* Initiated first-line treatment with drugs registered for mCRC in the respective country at the time of treatment between January 1st, 2016 and December 31st, 2018 (both days inclusive)
* Provision of informed consent or non-opposition to the patient (or next of kin, if applicable) for the use of data, according to local regulations

Exclusion Criteria:

Patients will be excluded from the study if they fulfil any of the following criteria:

* Patients with another concomitant cancer at the time of diagnosis\*
* Patients participating in interventional trials on investigational drugs at the time of initiation of first-line treatment

  * Except for non-metastatic non-melanoma skin cancers, or in situ or benign neoplasms; a cancer will be considered concomitant if it occurs within 5 years of mCRC diagnosis.

Primary outcomes:
measure: Treatment Patterns for First-line Systemic Anticancer Therapy (SACT) in BRAFV600E Mutant mCRC Patients
timeFrame: time from treatment initiation (for mCRC) up to 31 December 2020
measure: Duration of Treatment for First-line Systemic Anticancer Therapy (SACT) in BRAFV600E Mutant mCRC Patients
timeFrame: time from treatment initiation (for mCRC) up to 31 December 2020
measure: Switch in mCRC First-line Systemic Anticancer Therapy (SACT) Treatment in BRAFV600E Mutant mCRC Patients
timeFrame: time from treatment initiation (for mCRC) up to 31 December 2020
Secondary outcomes:
measure: Demographic and Clinical Characteristics
timeFrame: from the date of the start of first-line treatment for mCRC up to 31 December 2020
measure: Progression-free Survival (PFS)
timeFrame: from the date of the start of first-line treatment for mCRC up to 31 December 2020
measure: Overall Survival (OS)
timeFrame: from the date of the start of first-line treatment for mCRC up to 31 December 2020
measure: Overall Response Rate (ORR)
timeFrame: from the date of the start of first-line treatment for mCRC until the end of first-line treatment up to 31 December 2020
measure: Time to Treatment Cessation
timeFrame: from the date of the start of first-line treatment for mCRC until the documented disease progression up to 31 December 2020
measure: Description of BRAF Mutation Testing Procedure in Regards With the First-line Treatment in BRAFV600E Mutant mCRC Patients
timeFrame: from the date of the start of first-line treatment for mCRC until the end of first-line treatment up to 31 December 2020
measure: Description of BRAF Mutation Testing Timing in Regards With the First-line Treatment in BRAFV600E Mutant mCRC Patients
timeFrame: from the date of the start of first-line treatment for mCRC until the end of first-line treatment up to 31 December 2020
Other outcomes:
measure: Reasons for Alteration or Discontinuation of First-line Treatment in BRAFV600E Mutant mCRC Patients
timeFrame: from the date of the start of first-line treatment for mCRC until the end of first-line treatment up to 31 December 2020
measure: Other Types of SACT Treatment Line Regiments After First-line Treatment for mCRC in BRAFV600E Mutant mCRC Patients
timeFrame: from the date of the start of first-line treatment for mCRC until the end of first-line treatment up to 31 December 2020
Locations:
city: St. Veit/Glan
country: Austria
facility: Barmherzige Brüder Krankenhaus St. Veit/Glan.
city: Vienna
country: Austria
facility: Medizinische Universität Wien
city: Bonheiden
country: Belgium
facility: Imelda VZW
city: Brasschaat
country: Belgium
facility: AZ Klina
city: Leuven
country: Belgium
facility: UZ Leuven
city: Liège
country: Belgium
facility: CHC MontLégia
city: Besançon
country: France
facility: CHRU de Besançon
city: Creil
country: France
facility: GHPSO (Groupe Hospitalier Sud de l'Oise)
city: La Tronche
country: France
facility: CHU Grenoble Alpes
city: Levallois-Perret
country: France
facility: Hôpital Franco-Britannique
city: Lille
country: France
facility: Centre Oscar Lambert
city: Montpellier
country: France
facility: ICM Val d'Aurelle
city: Poitiers
country: France
facility: CHU de Poitiers
city: Villejuif
country: France
facility: Gustave Roussy
city: Aschaffenburg
country: Germany
facility: Klinikum Aschaffenburg Medical Klinik IV
city: Dresden
country: Germany
facility: Studienzentrale Gokos
city: Essen
country: Germany
facility: Universitätsklinikum Essen
city: Hamburg
country: Germany
facility: Facharztzentrum Eppendorf
city: Hamburg
country: Germany
facility: Oncoresearch Lerchenfeld
city: Leipzig
country: Germany
facility: MVZ Mitte Leipzig
city: Velbert
country: Germany
facility: MZ Onkologie Velbert/Ratingen/Mettmann
city: Ancona
country: Italy
facility: Clinica Oncologica Ospedali Riuniti di Ancona
city: Latina
country: Italy
facility: Santa Maria Goretti Hospital
city: Napoli
country: Italy
facility: Instituto Nazionale Tumori, IRCCS, Fondazione G. Pascale
city: Pisa
country: Italy
facility: Azienda Ospedaliero-Universitaria Pisana
city: Reggio Emilia
country: Italy
facility: AUSL-IRCCS of Reggio Emilia-Clinical Cancer Center
city: Saronno
country: Italy
facility: Asst Valle Olona
city: Barcelona
country: Spain
facility: Hospital del Mar
city: Madrid
country: Spain
facility: La Paz University Hospital
city: Valence
country: Spain
facility: Hospital General Universitario de Valencia
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham NHS Foundation Trust
city: Harrogate
country: United Kingdom
facility: Harrogate & District NHS Foundation Trust
city: London
country: United Kingdom
facility: Imperial College Healthcare NHS Trust
city: London
country: United Kingdom
facility: University College London Hospitals NHS Foundation Trust
Study officials:
name: Bernard Asselain, MD, PhD
role: STUDY_CHAIR
name: Dirk Arnold, MD, PhD
role: STUDY_CHAIR
name: Erika Martinelli, MD, PhD
role: STUDY_CHAIR
References: