A Retrospective Non Interventional Study on First Line Treatment for Patients With BRAFV600E Mutant Metastatic Colorectal Cancer (mCRC)

NCT04317599 | Completed Results DMC FDA Drug

• 1 other identifier: NIS-PF0-2020-3141
• Study Type: observational
• Target: 274
• Locations: 7 countries
• Sites: 34

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.

Conditions

BRAF V600E Mutation Positive; Metastatic Colorectal Cancer

Keywords

first line; retrospective study; mCRC; BRAF V600E

Outcome Measures

Primary Outcomes (3)

• Treatment Patterns for First-line Systemic Anticancer Therapy (SACT) in BRAFV600E Mutant mCRC Patients

  First-line SACT treatment patterns in BRAFV600E mutant mCRC patients described by agent or combination of agents received

  time from treatment initiation (for mCRC) up to 31 December 2020

• Duration of Treatment for First-line Systemic Anticancer Therapy (SACT) in BRAFV600E Mutant mCRC Patients

  First-line SACT treatment patterns in BRAFV600E mutant mCRC patients described by Duration of Treatment

  time from treatment initiation (for mCRC) up to 31 December 2020

• Switch in mCRC First-line Systemic Anticancer Therapy (SACT) Treatment in BRAFV600E Mutant mCRC Patients

  Switch in mCRC first-line SACT treatment in BRAFV600E mutant mCRC patients.

  time from treatment initiation (for mCRC) up to 31 December 2020

Secondary Outcomes (7)

• Demographic and Clinical Characteristics

  from the date of the start of first-line treatment for mCRC up to 31 December 2020

• Progression-free Survival (PFS)

  from the date of the start of first-line treatment for mCRC up to 31 December 2020

• Overall Survival (OS)

  from the date of the start of first-line treatment for mCRC up to 31 December 2020

• Overall Response Rate (ORR)

  from the date of the start of first-line treatment for mCRC until the end of first-line treatment up to 31 December 2020

• Time to Treatment Cessation

  from the date of the start of first-line treatment for mCRC until the documented disease progression up to 31 December 2020

Other Outcomes (2)

• Reasons for Alteration or Discontinuation of First-line Treatment in BRAFV600E Mutant mCRC Patients

  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 Types of SACT Treatment Line Regiments After First-line Treatment for mCRC in BRAFV600E Mutant mCRC Patients

  from the date of the start of first-line treatment for mCRC until the end of first-line treatment up to 31 December 2020

Study Arms (1)

Non interventional

All BRAFV600E mutant patients having initiated a first-line treatment for mCRC between 01 January, 2016 and 31 December, 2018 (both days inclusive) with drugs registered for mCRC in respective country

Other: Non interventional

Interventions

Non interventional OTHER

All BRAFV600E mutant patients having initiated a first-line treatment for mCRC between 01 January, 2016 and 31 December, 2018 (both days inclusive) with drugs registered for mCRC in respective country

Non interventional

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Pierre Fabre Medicament: lead
• Clinact: collaborator

Study Sites (34)

Barmherzige Brüder Krankenhaus St. Veit/Glan.

Saint Veit/Glan, Austria

Location

Medizinische Universität Wien

Vienna, Austria

Location

Imelda VZW

Bonheiden, Belgium

Location

AZ Klina

Brasschaat, Belgium

Location

UZ Leuven

Leuven, Belgium

Location

CHC MontLégia

Liège, Belgium

Location

CHRU de Besançon

Besançon, France

Location

GHPSO (Groupe Hospitalier Sud de l'Oise)

Creil, France

Location

CHU Grenoble Alpes

La Tronche, France

Location

Hôpital Franco-Britannique

Levallois-Perret, France

Location

Centre Oscar Lambert

Lille, France

Location

ICM Val d'Aurelle

Montpellier, France

Location

CHU de Poitiers

Poitiers, France

Location

Gustave Roussy

Villejuif, France

Location

Klinikum Aschaffenburg Medical Klinik IV

Aschaffenburg, Germany

Location

Studienzentrale Gokos

Dresden, Germany

Location

Universitätsklinikum Essen

Essen, Germany

Location

Facharztzentrum Eppendorf

Hamburg, Germany

Location

Oncoresearch Lerchenfeld

Hamburg, Germany

Location

MVZ Mitte Leipzig

Leipzig, Germany

Location

MZ Onkologie Velbert/Ratingen/Mettmann

Velbert, Germany

Location

Clinica Oncologica Ospedali Riuniti di Ancona

Ancona, Italy

Location

Santa Maria Goretti Hospital

Latina, Italy

Location

Instituto Nazionale Tumori, IRCCS, Fondazione G. Pascale

Naples, Italy

Location

Azienda Ospedaliero-Universitaria Pisana

Pisa, Italy

Location

AUSL-IRCCS of Reggio Emilia-Clinical Cancer Center

Reggio Emilia, Italy

Location

Asst Valle Olona

Saronno, Italy

Location

Hospital del Mar

Barcelona, Spain

Location

La Paz University Hospital

Madrid, Spain

Location

Hospital General Universitario de Valencia

Valencia, Spain

Location

University Hospitals Birmingham NHS Foundation Trust

Birmingham, United Kingdom

Location

Harrogate & District NHS Foundation Trust

Harrogate, United Kingdom

Location

Imperial College Healthcare NHS Trust

London, United Kingdom

Location

University College London Hospitals NHS Foundation Trust

London, United Kingdom

Location

MeSH Terms

Conditions

Colorectal Neoplasms

Condition Hierarchy (Ancestors)

Intestinal Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Colonic Diseases; Intestinal Diseases; Rectal Diseases

Results Point of Contact

• Title: Project leader
• Organization: Pierre Fabre

farida.beghdad@pierre-fabre.com

+33(0)1 49 10 81 84

Study Officials

• Bernard Asselain, MD, PhD

  STUDY CHAIR

• Dirk Arnold, MD, PhD

  STUDY CHAIR

• Erika Martinelli, MD, PhD

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• 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: March 18, 2020
• First Posted: March 23, 2020
• Study Start: April 12, 2020
• Primary Completion: March 4, 2021
• Study Completion: May 12, 2021
• Last Updated: January 2, 2026
• Results First Posted: July 15, 2024

Record last verified: 2025-12

Locations

AU (2); IT (6); BE (4); ES (3); FR (8); DE (7); GB (4)