Abstract OT1-11-02: PRAEGNANT - Real world evidence, translational research, big and smart data: A prospective academic translational research network for the optimization of the oncological health care quality in the adjuvant and advanced/ metastatic setting (NCT02338167)

Background During the last decades the treatment of advanced breast cancer (ABC) patients has improved due to a variety of new treatment strategies. Nevertheless, many ABC patients are faced with limited prognosis, and their treatment remains a challenge. New targeted therapies complement the well-established treatment options for ABC (CDK4/6 inhibition, anti-endocrine, chemotherapy, antibody based and bone related therapies), leading to improved treatment regimens tailored to the needs of special patient sub-populations. SPECIFIC AIMS/TRIAL DESIGN The PRAEGNANT network study is conducted as an academic, prospective registry and diagnostic translational study, accompanied by biomaterial collection. The pilot phase in more than 60 centers aims at including 3500 ABC patients. The primary objective is to discover biomarkers, which predict progression free survival (PFS). Secondary objectives include overall survival (OS), breast cancer specific survival, objective response rate, patient reported outcomes (PRO), description of therapies used in the metastatic setting, therapy adherence, health economics for patients with ABC, incidence of (serious) adverse events and big data/ machine learning algorithms. The exploratory objectives comprise correlations of gene alterations and their influence on OS, PFS, side effects and PRO. Exploratory biomarkers are assessed at baseline and at every change of therapy. These biomarkers include gene expression profiling of the primary tumor and corresponding metastasis, somatic mutations (measured in the tumor and in circulating tumor DNA), germline genetic variations, epigenetic changes and miRNA variations. Furthermore, plasma and serum markers are assessed. If actionable molecular alterations are detected patients are informed and recruited into suitable studies if available. ELIGIBILITY CRITERIA: Any adult patient (>18 years) with the diagnosis of ABC and who is willing and able to sign the informed consent can be enrolled. STATISTICAL METHODS/TARGET ACCRUAL: The PRAEGNANT study as a prospective real world registry and diagnostic translational study aims to identify biomarkers in ABC patients, which may predict PFS. Target accrual for the pilot phase is 3500 patients. Each patient will be documented for up to 36 months with an estimated median PFS for all patients of 11 months across all treatment lines. Citation Format: Müller V, Huober J, Volz B, Overkamp F, Kolberg H-C, Hadji P, Haeberle L, Ettl J, Hartkopf AD, Lux MP, Lüftner DI, Wallwiener M, Taran F-A, Beckmann MW, Belleville E, Wimberger P, Hielscher C, Geberth M, Lermann J, Abenhardt A, Kurbacher C, Wuerstlein R, Thomssen C, Untch M, Janni W, Fehm TN, Brucker SY, Wallwiener D, Schneeweiss A, Fasching PA, Tesch H. PRAEGNANT - Real world evidence, translational research, big and smart data: A prospective academic translational research network for the optimization of the oncological health care quality in the adjuvant and advanced/ metastatic setting (NCT02338167) [abstract]. In: Proceedings of the 2018 San Antonio Breast Cancer Symposium; 2018 Dec 4-8; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2019;79(4 Suppl):Abstract nr OT1-11-02.