Abstract 1666
Background
AF is a rare non-metastasizing connective tissue tumor (< 300 cases/year in France), associated with high risk of local relapse, functional impairment and pain. AF is most frequently (about 85%) sporadic and then associated with a somatic mutation of the CTNNB1 gene. In about 15% of cases, AF is associated with heredity condition, as complication of familial adenomatous polyposis FAP (with germinal mutation of APC gene). The natural course of AF is unpredictable. As the consequence the decision making for starting curative intent treatment is difficult. Therapeutic options are: wait-and-see policy, surgery (sometimes mutilating), radiotherapy or systemic treatment (non-steroidal anti-inflammatory drugs, hormonal therapy, chemotherapy, sorafenib). Regarding these uncertainties, physicians can hardly answer to patient questions.
Trial design
ALTITUDES is a national multicenter cohort of 600 patients who will be included in 39 centers (NCT02867033). The primary outcome is number of cases diagnosed in reference centers. Secondary outcomes are rate of cases related to FAP, rate of cases associated with CTNNB1 mutations, description of natural history, description of management, impact in terms of Anxiety and Depression, in QoL. Main eligibility criteria are a diagnosis later than 01/01/2016, with central pathological review. A translational part is attached to this cohort. On the one hand, the purpose is detection of FA specific CTNNB1 mutations using a targeted strategy Digital-droplet PCR (ddPCR) on cell-free DNA (cfDNA) extracted from blood samples and to correlate total plasmatic cfDNA concentration with natural history. On the other hand, We hypothesize that APC and CTNNB1 mutations are mutually exclusive and that patients with a CTNNB1 mutation do not have an increased risk of developing polyposis and/or colon cancer and therefore should not benefit from screening colonoscopy. In this study, colonoscopy is mandatory and a search for somatic mutations of CTNNB1 and APC. Since opening of enrolment (03/2016), 360 patients were included. Along these patients, 316 were included in the translational part. End of recruitment is planned 03/2022.
Clinical trial identification
NCT02867033.
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Institut Curie.
Disclosure
All authors have declared no conflicts of interest.
Resources from the same session
4615 - Proteomic Profiling Identifies Molecular Basis of Adverse Event to BPM31510 Exposure: Rationale for Comprehensive Molecular Pharmacodynamics (PD) in Phase 1 Clinical Trial Design
Presenter: Vivek Subbiah
Session: Poster Display session 1
Resources:
Abstract
5052 - Identification of first-in-class, naturally occurring LAG3 checkpoint inhibitor
Presenter: Gennady Bratslavsky
Session: Poster Display session 1
Resources:
Abstract
5336 - Are Epigenetic therapies modifying sensitivity to conventional chemotherapy?
Presenter: Alexandra Bizot
Session: Poster Display session 1
Resources:
Abstract
5739 - Oncogenic mutations at the dimer interface of EGFR lead to formation of covalent homo-dimers and allosteric activation of the kinase domain: A mechanism which alters the selectivity profile of oncogenic EGFR.
Presenter: Elizabeth Buck
Session: Poster Display session 1
Resources:
Abstract
5492 - Basic selective estrogen receptor degraders (B-SERDs) in combination with novel BET inhibitors in ER+ breast cancer
Presenter: Rui Xiong
Session: Poster Display session 1
Resources:
Abstract
5965 - EPI-7386 is a novel N-terminal domain androgen receptor inhibitor for the treatment of prostate cancer
Presenter: Ronan Le Moigne
Session: Poster Display session 1
Resources:
Abstract
3582 - AVID200 neutralizes TGF-beta1 and -beta3, the principal immunosuppressive TGF-beta isoforms overexpressed by tumors, and sensitizes tumors to immune checkpoint inhibitors.
Presenter: Tina Gruosso
Session: Poster Display session 1
Resources:
Abstract
1996 - High NAMPT expression and anti-tumor activity of NAMPT inhibitor in adult T-cell leukemia/lymphoma
Presenter: Tomohiro Kozako
Session: Poster Display session 1
Resources:
Abstract
4307 - TPX-0046 is a novel and potent RET/SRC inhibitor for RET-driven cancers
Presenter: Alexander Drilon
Session: Poster Display session 1
Resources:
Abstract
4869 - In Vivo Evaluation of Cisplatin-loaded PEG-PCL Block Copolymeric Nanoparticles for Anticancer Drug Delivery
Presenter: Yingtzu Yen
Session: Poster Display session 1
Resources:
Abstract