EvIDHence - Longitudinal retrospective CCA chart review study
Research type
Research Study
Full title
Longitudinal retrospective CCA chart review study
IRAS ID
319729
Contact name
John Bridgewater
Contact email
Sponsor organisation
Servier Affaires Medicales
Clinicaltrials.gov Identifier
CANC 53394, NIHR CRN
Duration of Study in the UK
0 years, 4 months, 29 days
Research summary
This is an non-interventional, descriptive, multicenter, international, longitudinal, retrospective, chart review study involving advanced and metastatic CCA patients.
Cholangiocarcinoma (CCA) is the most common biliary tract malignancy and the second most common hepatic cancers after hepatocellular carcinoma (HCC). CCA is categorized according to its anatomical location as intrahepatic (iCCA), perihilar (pCCA), or distal extrahepatic (dCCA).
In general, CCA is linked with several risk factors that cause chronic inflammation and cholestasis, resulting in a cycle of reactive cell proliferation, genetic and epigenetic mutations, and eventual cholangiocarcinogenesis. These cancers are generally without symptoms at early stages and usually diagnosed at a locally advanced stage or when the disease has already spread.
Until recently, there were no established second-line chemotherapy treatments following progression after first-line treatment nor any evidence to support the use of targeted therapies, and patients were encouraged to participate in clinical trials. Considering the extremely poor response of CCA to conventional anticancer therapies, several clinical trials are currently ongoing and new anticancer agents are being developed following advances in molecular profiling techniques and discovery of new driver mutations.
This non-interventional, retrospective chart review study aims to address these gaps and evaluate the care situation of CCA patients and the methods of treatment.
Lay summary of study results: This observational, non-interventional, non-comparative study conducted between July 2019 and December 2021 in Europe and the USA, examined patient profiles and treatments outcomes, in adults diagnosed with advanced/metastatic Cholangiocarcinoma before arrival of targeted therapies.
Due to the retrospective nature of the study, the reported adverse events were only adverse drug reactions, which were consistent with the label of the administered regimens. There were many limitations and confounding factors. A major limitation of this analysis was the limited number of patients enrolled, most of which were treated in the context of clinical trials. Moreover, the included patients were only those that survived 1L and were fit enough to initiate 2L. Therefore, due to limited number of patients, the study did not allow to establish the role of IDH1 mutation as a prognostic biomarker.
REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
22/EM/0230
Date of REC Opinion
31 Oct 2022
REC opinion
Further Information Favourable Opinion