Detailed Description:
PLATON (Platform for Analyzing Targetable Mutations) is designed to improve personalized
therapy for patients in different cancer entities, such as in hepatocellular cancer (HCC),
intra- and extrahepatic cholangiocellular carcinoma (CCA), gallbladder carcinoma (GBCA),
pancreatic cancer (PanCa) and esophagogastric cancer (EC/GC), and elevate the treatment
guidance within its framework. The key to understand the mechanisms in initiation,
progression and response to treatment of cancer is the data integration of genetic mutational
signatures with medical and physiological data of diseased cohorts.
PLATON is a prospective, multicentre, observational cohort study with biobanking and does not
define any medical intervention or evaluate the efficacy or safety of the treatment decision
made by the investigator.In a first approach PLATON's pilot-study assess genomic profiling in
gastrointestinal cancer therapy and the frequencies of targetable mutations including Tumor
Mutational Burden (TMB) and Microsatellite Instability Status (MSI), performing
Next-generation deep sequencing (NGS) using the Foundation Medicine assays on tumor specimen
and EDTA-whole blood samples. Another important objective of PLATON's pilot project is to
evaluate whether and how many patients are treated based on their genomic profiles.
The pilot-study starts with the national-wide enrolment of 200 participants of both sexes and
ages over 18 at 40 german study sites. The long-term vision is to enable cancer patients to
receive the best available, scientifically founded, biomarker-based care, tailored to his or
her individual needs
Inclusion Criteria:
- Histologically confirmed diagnosis of hepatocellular carcinoma or intra-/extrahepatic
cholangiocarcinoma or gallbladder carcinoma or pancreatic ductal adenocarcinoma or
esophagogastric adenocarcinoma in the advanced setting and no local curative therapy
available.
- Standard first line therapy is planned, or patient is currently receiving first line
therapy (started within the last 2 months before enrolment)
- ECOG 0-2
- Life expectancy ≥ 6 months
Exclusion Criteria:
- Not able to understand all implications of study participation
- No written informed consent
- age < 18 years
Primary outcome:
1. Distribution of mutations in patients with HCC, intra- and extrahepatic CCA, GBCA, PDAC and gastric cancer (Time Frame - up to 4 weeks after biospecimen provision):
Relative frequency of targetable mutations (incl. TMB and MSI status) computed as the number of patients who harbors at least one mutation divided by the number of total patients in the pooled patient population.
Secondary outcome:
1. Heterogeneity of targetable alterations in paraffin embedded specimen vs. cfDNA (Time Frame - up to 4 weeks after biospecimen provision):
Number of differences (heterogeneity) in targetable alterations in paraffin specimen vs. cfDNA
2. Relative frequency of targetable mutations (incl. TMB and MSI status) per disease group (Time Frame - up to 4 weeks after biospecimen provision):
Relative frequency of targetable mutations (incl. TMB and MSI status) per disease group
3. Number of patients receiving therapies in accordance to their genomic profiles (Time Frame - up to 4 weeks after biospecimen provision):
Number of patients receiving therapies in accordance to their genomic profiles
- Other: Hepatocellular Cancer
molecular profiling - hepatocellular cancer (HCC) - Other: Cholangiocarcinoma
molecular profiling - intra- and extrahepatic cholangiocellular carcinoma (CCA) - Other: Gallbladder Cancer
molecular profiling - gallbladder carcinoma (GBCA) - Other: Pancreatic Cancer
molecular profiling - pancreatic cancer (PanCa) - Other: Oesophageal Cancer + Stomach Cancer
molecular profiling - esophagogastric cancer (EC/GC)
- FoundationOne®CDx and FoundationOne®Liquid:
FoundationOne CDx is a FDA-approved broad companion diagnostic (CDx) that is clinically and analytically validated for solid tumors. The test is designed to provide physicians with clinically actionable information - both to consider appropriate therapies for patients and understand results with evidence of resistance - based on the individual genomic profile of each patient's cancer. Every test result includes microsatellite instability (MSI) and tumor mutational burden (TMB) to help inform immunotherapy decisions. FoundationOne®Liquid is a liquid biopsy test for solid tumors that analyzes circulating tumor DNA (ctDNA) in blood.
Quelle: ClinicalTrials.gov
