PLATON - Platform for Analyzing Targetable Tumor Mutations (pilot-study) (PLATON)

PLATON - Platform for Analyzing Targetable Tumor Mutations (pilot-study) a Multicenter, Prospective, Cohort Study to Assess the Genomic Profiles and Associated Therapy Decision in Gastrointestinal Cancer

PLATON (Platform for Analyzing Targetable Mutations) is a prospective, multicentre, observational cohort study with biobanking. In a first approach PLATON's pilot-study assesses 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. The Study Protocol does not define any further medical intervention or evaluate the efficacy or safety of the treatment decision made by the investigator. Another important objective of PLATON's pilot project is to evaluate whether and how many patients are treated based on their genomic profiles.

Study Overview

• Status: Completed
• Conditions: Stomach Cancer; Pancreatic Cancer; Cholangiocarcinoma; Gallbladder Cancer; Hepatocellular Cancer; Oesophageal Cancer
• Intervention / Treatment: Diagnostic test: FoundationOne®CDx and FoundationOne®Liquid

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

• Study Type: Interventional
• Enrollment (Actual): 400
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Germany
  • Bad Saarow, Germany
    • HELIOS Klinikum Bad Saarow
  • Bayreuth, Germany, 95445
    • Klinikum Bayreuth
  • Berlin, Germany
    • Charité - Universitätsmedizin Berlin
  • Berlin, Germany, 13585
    • Vivantes Klinikum Spandau
  • Berlin, Germany
    • Evangelisches Waldkrankenhaus Spandau
  • Berlin, Germany
    • MVZ Oskar-Helene-Heim Berlin
  • Bochum, Germany
    • Augusta-Kranken-Anstalt Bochum
  • Bochum, Germany
    • Bochum Uni
  • Chemnitz, Germany
    • Klinikum Chemnitz
  • Darmstadt, Germany
    • Klinikum Darmstadt
  • Dortmund, Germany
    • GEFOS - Gesellschaft für onkologische Studien Dortmund
  • Dresden, Germany
    • Onkozentrum Dresden
  • Frankfurt, Germany, 65929
    • Klinikum Frankfurt Höchst
  • Fulda, Germany, 36043
    • Klinikum Fulda
  • Goslar, Germany
    • MVZ Onkologische Kooperation Harz
  • Halle, Germany
    • Universitätsklinikum Halle (Saale)
  • Hamburg, Germany, 22457
    • Hamburg Onkologisch-Hämatologische Schwerpunktpraxis
  • Hamm, Germany, 59063
    • Evangelisches Krankenhaus Hamm
  • Herne, Germany
    • St. Anna Hospital Herne
  • Lahr, Germany
    • Ortenau Klinikum Lahr-Ettenheim
  • Landshut, Germany
    • ÜBAG - MVZ Dr. Vehling-Kaiser GmbH
  • Langen, Germany
    • Langen, Gemeinschaftspraxis für Hämatologie und Onkologie
  • Leer, Germany
    • Studienzentrum UnterEms
  • Lemgo, Germany
    • Klinikum Lippe
  • Ludwigsburg, Germany
    • Klinikum Ludwigsburg
  • Marburg, Germany, 35043
    • Universitätsklinikum Gießen und Marburg GmbH
  • München, Germany
    • Klinik München-Bogenhausen
  • Münster, Germany
    • Münster, Gemeinschaftspraxis für Hämatologie und Onkologie
  • Osnabrück, Germany, 49074
    • Niels-Stensen-Kliniken Osnabrück
  • Ostfildern, Germany
    • Medius Klinik Osterfildern-Ruit
  • Regensburg, Germany
    • Krankenhaus Barmherzige Brüder
  • Rheine, Germany
    • Klinikum Rheine, Mathias-Spital Rheine
  • Rosenheim, Germany, 83022
    • RoMed Klinikum Rosenheim
  • Rostock, Germany, 18059
    • Klinikum Südstadt Rostock
  • Saarbrücken, Germany
    • CaritasKlinikum Saarbrücken
  • Singen, Germany
    • Onkologie Bodensee
  • Wiesbaden, Germany, 65199
    • Helios Dr. Horst Schmidt Kliniken Wiesbaden GmbH
  • Witten, Germany
    • Marien Hospital Witten
  • Wolfsburg, Germany
    • Klinikum Wolfsburg
  • Wolfsburg, Germany
    • Onkologisches Zentrum Wolfsburg-Helmstedt MVZ GmbH
  • Hessen
    • Frankfurt, Hessen, Germany, 60488
      • KHNW Frankfurt
  • Niedersachsen
    • Hannover, Niedersachsen, Germany, 30625
      • Hannover Medical School
  • Rheinland-Pfalz
    • Speyer, Rheinland-Pfalz, Germany, D-67346
      • Onkologische Schwerpunktpraxis Speyer
  • Sachsen
    • Ried, Sachsen, Germany, D-01589
      • ELBLANDKLINIKUM Riesa
  • Schleswig-Holstein
    • Neumünster, Schleswig-Holstein, Germany, 24534
      • Friedrich-Ebert-Krankenhaus Neumünster
  • Thüringen
    • Borna, Thüringen, Germany, D-04552
      • Sana Kliniken Leipziger Land

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Hepatocellular Cancer; molecular profiling - hepatocellular cancer (HCC)	Diagnostic test: 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.
Other: Cholangiocarcinoma; molecular profiling - intra- and extrahepatic cholangiocellular carcinoma (CCA)	Diagnostic test: 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.
Other: Gallbladder Cancer; molecular profiling - gallbladder carcinoma (GBCA)	Diagnostic test: 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.
Other: Pancreatic Cancer; molecular profiling - pancreatic cancer (PanCa)	Diagnostic test: 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.
Other: Oesophageal Cancer + Stomach Cancer; molecular profiling - esophagogastric cancer (EC/GC)	Diagnostic test: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Distribution of mutations in patients with HCC, intra- and extrahepatic CCA, GBCA, PDAC and gastric cancer	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.	up to 4 weeks after biospecimen provision

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Heterogeneity of targetable alterations in paraffin embedded specimen vs. cfDNA	Number of differences (heterogeneity) in targetable alterations in paraffin specimen vs. cfDNA	up to 4 weeks after biospecimen provision
Relative frequency of targetable mutations (incl. TMB and MSI status) per disease group	Relative frequency of targetable mutations (incl. TMB and MSI status) per disease group	up to 4 weeks after biospecimen provision
Number of patients receiving therapies in accordance to their genomic profiles	Number of patients receiving therapies in accordance to their genomic profiles	up to 4 weeks after biospecimen provision

Collaborators and Investigators

• Sponsor: Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest
• Collaborators: Roche Pharma AG
• Investigators: Study Director: Salah-Eddin Al-Batran, Prof., Institut für Klinische Krebsforschung IKF GmbH; Principal Investigator: Arndt Vogel, Prof., Hannover Medical School

Publications and helpful links

Helpful Links

• The PLATON Network

Study record dates

Study Major Dates

• Study Start (Actual): October 28, 2020
• Primary Completion (Actual): August 30, 2024
• Study Completion (Actual): December 31, 2024

Study Registration Dates

• First Submitted: July 10, 2020
• First Submitted That Met QC Criteria: July 21, 2020
• First Posted (Actual): July 23, 2020

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 17, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: Gastric cancer; Pancreatic Cancer; Cholangiocarcinoma; Oesophageal Cancer; Stomach Cancer; Gallbladder Cancer; Hepatocellular Cancer; Biobanking; Individualized cancer therapy; Targetable mutations; Foundation Medicine assays; Platform-Design
• Additional Relevant MeSH Terms: Endocrine System Diseases; Neoplasms by Site; Neoplasms; Neoplasms by Histologic Type; Gastrointestinal Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Stomach Diseases; Endocrine Gland Neoplasms; Pancreatic Diseases; Biliary Tract Diseases; Liver Diseases; Neoplasms, Glandular and Epithelial; Adenocarcinoma; Carcinoma; Gallbladder Diseases; Biliary Tract Neoplasms; Stomach Neoplasms; Carcinoma, Hepatocellular; Pancreatic Neoplasms; Liver Neoplasms; Cholangiocarcinoma; Gallbladder Neoplasms
• Other Study ID Numbers: PLATON pilot-study; AIO-HEP/STO-0219/ass (Other Identifier: AIO Arbeitsgemeinschaft Internistische Onkologie)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Depending on particpant informed concent individual-participant data (IPD) will only be shared in research-cooperations according the terms of PLATON's Use and Access Regulation. Scientific projects have to be approved by PLATON'S Scientific Steering Committee.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No