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

Sponsor

Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest (Other)

Overall Status

Recruiting

CT.gov ID

NCT04484636

Collaborator

Roche Pharma AG (Industry)

200

Enrollment

Locations

Arms

Anticipated Duration (Months)

6.7

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Hepatocellular Cancer Cholangiocarcinoma Gallbladder Cancer Pancreatic Cancer Oesophageal Cancer Stomach Cancer	Diagnostic Test: FoundationOne®CDx and FoundationOne®Liquid	N/A

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 Design

Study Type:

Interventional

Anticipated Enrollment :

200 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Intervention Model Description:

Experimental StudyExperimental Study

Masking:

None (Open Label)

Primary Purpose:

Other

Official Title:

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

Actual Study Start Date :

Oct 28, 2020

Anticipated Primary Completion Date :

Dec 31, 2022

Anticipated Study Completion Date :

Jun 30, 2023

Arms and Interventions

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.

Outcome Measures

Primary Outcome Measures

Distribution of mutations in patients with HCC, intra- and extrahepatic CCA, GBCA, PDAC and gastric cancer [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 Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 (adjuvant or neoadjuvant therapy is allowed if completed 6 months prior to enrolment) 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	KHNW Frankfurt	Frankfurt	Hessen	Germany	60488
2	Hannover Medical School	Hannover	Niedersachsen	Germany	30625
3	Friedrich-Ebert-Krankenhaus Neumünster	Neumünster	Schleswig-Holstein	Germany	24534
4	HELIOS Klinikum Bad Saarow	Bad Saarow		Germany
5	Evangelisches Waldkrankenhaus Spandau	Berlin		Germany
6	MVZ Oskar-Helene-Heim Berlin	Berlin		Germany
7	Augusta-Kranken-Anstalt Bochum	Bochum		Germany
8	Bochum Uni	Bochum		Germany
9	Klinikum Chemnitz	Chemnitz		Germany
10	GEFOS - Gesellschaft für onkologische Studien Dortmund	Dortmund		Germany
11	Onkozentrum Dresden	Dresden		Germany
12	MVZ Onkologische Kooperation Harz	Goslar		Germany
13	Universitätsklinikum Halle (Saale)	Halle		Germany
14	St. Anna Hospital Herne	Herne		Germany
15	Ortenau Klinikum Lahr-Ettenheim	Lahr		Germany
16	ÜBAG - MVZ Dr. Vehling-Kaiser GmbH	Landshut		Germany
17	Langen, Gemeinschaftspraxis für Hämatologie und Onkologie	Langen		Germany
18	Studienzentrum UnterEms	Leer		Germany
19	Klinikum Lippe	Lemgo		Germany
20	Klinikum Ludwigsburg	Ludwigsburg		Germany
21	Klinik München-Bogenhausen	München		Germany
22	Münster, Gemeinschaftspraxis für Hämatologie und Onkologie	Münster		Germany
23	Medius Klinik Osterfildern-Ruit	Ostfildern		Germany
24	Krankenhaus Barmherzige Brüder	Regensburg		Germany
25	Klinikum Rheine, Mathias-Spital Rheine	Rheine		Germany
26	CaritasKlinikum Saarbrücken	Saarbrücken		Germany
27	Onkologie Bodensee	Singen		Germany
28	Marien Hospital Witten	Witten		Germany
29	Klinikum Wolfsburg	Wolfsburg		Germany
30	Onkologisches Zentrum Wolfsburg-Helmstedt MVZ GmbH	Wolfsburg		Germany

Sponsors and Collaborators

Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest
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