EU Patient- cEntric clinicAl tRial pLatform

CORDIS bietet Links zu öffentlichen Ergebnissen und Veröffentlichungen von HORIZONT-Projekten.

Links zu Ergebnissen und Veröffentlichungen von RP7-Projekten sowie Links zu einigen Typen spezifischer Ergebnisse wie Datensätzen und Software werden dynamisch von OpenAIRE abgerufen.

Leistungen

List of Non-Clinical, Early Development Biomarkers and Strategy for Validation

Bringing together a comprehensive overview of the current state of scientific validation to guarantee safety and to predict the outcome of late stage phase 2 and 2 clinical studies

Operational Plan for the implementation of the NF IRP

The operational plan for a LNHS and phase 23 IRP trial in NF patients based on WP2

Roadmap for collaboration with other projects/networks

A roadmap of synergies with other projects/networks will be established, followed by implementation of the most relevant collaborations

IRP-Integrated Sustainability Business Plan adapted to NASH-PT

An IRPspecific exploitation and sustainability plan

IRP-Integrated Sustainability and Business plan adapted to TB

An IRPspecific exploitation and sustainability plan

EU-PEARL Sustainability Plan

Sustainability plan defining an operations ready concern including legal regulatory and ethically compliant operations and governance structure ready for exploitation The plan will be based on a Cost Benefit Assessment and Business Impact Assessment to develop a business model for adoption scale up and the creation of a sustainable entity It will include impacts on stakeholders and organisational change recommendations

LNHS/Readiness cohort Protocol for MDD platform trial

This deliverable is the LNHS protocol that will collect longitudinal data to help understand the clinical course of inadequately responding patients. A defined subset of the enrolled patients will serve as the readiness cohort of the future MDD platform trial.

TRD/PRD final report on Regulatory and Ethics Consultation

Report on meetings held with WP4 and Regulators along with expertise from the biopharmaceutical industry to discuss regulatory and ethical issues involved in MDD platform trial and LNHS with particular reference to TRDPRD populations and studies Formats may include formal EMA regulatory consultation with EMA Innovation Task Force ITF or informal workshops Compound ownersdevelopers may include industry academia or others

Operational Plan to implement TB IRP

Framework protocol will be developed to allow rapid implementation of the IRP trial with the development of a TB specific operating plan to oversee such trials across the networks of clinical sites

Final portfolio of policies that need to be adopted by any cluster of companies and sites IRPs as final versions of instruments created in tasks 11 and 13 informed by the project experiences in WPs 27

Digital Phenotyping Tool for TRD/ PRD review

This deliverable will be a general review of the top digital phenotyping tools currently in use in MDD clinical trials The review should contain indepth evaluation of the top 23 tools in the market in addition to DocuMental This review should compare and contract these tools with respect to consensus definition used for TRDPRD as well as other important features relevant for the conduct of clinical trials as opposed to a consumer product or a patient management product targeting physicians A publication may result from this report if company confidential information can be redacted

Report on challenges and clinical trial scenarios in NASH specific IRPs

A manuscript for submission to a peerreviewed journal describing the challenges in designing a NASH specific IRP including various approaches to conducting studies patient subphenotypes outcomes and endpoints and standard assessments such as liver biopsy and other biomarkers

Final Report on Clinical Operations Best Practices

Includes methods for the implementation of interim analyses randomisation patient allocation and blinding safety monitoring and pharmacovigilance data management governance sponsorship site management selection of third party vendors clinical supplies and manufacturing issues around introduction and removal of treatments protocol deviations trial longevity and intervention selection

TRD/PRD final master protocol for IRP

This report will develop protocol templates for POC Phase II and III for TRDPRD IRP adapting the master protocol template from WP2 The trial design report and simulations will be created for IRP including statistics and methods tools

Operational Plan to implement the NASH IRP

Framework protocol will be developed to allow rapid implementation of the IRP trial with the development of a NASHspecific operating plan to oversee such trials across the networks of clinical sites

Operational Plan to implement TRD/PRD IRP and LNHS

A framework operational plan will be developed to allow rapid implementation of a MDDspecific LNHS and platform trial using the networks of clinical sites being established in D48

Report on the Consensus Definition of Current Challenges in NF

This will define the scope of disease spectrum, patient definition, list and prioritise the main challenges in disease to be addressed by the IRP and the LNHS, define outcomes/endpoints, and how to identify key risk predictors to identify patients that can qualify for future trials and assess potential biomarkers.

Provisional Generic Master Protocol Template and Appendix for IRPs

Definition of the master protocol template structure including diseasespecific appendices statistical analysis plans and additional elements such as charts of data andor safety monitoring committees

NASH final Master Protocol for IRP

This report will develop protocol template for Phase 2 and considerations for Phase 3 for NASH IRP adapting the master protocol template from WP2 The trial design report and simulations will be created for IRP including statistics and methods tools

European Certification Programme for IRP Platforms

Organisational and business model for a scalable and sustainable certification programme initially within Europe for certifying IRP products and services operationalising the results in D16

TB final report on Regulatory and Ethics Consultation

Report on meetings held with ECs and Regulators along with expertise from the biopharmaceutical industry to discuss regulatory and ethical issues involved in IRP with particular reference to TB populations and studies

TB final Master Protocol for IRP

This report will develop protocol template for Phase 2a bc and considerations for Phase 3 for TB IRP adapting the master protocol template from WP2 The trial design report and simulations will be created for IRP including statistics and methods tools

Clinical Operations Best Practices Report

Includes a systematic literature review on integrated research platforms covering methodological research, ongoing and completed platform trials, relevant European and US regulatory guidance and other relevant documents to identify the current state of the art, methodological gaps and major operational challenges.

Protocol for a Longitudinal Natural History Study (LNHS) in NF

Continuing from D71 we will define the content and time intervals of evaluation within the LNHS including standardised tools to evaluate complications

NASH final Report on Regulatory and Ethics Consultation

Interim Report on TRD/PRD master protocol and regulatory requirements

It includes the results of the first TRDPRD master protocol synopsis It will also describe the first outcome of the consultation regarding the minimum regulatory requirements of a platform study design

Outcome of workshops delivered by EPF and Patient Representatives

This deliverable will inform on the outcomes of two workshops delivered to the consortium by EPF with input from patient representatives

Trial feasibility in federated hospital network

This deliverable will document the outcomes of the evaluation for the tools and scaffolding developed extended or integrated during the project

Functional requirements for a federated network of patient cohorts for clinical trial feasibility and recruitment

This deliverable will describe architecture and processes for establishing an IRP involving real world clinical data sources disease registries and other patientlevel data sources

Interim Report on NASH master protocol and regulatory requirements

It includes the results of the first NASH master protocol synopsis and corresponding operational plan for implementation It also describes the first outcome of the consultation regarding the minimum regulatory requirements of the NASH platform study design

Project Communication and Dissemination Strategy Report and Initial Toolset

Plan for communicating the project progress and its results. It will comprise dissemination objectives, target audiences, tools and planned activities

Interim Report on TB master protocol and regulatory requirements

It includes the results of the first TB master protocol synopsis and corresponding site readiness for implementation It also describes the first outcome of the consultation regarding the minimum regulatory requirements for the TB platform study design

Report on Consensus Disease Definition for TRD and PRD

A oneday Scientific Meeting on definitions for TRD and PRD will be organised followed by a written report An openaccess publication with disease definitions will be pursued subsequently

Generic process for an EHR based patient cohort for IRPs

This report will define the standards and processes for IRPs patient identification selection and recruitment including multisponsors Long Term Registries to support patient recruitments

Interim Report on NF master protocol and regulatory requirements

It includes the results of the first NF master protocol synopsis and corresponding operational plan for implementation It also describes the first outcome of the consultation regarding the minimum regulatory requirements of the NF platform study design

Report on stakeholders' event

This deliverable will contain a summary of the discussions and outcomes of the stakeholders event that WP3 will organize as a satellite session of WP2stakeholder event to share results and present methodologies developed for clinical trial feasibility

Capacity Building and Assessment Handbook for site readiness

Based on need assessment and requirements from platform short guide for site implementation focusing on the laboratory aspects human resources data management ethics integrity

Framework for Ensuring and Benchmarking Acceptable Data Quality Standards for Platform Trials

Organisational and business model for a scalable and sustainable data quality assessment service to assure future IRP studies and platforms that the data they reuse eg from EHR systems is of a satisfactory quality drawing on the results and experiences in WPs 27

Final Generic Master Protocol Template and Appendix for IRPs

See D23 Definition of the master protocol template structure including diseasespecific appendices statistical analysis plans and additional elements such as charts of data andor safety monitoring committees

NF Specific Report on Regulatory and Ethics Consultation

Report that identifies the NFspecific adaptations to the generic regulatory endorsement of WP2 eg age 18 years learning and behavioural disabilities patients with multiple complications offering themselves for multiple trials

Report on Terminology and Scenarios for Platform Trials

Includes a definition of common terminology for IRP and description of a number of disease and product development scenarios (including those of DSWPs) where a platform trial would be appropriate. These scenarios will be considered in the production of the generic master protocol template.

IRP-Integrated Sustainability and Business Plan Adapted to NF

An IRPspecific exploitation and sustainability plan will be created

Report on Methodological Tools for Intervention Selection

Includes tools and best practice advice for presentation assessment and decisionmaking of additional intervention to already running platform trial

Clinical Network of NF Sites Report

This will identify the required and available facilities of sites for trial and patient data and define minimal criteria for a viable network of sites for IRP and LNHS

Report on Regulatory Issues of IRPs

Includes requirements of regulatory authorities and ethics committees for approval of IRPs informed consent protocol amendments openingclosing of arms and issues in assessment of IRPs for regulatory approval of new medicinal products by regulatory authorities eg European National Authorities EMA and FDA and assessment by HTAs

NF Master Protocol for IRPs

This report will develop protocol template for Phase 2 and considerations for Phase 3 for NF IRP adapting the master protocol template from WP2 The trial design report and simulations will be created for IRP including statistics and methods tools

Patient Engagement Platform

An online portal and resources that support patientpublic awareness about IRP trials enable their engagement in design and participation in relevant studies

Clinical research network report to support future NASH IRPs

This report will document the identification of clinical sites qualified for execution of the IRP in NASH endemic countries (particularly focused on the ability to develop a readiness cohort). It will also evaluate overall site readiness and identify common gaps.

Veröffentlichungen

An Adaptive-Discard-Graph for online error control

Autoren: Fisher, L., Bofill Roig, M., & Brannath, W
Veröffentlicht in: Arxiv gov, Ausgabe N/A, 2023, Seite(n) --
Herausgeber: --
DOI: 10.48550/arxiv.2301.11711

Platform trials: the impact of shared controls on type one error and power

Autoren: Nguyen, Q., Hees, K., Hofner, B
Veröffentlicht in: Arxiv gov, Ausgabe N/A, 2023, Seite(n) --
Herausgeber: --
DOI: 10.48550/arxiv.2302.04713

Commentary: Two approaches to analyze platform trials incorporating non-concurrent controls with a common assumption

Autoren: Bofill Roig, M., König, F., Meyer, E., & Posch, M.
Veröffentlicht in: Clinical Trials, Ausgabe Bimonthly, 2022, Seite(n) Volume 19, Ausgabe 5, October 2022, Pages 502-503, ISSN 1740-7745
Herausgeber: Sage Publications
DOI: 10.1177/17407745221112016

The Online Closure Principle

Autoren: Fischer, L., Bofill Roig, M., & Brannath, W
Veröffentlicht in: Arxiv gov, Ausgabe N/A, 2022, Seite(n) --
Herausgeber: --
DOI: 10.48550/arxiv.2211.11400

Platform trials and the future of evaluating therapeutic behavioural interventions

Autoren: Stefan M. Gold, Marta Bofill Roig, J. Jaime Miranda, Carmine Pariante, Martin Posch & Christian Otte.
Veröffentlicht in: Nature Reviews Psychology, 2022, ISSN 2731-0574
Herausgeber: Springer Nature Limited
DOI: 10.1038/s44159-021-00012-0

CohortPlat: Simulation of cohort platform trials investigating combination therapies

Autoren: Meyer E., Mesenbrink P, Duner-Baldauf C, Glimm E., Li Y, König F.
Veröffentlicht in: Arxiv gov, Ausgabe N/A, 2022, Seite(n) 1-24
Herausgeber: ARXIV.GOV
DOI: 10.48550/arxiv.2202.02182

Adaptive clinical trial designs with blinded selection of binary composite endpoints and sample size reassessment

Autoren: Bofill Roig, M., Melis, G. G., Posch, M., & Koenig, F.
Veröffentlicht in: Biostatistics, Ausgabe Quarterly, 2022, ISSN 1465-4644
Herausgeber: Oxford University Press
DOI: 10.1093/biostatistics/kxac040

Systematic review of available software for multi-arm multi-stage and platform clinical trial design

Autoren: Elias Laurin Meyer, Peter Mesenbrink, Tobias Mielke, Tom Parke, Daniel Evans, Franz König
Veröffentlicht in: Trials, Ausgabe 22/1, 2021, ISSN 1745-6215
Herausgeber: BioMed Central
DOI: 10.1186/s13063-021-05130-x

Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and Cons

Autoren: Juan M. Pericàs, Frank Tacke, Quentin M. Anstee, Nicholas A. Di Prospero, Mette Skalshøj Kjær, Peter Mesenbrink, Franz Koenig, Joan Genescà, Vlad Ratziu
Veröffentlicht in: Journal of Hepatology, Ausgabe Monthly, 2022, Seite(n) Vol.78 442–447, ISSN 0168-8278
Herausgeber: Elsevier BV
DOI: 10.1016/j.jhep.2022.09.021

On model-based time trend adjustments in platform trials with non-concurrent controls

Autoren: Marta Bofill Roig, Pavla Krotka, Carl-Fredrik Burman, Ekkehard Glimm, Katharina Hees, Peter Jacko, Franz Koenig, Dominic Magirr, Peter Mesenbrink, Kert Viele, Martin Posch
Veröffentlicht in: BMC Medical Research Methodology, 2022, ISSN 1471-2288
Herausgeber: BioMed Central
DOI: 10.1186/s12874-022-01683-w

A Delphi-method-based consensus guideline for definition of treatment-resistant depression for clinical trials

Autoren: Luca Sforzini, Courtney Worrell, Melisa Kose, Ian M. Anderson, Bruno Aouizerate, Volker Arolt, Michael Bauer, Bernhard T. Baune, Pierre Blier, Anthony J. Cleare, Philip J. Cowen, Timothy G. Dinan, Andrea Fagiolini, I. Nicol Ferrier, Ulrich Hegerl, Andrew D. Krystal, Marion Leboyer, R. Hamish McAllister-Williams, Roger S. McIntyre, Andreas Meyer-Lindenberg, Andrew H. Miller, Charles B. Nemeroff, Cl
Veröffentlicht in: Molecular Psychiatry, 2021, ISSN 1476-5578
Herausgeber: Springer Nature Limited
DOI: 10.1038/s41380-021-01381-x

Decision rules for identifying combination therapies in open-entry, randomized controlled platform trials

Autoren: Elias Laurin Meyer,Peter Mesenbrink,Cornelia Dunger-Baldauf,Ekkehard Glimm,Yuhan Li,Franz König,EU-PEARL (EU Patient-cEntric clinicAl tRial pLatforms) Consortium
Veröffentlicht in: Pharmaceutical Statistics, 2022, ISSN 1539-1612
Herausgeber: John Wiley & Sons Ltd
DOI: 10.1002/pst.2194

Community engagement in tuberculosis research: the EU-Patient-cEntric clinicAl tRial pLatforms (EU-PEARL) experience

Autoren: Dressler S, Tàvora Dos Santos Filho E, Seidel S, Gonzalez Moreno J, Heinrich N, Sanchez-Montalva A, Cirillo D
Veröffentlicht in: International journal of infectious diseases : IJID : official publication of the International Society for Infectious Diseases,, Ausgabe Monthly, 2023, Seite(n) Volume 130 Suppl.1 S-20-24, ISSN 1201-9712
Herausgeber: Elsevier BV
DOI: 10.1016/j.ijid.2023.03.008

Estimands and Complex Innovative Designs

Autoren: Olivier Collignon,Anja Schiel,Carl-Fredrik Burman,Kaspar Rufibach,Martin Posch,Frank Bretz
Veröffentlicht in: Clinical Pharmacology & Therapeutics, 2022, ISSN 1532-6535
Herausgeber: John Wiley & Sons, Inc
DOI: 10.1002/cpt.2575

Identifying challenges in neurofibromatosis: a modified Delphi procedure

Autoren: Britt A. E. Dhaenens, Rosalie E. Ferner, Annette Bakker, Marco Nievo, D. Gareth Evans, Pierre Wolkenstein, Cornelia Potratz, Scott R. Plotkin, Guenter Heimann, Eric Legius & Rianne Oostenbrink
Veröffentlicht in: European Journal of Human Genetics, Ausgabe 12 issues per year, 2021, ISSN 1018-4813
Herausgeber: Natue Publishing Group
DOI: 10.1038/s41431-021-00892-z

Online error rate control for platform trials.

Autoren: Robertson, D. S., Wason, J. M., König, F., Posch, M., & Jaki, T.
Veröffentlicht in: Statistics in Medicine, Ausgabe 30/year, 2023, Seite(n) Volume 42, Ausgabe 14Jun 2023 Pages 2475–2495, ISSN 0277-6715
Herausgeber: John Wiley & Sons Inc.
DOI: 10.1002/sim.9733

Review Article: The need for more efficient and patient-oriented drug development pathways in NASH-setting the scene for platform trials

Autoren: Juan M. Pericàs, Nicholas A. Di Prospero, Quentin M. Anstee, Peter Mesenbrinck, Mette S. Kjær, Jesús Rivera-Esteban, Franz Koenig, Elena Sena, Raluca Pais, Ramiro Manzano, Joan Genescà, Frank Tacke, Vlad Ratziu,
Veröffentlicht in: Alimentary Pharmacology and Therapeutics, Ausgabe Bimonthly, 2023, Seite(n) Volume 57, Ausgabe 9 Pages: 948–961, ISSN 1365-2036
Herausgeber: John Wiley and Sons Ltd.
DOI: 10.1111/apt.17456

Lessons learned from drug trials in neurofibromatosis: A systematic review

Autoren: Britt A.E.Dhaenens, Rosalie E.Ferner, D. Gareth Evans, Guenter Heimann, Cornelia Potratz, Edwin van de Ketterij, Angela M.Kaindl, Geesje Hissink, Charlotte Carton, Annette Bakker, Marco Nievo. Eric Legius; Rianne Oostenbrink
Veröffentlicht in: European Journal of Human Genetics, Ausgabe Volume 64, Ausgabe 9, 2021, ISSN 1769-7212
Herausgeber: Elsevier BV
DOI: 10.1016/j.ejmg.2021.104281

Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL

Autoren: Franz Koenig, Cécile Spiertz, Daniel Millar, Sarai Rodríguez-Navarro, Núria Machín, Ann Van Dessel, Joan Genescà, Juan M. Pericàs, Martin Posch, Adrian Sánchez-Montalva, Ana Belén Estevez, Àlex Sánchez, Anna Sanjuan, Elena Sena, Emma Granados, Esther Arévalo de Andrés, Fátima Nuñez, Gara Arteaga, Gabriela Perez Fuentes Ruiz, Guillermo Fernández, Jesus Rivera-Esteban, Joan Comella, J
Veröffentlicht in: eClinicalMedicine, Ausgabe 11, 2024, Seite(n) n/a, ISSN 2589-5370
Herausgeber: Elsevier
DOI: 10.1016/j.eclinm.2023.102384

Efficient Adaptive Designs for Clinical Trials of Interventions for COVID-19

Autoren: Nigel Stallard, Lisa Hampson, Norbert Benda, Werner Brannath, Thomas Burnett, Tim Friede, Peter K. Kimani, Franz Koenig, Johannes Krisam, Pavel Mozgunov, Martin Posch, James Wason, Gernot Wassmer, John Whitehead, S. Faye Williamson, Sarah Zohar, Thomas Jaki
Veröffentlicht in: Statistics in Biopharmaceutical Research, Ausgabe 12/4, 2020, Seite(n) 483-497, ISSN 1946-6315
Herausgeber: Taylor & Francis Group, LLC.
DOI: 10.1080/19466315.2020.1790415

On the use of non-concurrent controls in platform trials: a scoping review.

Autoren: Bofill Roig, M., Burgwinkel, C., Garczarek, U., Koenig, F., Posch, M., Nguyen, Q. and Hees, K.,
Veröffentlicht in: Trials, Ausgabe Upon Acceptance, 2023, Seite(n) 1-17, ISSN 1745-6215
Herausgeber: BioMed Central
DOI: 10.1186/s13063-023-07398-7

Platform trials as the way forward in infectious disease’ clinical research: the case of coronavirus disease 2019

Autoren: Juan M.Pericàs; Lennie P.G. Derde; Scott M.Berry
Veröffentlicht in: Clinical Microbiology and Infection, Ausgabe Monthly, 2022, Seite(n) 277-280, ISSN 1198-743X
Herausgeber: Blackwell Publishing Inc.
DOI: 10.1016/j.cmi.2022.11.022

NCC: An R-package for analysis and simulation of platform trials with non-concurrent controls

Autoren: Pavla Krotka, Katharina Hees, Peter Jacko, Dominic Magirr, Martin Posch, Marta Bofill Roig
Veröffentlicht in: Software X, Ausgabe Quarterly, 2023, ISSN 2352-7110
Herausgeber: Elsevier B.V.
DOI: 10.1016/j.softx.2023.101437

Online control of the False Discovery Rate in group sequential platform trials

Autoren: Zehetmayer, S., Posch, M., & Koenig, F.
Veröffentlicht in: Statistical Methods in Medical Research, Ausgabe Bimonthly, 2022, Seite(n) Volume 31, Ausgabe 12, December 2022, Pages 2470-2485, ISSN 0962-2802
Herausgeber: SAGE Publications
DOI: 10.1177/09622802221129051

Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints

Autoren: Elias Laurin Meyer, Peter Mesenbrink,Nicholas A. Di Prospero,Juan M. Pericàs,Ekkehard Glimm,Vlad Ratziu,Elena Sena,Franz König
Veröffentlicht in: PLOS one, 2023, ISSN 1932-6203
Herausgeber: Public Library of Science
DOI: 10.1371/journal.pone.0281674

An interactive R-Shiny app for quickly visualizing a tidy, long dataset with multiple dimensions with an application in clinical trial simulations for platform trials

Autoren: Meyer, E. L., Kumaus, C., Majka, M., König, F
Veröffentlicht in: Software X, Ausgabe Quarterly, 2023, Seite(n) Volume 22, ISSN 2352-7110
Herausgeber: Elsevier BV
DOI: 10.1016/j.softx.2023.101347

EU-PEARL: Changing the paradigm of clinical trials in Europe

Autoren: L Cash-Gibson, JM Pericàs, C Spiertz, E van de Ketterij, E Molero, F Patalano, D Kalra, A Ussi, A Van Dessel, J Genescà
Veröffentlicht in: European Journal of Public Health, Ausgabe Volume 31, Ausgabe Supplement_3, October 2021, 2021
Herausgeber: Oxford University Press
DOI: 10.1093/eurpub/ckab165.657

Suche nach OpenAIRE-Daten ...

Leistungen

Veröffentlichungen

Diese Seite teilen Diese Seite in sozialen Netzwerken teilen

Herunterladen Den Inhalt der Seite herunterladen