Risultati finali
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 IRPThe operational plan for a LNHS and phase 23 IRP trial in NF patients based on WP2
Roadmap for collaboration with other projects/networksA 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-PTAn IRPspecific exploitation and sustainability plan
IRP-Integrated Sustainability and Business plan adapted to TBAn IRPspecific exploitation and sustainability plan
EU-PEARL Sustainability PlanSustainability 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 trialThis 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 ConsultationReport 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 IRPFramework 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
Recommended Ethical, Information Governance and Security Policies for IRPsFinal 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 reviewThis 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 IRPsA 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 PracticesIncludes 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 IRPThis 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 IRPFramework 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 LNHSA 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 NFThis 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 IRPsDefinition 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 IRPThis 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 PlatformsOrganisational 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 ConsultationReport 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 IRPThis 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 ReportIncludes 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 NFContinuing 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 ConsultationReport 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 NASH populations and studies
Interim Report on TRD/PRD master protocol and regulatory requirementsIt 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 RepresentativesThis 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 networkThis 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 recruitmentThis 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 requirementsIt 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 ToolsetPlan 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 requirementsIt 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 PRDA 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 IRPsThis 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 requirementsIt 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' eventThis 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 readinessBased 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 TrialsOrganisational 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 IRPsSee 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 ConsultationReport 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 TrialsIncludes 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 NFAn IRPspecific exploitation and sustainability plan will be created
Report on Methodological Tools for Intervention SelectionIncludes tools and best practice advice for presentation assessment and decisionmaking of additional intervention to already running platform trial
Clinical Network of NF Sites ReportThis 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 IRPsIncludes 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 IRPsThis 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
An online portal and resources that support patientpublic awareness about IRP trials enable their engagement in design and participation in relevant studies
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.
Pubblicazioni
Autori:
Fisher, L., Bofill Roig, M., & Brannath, W
Pubblicato in:
Arxiv gov, Numero N/A, 2023, Pagina/e --
Editore:
--
DOI:
10.48550/arxiv.2301.11711
Autori:
Marta Bofill Roig, Pavla Krotka, Carl-Fredrik Burman, Ekkehard Glimm, Katharina Hees, Peter Jacko, Franz Koenig, Dominic Magirr, Peter Mesenbrink, Kert Viele, Martin Posch
Pubblicato in:
2021
Editore:
Cornell University
Autori:
Nguyen, Q., Hees, K., Hofner, B
Pubblicato in:
Arxiv gov, Numero N/A, 2023, Pagina/e --
Editore:
--
DOI:
10.48550/arxiv.2302.04713
Autori:
Bofill Roig, M., König, F., Meyer, E., & Posch, M.
Pubblicato in:
Clinical Trials, Numero Bimonthly, 2022, Pagina/e Volume 19, Numero 5, October 2022, Pages 502-503, ISSN 1740-7745
Editore:
Sage Publications
DOI:
10.1177/17407745221112016
Autori:
Fischer, L., Bofill Roig, M., & Brannath, W
Pubblicato in:
Arxiv gov, Numero N/A, 2022, Pagina/e --
Editore:
--
DOI:
10.48550/arxiv.2211.11400
Autori:
Stefan M. Gold, Marta Bofill Roig, J. Jaime Miranda, Carmine Pariante, Martin Posch & Christian Otte.
Pubblicato in:
Nature Reviews Psychology, 2022, ISSN 2731-0574
Editore:
Springer Nature Limited
DOI:
10.1038/s44159-021-00012-0
Autori:
Meyer E., Mesenbrink P, Duner-Baldauf C, Glimm E., Li Y, König F.
Pubblicato in:
Arxiv gov, Numero N/A, 2022, Pagina/e 1-24
Editore:
ARXIV.GOV
DOI:
10.48550/arxiv.2202.02182
Autori:
Bofill Roig, M., Melis, G. G., Posch, M., & Koenig, F.
Pubblicato in:
Biostatistics, Numero Quarterly, 2022, ISSN 1465-4644
Editore:
Oxford University Press
DOI:
10.1093/biostatistics/kxac040
Autori:
Elias Laurin Meyer, Peter Mesenbrink, Tobias Mielke, Tom Parke, Daniel Evans, Franz König
Pubblicato in:
Trials, Numero 22/1, 2021, ISSN 1745-6215
Editore:
BioMed Central
DOI:
10.1186/s13063-021-05130-x
Autori:
Juan M. Pericàs, Frank Tacke, Quentin M. Anstee, Nicholas A. Di Prospero, Mette Skalshøj Kjær, Peter Mesenbrink, Franz Koenig, Joan Genescà, Vlad Ratziu
Pubblicato in:
Journal of Hepatology, Numero Monthly, 2022, Pagina/e Vol.78 442–447, ISSN 0168-8278
Editore:
Elsevier BV
DOI:
10.1016/j.jhep.2022.09.021
Autori:
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
Pubblicato in:
Molecular Psychiatry, 2021, ISSN 1476-5578
Editore:
Springer Nature Limited
DOI:
10.1038/s41380-021-01381-x
Autori:
Elias Laurin Meyer,Peter Mesenbrink,Cornelia Dunger-Baldauf,Ekkehard Glimm,Yuhan Li,Franz König,EU-PEARL (EU Patient-cEntric clinicAl tRial pLatforms) Consortium
Pubblicato in:
Pharmaceutical Statistics, 2022, ISSN 1539-1612
Editore:
John Wiley & Sons Ltd
DOI:
10.1002/pst.2194
Autori:
Dressler S, Tàvora Dos Santos Filho E, Seidel S, Gonzalez Moreno J, Heinrich N, Sanchez-Montalva A, Cirillo D
Pubblicato in:
International journal of infectious diseases : IJID : official publication of the International Society for Infectious Diseases,, Numero Monthly, 2023, Pagina/e Volume 130 Suppl.1 S-20-24, ISSN 1201-9712
Editore:
Elsevier BV
DOI:
10.1016/j.ijid.2023.03.008
Autori:
Olivier Collignon,Anja Schiel,Carl-Fredrik Burman,Kaspar Rufibach,Martin Posch,Frank Bretz
Pubblicato in:
Clinical Pharmacology & Therapeutics, 2022, ISSN 1532-6535
Editore:
John Wiley & Sons, Inc
DOI:
10.1002/cpt.2575
Autori:
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
Pubblicato in:
European Journal of Human Genetics, Numero 12 issues per year, 2021, ISSN 1018-4813
Editore:
Natue Publishing Group
DOI:
10.1038/s41431-021-00892-z
Autori:
Robertson, D. S., Wason, J. M., König, F., Posch, M., & Jaki, T.
Pubblicato in:
Statistics in Medicine, Numero 30/year, 2023, Pagina/e Volume 42, Numero 14Jun 2023 Pages 2475–2495, ISSN 0277-6715
Editore:
John Wiley & Sons Inc.
DOI:
10.1002/sim.9733
Autori:
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,
Pubblicato in:
Alimentary Pharmacology and Therapeutics, Numero Bimonthly, 2023, Pagina/e Volume 57, Numero 9 Pages: 948–961, ISSN 1365-2036
Editore:
John Wiley and Sons Ltd.
DOI:
10.1111/apt.17456
Autori:
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
Pubblicato in:
European Journal of Human Genetics, Numero Volume 64, Numero 9, 2021, ISSN 1769-7212
Editore:
Elsevier BV
DOI:
10.1016/j.ejmg.2021.104281
Autori:
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
Pubblicato in:
eClinicalMedicine, Numero 11, 2024, Pagina/e n/a, ISSN 2589-5370
Editore:
Elsevier
DOI:
10.1016/j.eclinm.2023.102384
Autori:
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
Pubblicato in:
Statistics in Biopharmaceutical Research, Numero 12/4, 2020, Pagina/e 483-497, ISSN 1946-6315
Editore:
Taylor & Francis Group, LLC.
DOI:
10.1080/19466315.2020.1790415
Autori:
Bofill Roig, M., Burgwinkel, C., Garczarek, U., Koenig, F., Posch, M., Nguyen, Q. and Hees, K.,
Pubblicato in:
Trials, Numero Upon Acceptance, 2023, Pagina/e 1-17, ISSN 1745-6215
Editore:
BioMed Central
DOI:
10.1186/s13063-023-07398-7
Autori:
Juan M.Pericàs; Lennie P.G. Derde; Scott M.Berry
Pubblicato in:
Clinical Microbiology and Infection, Numero Monthly, 2022, Pagina/e 277-280, ISSN 1198-743X
Editore:
Blackwell Publishing Inc.
DOI:
10.1016/j.cmi.2022.11.022
Autori:
Pavla Krotka, Katharina Hees, Peter Jacko, Dominic Magirr, Martin Posch, Marta Bofill Roig
Pubblicato in:
Software X, Numero Quarterly, 2023, ISSN 2352-7110
Editore:
Elsevier B.V.
DOI:
10.1016/j.softx.2023.101437
Autori:
Zehetmayer, S., Posch, M., & Koenig, F.
Pubblicato in:
Statistical Methods in Medical Research, Numero Bimonthly, 2022, Pagina/e Volume 31, Numero 12, December 2022, Pages 2470-2485, ISSN 0962-2802
Editore:
SAGE Publications
DOI:
10.1177/09622802221129051
Autori:
Elias Laurin Meyer, Peter Mesenbrink,Nicholas A. Di Prospero,Juan M. Pericàs,Ekkehard Glimm,Vlad Ratziu,Elena Sena,Franz König
Pubblicato in:
PLOS one, 2023, ISSN 1932-6203
Editore:
Public Library of Science
DOI:
10.1371/journal.pone.0281674
Autori:
Meyer, E. L., Kumaus, C., Majka, M., König, F
Pubblicato in:
Software X, Numero Quarterly, 2023, Pagina/e Volume 22, ISSN 2352-7110
Editore:
Elsevier BV
DOI:
10.1016/j.softx.2023.101347
Autori:
L Cash-Gibson, JM Pericàs, C Spiertz, E van de Ketterij, E Molero, F Patalano, D Kalra, A Ussi, A Van Dessel, J Genescà
Pubblicato in:
European Journal of Public Health, Numero Volume 31, Numero Supplement_3, October 2021, 2021
Editore:
Oxford University Press
DOI:
10.1093/eurpub/ckab165.657
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