TOOLBOX
The Adaptive Platform Trial Toolbox
Objective of this toolbox
The Adaptive Platform Trial Toolbox aims to collect the accumulated knowledge, experience, and resources (collectively termed as ‘tools’) from multiple projects and trials into a practical and guided toolbox to facilitate planning and conduct of future adaptive platform trials in any therapeutic area.
Why should you use the toolbox?
Adaptive platform trials constitute a dynamic topic in the current clinical research environment. Design, conduct, and even the naming of adaptive platform trials are in active discussion. Therefore, the toolbox has been structured as a practical aid to support complex clinical research. If you need resources regarding adaptive platform trials including, general information as well as specific resources for the different steps in the design or conduct, you can look for recommendations within the toolbox. You can consult the toolbox for training purposes as well.
How should you use the toolbox?
The toolbox has been structured with different categories for different trial steps. Each tool is presented with a brief explanation and a resource type, indicating the tool's format and level of validation. The tools are grouped into categories, each with its own tab. On the reference for each tool, the link to access the outside source is included. Further information is also available within the references section. The tools have been selected for their relevance and utility, but the views and opinions expressed in the tools may not represent the specific opinions of the EU-RESPONSE and RECOVER consortia.
The toolbox is still under development and is currently available in a beta version. If you would like to provide feedback or gather more information about the rationale and methodology of the toolbox, please contact us.
| Name | Explanation | Type | Year | Reference |
|---|---|---|---|---|
| A Practical Adaptive & Novel Designs and Analysis toolkit (PANDA) | PNDA is a toolkit developed by a collaborative initiative in UK funded by NIHP. It presents the key information for adaptive design development and trial conduct as well as provides resources to help researchers use adaptive design in practice, such as statistical software, guidance to enhance the reporting and case studies/ examples of different types of adaptive design. | Website, video, guide, checklist, | 2023 | PANDA 2023 |
| Review article: The need for more efficient and patient-oriented drug development pathways in NASH—setting the scene for platform trials | Non-alcoholic steatohepatitis (NASH) constitutes a significant unmet medical need with a burgeoning field of clinical research and drug development. Platform trials (PT) might help accelerate drug development This review provides a comprehensive and nuanced assessment of the NASH clinical development landscape. | Document, journal article, review, expert opinion | 2023 | Pericàs et al, 2023a |
| Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints | This article describe some of the activities of the EU-PEARL consortium (EU Patient-cEntric clinicAl tRial pLatforms) regarding the use of platform trials in NASH, in particular the proposed trial design, decision rules and simulation results. | Document, journal article, methodology | 2023 | Meyer et al, 2023 |
| Platform trials as the way forward in infectious disease’ clinical research: the case of coronavirus disease 2019 | This Commentary reviews the experiences of several platform trials for COVID sicu as , REMAP-CAP), WHO Solidarity, RECOVERY,ATTACC, PRINCIPLE, AGILE, COPPS, and OPTIMISE C-19, and focus on the basic concepts and methodological keys of PTs using some of the recent developments in COVID-19 treatment to illustrate the potential of these trial designs. | Document, journal article, commentary | 2023 | Pericàs et al, 2023b |
| Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and Cons | Although the pipeline for drug development in NASH, active conventional Phase 3, often fails to achieve the endpoints necessary for approval by regulatory agencies. APT might be an attractive complement or alternative to conventional clinical trials by using a master protocol that allows for evaluating multiple investigational medicinal products concurrently or sequentially with a single, shared control arm. The possibility of using APT in NASH drug development is discussed. | Document, journal article, commentary, expert opinion | 2023 | Pericàs et al, 2023c |
| Platform Trial Best Practices Tool | The tool looks at the main tasks involved in planning a clinical trial and highlights the special considerations that arise when designing a platform trial on top of those that are typical for a conventional trial. | Document, project deliverable, template | 2023 | EUPEARL template 6 2023 |
| EU-PEARL Patient and Community Engagement in Platform Trials (PaCEPT) | This website, created by IMI EU PEARL, shows the information resources to support patient-centric platform trials. Resources are shown with each stakeholder category. | Document, video, presentation, website | 2023 | EUPEARL PE 2023 |
| Outcomes of workshops delivered by EPF and Patient representatives | Within EU-PEARL, several Patient and Community Engagement workshops and sessions have been organised with different aims, evolving as the project progresses. This deliverable informs on the EU-PEARL Platform for Patient and Community Engagement in Platform Trials (PaCEPT) and the outcomes of the patient and community engagement (PCE) workshops delivered together with patient and community representatives and covers the design and development of the PaCEPT platform. | Document, project deliverable, discussion | 2023 | EU PEARL D1.13 |
| Methodology and design of platform trials: a meta-epidemiological study. | The objective of this review is to summarize published platform trials, examine specific methodological design features among these studies, including the basics of the methodological and statistical considerations. | Document, journal article, review | 2023 | Petre et al 2023 |
| Platform trials for anaesthesia and perioperative medicine: a narrative review. | This paper provides an overview of platform trials and discusses the possibility of this design for anaesthesia. | Document, journal article, review, expert opinion | 2023 | Myles et al. 2023 |
| Feasibility of a Platform Trial Design for the Development of Mobile Health Applications: A Review. | A novel adaptive trial design called platform trials (PTs) may offer an effective, efficient, and unbiased approach to evaluate different developer versions of mobile health (m-health) apps. This review discussed the feasibility of PTs for mobile health application | Document, journal article, descriptive review | 2023 | Aqib et al, 2023 |
| Final report on Clinical operations best practices report | The deliverable is a project review of the current platform trial landscape to understand which challenges have the most complexity and impact. It is developed within EU-PEARL project and includes a literature search, website review, and an outreach survey to trial teams. | Document, project deliverable, review | 2023 | Parke et al. 2023 |
| Patient Protocol Engagement Toolkit | Transcelerate developed the Patient Protocol Engagement Toolkit (P-PET) ,a comprehensive set of materials that sponsors and other stakeholders can use to engage patients and caregivers during protocol development. | Document, video, presentation by private company, template | 2022 | Trancelerate |
| The European clinical research response to optimise treatment of patients with COVID-19: lessons learned, future perspective, and recommendations | This review investigates characteristics of implemented adaptive design clinical trials and provides examples of regulatory experience with such trials. | Document, journal article, discussion and comment | 2022 | Goosens et al. 2022 |
| Choosing drugs for UK COVID-19 treatment trials | The paper discussed the UK government funded a portfolio of platform trials to develop new treatments for COVID-19. A key feature was the independent prioritization of candidate drugs with central coordination to prevent duplication, accelerating recruitment to deliver definitive trial results. | Document, journal article, discussion and comment | 2022 | Chinnery et al. 2022 |
| Platform trials and the future of evaluating therapeutic behavioural interventions | This artcle discussed the platform trials on clinical psychology. It could have a transformative effect more broadly by increasing the speed with which treatment effects are estimated, getting effective interventions into clinical care more quickly, and providing much needed comparisons between different interventions for the same condition. | Document, journal article, discussion and comment | 2022 | Gold et al. 2022 |
| Uptake of the multi-arm multi-stage (MAMS) adaptive platform approach: a trial-registry review of late-phase randomised clinical trials | Thuis reviews examined uptake of registered, late-phase MAMS platforms in the EU clinical trials register etc. and highlights the potential scope of MAMS platform trials and may assist research teams considering use of this approach in the late-phase randomised clinical trial setting |
Document, journal article, review and discussion | 2022 | Noor et al. 2022 |
| On model-based time trend adjustments in platform trials with non-concurrent control | This article discussed a use of non-concurrent controls in APT. In addition to concurrent controls, using non-concurrent controlscan improve the trial’s efficiency by increasing power and reducing the required sample size, but can introduce bias due to time trends. | Document, journal article, methodology | 2022 | Roig et al. 2022 |
| Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development. | The platform trial approach is now being employed more often in neurology. This paper describes a newly launched platform trial for ALS. The Healey ALS Platform Trial is testing multiple investigational products concurrently in people with ALS, with the goal of rapidly identifying novel treatments, biomarkers, and trial endpoints. | Document, journal article, commentary | 2022 | Paganoni et al. 2022 |
| Clinical trials in global health | This Lancet Global Health Series of four papers and a commentary show the challenges in clinical trial research in global health and offers solutions that can optimise statistical efficiency and sustainability, including adaptive trial designs and master protocols, with considerations for the required clinical research infrastructure. | Document, journal article, discussion and comment | 2021 | Bhandari et al. 2021, Dron et al. 2021, Park et al. 2021a, Park et al. 2021b, Park et al. 2021c |
| PrecISE: Precision Medicine in Severe Asthma: An adaptive platform trial with biomarker ascertainment | PrecISE, or Precision Interventions for Severe and/or Exacerbation-Prone Asthma Network, is a network of medical centres, doctors, and scientists sponsored by the U.S. National Heart, Lung, and Blood Institute (NHLBI). It was established with the goal of designing and executing a trial that uses adaptive platform design techniques. This paper discussed a rapid evaluation of novel interventions in biomarker-defined subgroups of severe asthma, seeking to refine these biomarker subgroups, and to identify early markers of response to therapy. | Document, journal article, methodology, discussion | 2021 | Israel et al. 2021 |
| Accelerating Adoption of Complex Clinical Trials in Europe and beyond | Report and presentations from a multi-stakeholder workshop organized by EFPIA to develop shared solutions for the use of complex clinical trials to optimise drug development in Europe | Website, presentation, workshop | 2021 | EFPIA |
| Practical Considerations and Recommendations for Master Protocol Framework: Basket, Umbrella and Platform Trials. | This paper provides an overview of the master protocol framework, unify the definitions with some examples, review the statistical methods for the designs and analyses, and focus our discussions on some practical considerations and recommendations of master protocols to help practitioners better design and implement such studies. | Document, journal article, review | 2021 | Lu 2021 |
| Collaborative Platform Trials to Fight COVID-19: Methodological and Regulatory Considerations for a Better Societal Outcome. | This paper provides corresponding regulatory perspectives on approval and reimbursement, and notes that the optimal design of a platform trial will differ with our societal objective and by stakeholder. | Document, journal article,review | 2021 | Collignon 2021 |
| Report on terminology, references and scenarios for platform trials and master protocols | The project deliverable contains a selective glossary of terms for complex trials and platform trials. It also lists the current published platform trials and contains a curated and rated set of seminal publication references for Platform Trials. Developed within EU-PEARL project. | Document, project deliverable, review | 2020 | Parke et al. 2020 |
| Project: master protocol studies. Using master protocol studies to drive medical product development | A set of project resources that guide the appropriate use of master protocols, developed by CTTI. | Document, project outcome, guide | 2020 | CTTI 2020a |
| The evolution of master protocol clinical trial designs: a systematic literature review | The journal review identifies existing master protocol studies and summarizes their characteristics. The review also identifies articles relevant to the design of master protocol trials, such as proposed trial designs and related methods. | Document, journal article, systematic review | 2020 | Meyer et al. 2020a |
| Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs | The journal article introduces the main features of adaptive designs and commonly used terminology, highlighting their utility and pitfalls, and illustrate their use through case studies of adaptive trials. | Document, journal article, correspondence | 2020 | Burnett et al. 2020 |
| Effective delivery of Complex Innovative Design (CID) cancer trials—a consensus statement | The journal article provides ten consensus recommendations to improve the conduct, quality and acceptability of complex oncology trials. While it has been prepared for oncology within the United Kingdom setting, the recommendations could be of general use for adaptive platform trials. | Document, journal article, consensus statement | 2020 | Blagden et al. 2020 |
| Delivering complex and innovative trials: case study library | This website includes podcast series and case studies of adaptive platform trials performed in the United Kingdom. Hosted by the National Institute of Health Research. | Document, video, and podcast, website, case study library | 2020 | NIHR 2020 |
| PARADIGM patient engagement toolbox | This project deliverable centralises all PARADIGM’s co-created recommendations, tools and relevant background information to make patient engagement in medicines development easier for all. The toolbox could help develop clinical trials with a further enhanced patient-focus and improve the experience of patients participating in the trials. Developed by PARADIGM project. | Document, project deliverable, guide, checklist, and article | 2020 | PARADIGM 2020 |
| Innovation in Clinical Trial Design: A review of The Clinical Trial Design Landscape, A white paper by the EFPIA Clinical Trial Design Taskforce on behalf of the EFPIA Clinical Research Expert Group | European Federation of Pharmaceutical Industries and Associations (EFPIA) Clinical Research Expert Group developed this white paper for a landscape review of innovation in clinical trial design, such as adaptive designs. | Document, rerview | 2020 | EFPIA 2020 |
| EU-PEARL: Changing the paradigm of clinical trials in Europe | This article commented that The EU Patient-cEntric clinicAl tRial pLatforms (EU-PEARL) project aims to expand the use of platform trials as the backbone of drug development. | Document, journal article, commentary | 2020 | Gibson et al, 2020 |
| Efficient adaptive designs for clinical trials of interventions for COVID-19 | This is the review of 4 ongoing platform trials ( REMAP CAP, AGILE, RECOVERY, VAPE-COVID) | Document, journal article, review | 2020 | Stallard et al, 2020 |
| Adaptive trial designs - introduction for non-statisticians | This video introduces adaptive trial design options for non-statisticians with trial examples. Presented by Cytel. | Video, presentation by private company | 2019 | Cytel 2019 |
| The COMET handbook: version 1.0 | A key consideration for platform trials is to designate appropriate outcomes. These outcomes must be relevant for new interventional arms and suitable for adaptive elements. This journal article of Core Outcome Measures in Effectiveness Trials (COMET) Initiative addresses issues to consider when selecting or developing a core outcome set. Available outcome or data element repositories are also identified. | Document, journal article, review | 2017 | Williamson et al. 2017 |
| Adaptive design clinical trials: a review of the literature and ClinicalTrials.gov | The journal article reviews the characteristics of implemented adaptive design clinical trials and provides examples of regulatory experience with such trials. | Document, journal article, discussion and comment | 2017 | Bothwell et al. 2017 |
| Adaptive designs for clinical trials | This journal review focuses on adaptive designs of confirmatory clinical trials. It discusses the benefits and limitations of such designs, using four case studies that highlight the statistical and operational considerations. | Document, journal article, review | 2016 | Bhatt and Mehta 2016 |
| New approaches to clinical trials: adaptive designs | The document and attached presentation discuss adaptive design elements for clinical trials, including advantages, disadvantages, rare diseases, and involvement of patients. Prepared by EUPATI. | Document, website, article and presentation | 2015 | EUPATI 2015 |
| Name | Explanation | Type | Year | Reference |
|---|---|---|---|---|
| WHO guidance for best practices for clinical trials draft for public consultation | This guidance was developed to implement the request of the director general for WHA resolution 75.8 . It is under public counsultation. | Document, regulatory guide by health authority | 2023 | WHO Guidance |
| Concept for platfrom trial | This is a concept paper for platform trials published by EMA. | Document, regulatory guide by health authority | 2023 | EMA/CHMP 2023 |
| Master protocols: efficient clinical trial design strategies to expedite development of oncology drugs and biologics - guidance for industry | This FDA guidance provides advice to sponsors of drugs and biologics for cancer treatment regarding the design and conduct of clinical trials, other than first-in-human trials, intended to simultaneously evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure (master protocols) in adult and pediatric cancers. | Document, regulatory guide by health authority | 2022 | FDA 2022a |
| Complex clinical trials - Questions and answers | This document aims to address challenges sponsors may face as regards scientific and operational aspects of complex clinical trials in the EU/EEA and to encourage considering choices in development as early as possible, in order to generate useful evidence and inform clinical and regulatory decision making | Document, regulatory questions and answers | 2022 | EC,EMA, HMA 2022 |
| Complex Innovative Trial Design Meeting Program | FDA program to facilitate drug development using complex innovative trial design. Five cases were selected and their master protocols are discussed. This webpage presents public meeting records and discussion minutes. | Document, Regulatory program website, video presentation, discussion | 2022 | FDA 2022b |
| WHA 75.8 resolution Strengthening clinical trials to provide high-quality evidence on health interventions and to improve research quality and coordination. | To strengthen the global clinical trial ecosystem, WHA Trial ecosystem includes prioritization of trials relevant to all major population groups, trial funding, design, conduct and reporting, oversight by regulatory bodies, coordination and collaboration, liaison with regulatory bodies, health technology assessment authorities, the perspective of those conducting systematic reviews, the perspective of those conducting systematic reviews etc. | Document, regulatory guide by health authority | 2022 | WHA |
| European Medicines Agency Guidance for Applicants seeking scientific advice and protocol assistance | This guidance document addresses a number of questions that users of the scientific advice or protocol assistance procedures may have. It provides an overview of the procedure to obtain scientific advice or protocol assistance and gives guidance to Applicants in preparing their request. This guidance document also explains the scope and nature of scientific advice and protocol assistance. It will enable Applicants to submit requests which are in line with Scientific Advice Working Party (SAWP) requirements and which can be validated and evaluated quickly and efficiently. | Document, regulatory guideline concept | 2022 | EMA 2022 |
| COVID-19: master protocols evaluating drugs and biological products for treatment or prevention | FDA is issuing this guidance to provide recommendations to sponsors of master protocols evaluating drugs for the treatment or prevention of COVID-19. | Document, regulatory guide by health authority | 2021 | FDA 2021 |
| ICH guideline E8 (R1) on general considerations for clinical studies (step 5 8.8.2023) |
Describes internationally accepted principles and practices in the conduct of both individual clinical trials and overall development strategy for new medical products. R1 includes statements for a wide range of study designs (platform trials etc) and a various data sources. | Document, regulatory guideline | 2021 | EMA/CHMP/ICH/, 2021 |
| Interacting with the FDA on complex innovative trial designs for drugs and biological products - guidance for industry | This FDA document provides guidance to sponsors and applicants on interacting with the FDA on complex innovative trial design proposals for drugs or biological products. It also covers some key aspects of complex trial designs. | Document, regulatory guide by health authority | 2020 | FDA 2020 |
| Recommendation paper on the initiation and conduct of complex clinical trials | This key document provides recommendations for sponsors regarding the authorisation and conduct of complex clinical trials. It describes the current perspective of the CTFG on these trials and outlines the major issues that sponsors should address in the process of initiating and conducting complex clinical trials in the EU/EEA. | Document, regulatory guide by health authority | 2019 | CTFG 2019 |
| Final Concept Paper E20: Adaptive Clinical Trials (step1, 8.8.2023) | The E20 EWG is working on the development of a new E20 Guideline on “Adaptive Clinical Trials” on the design, conduct, analysis, and interpretation of adaptive clinical trials that provides a transparent and harmonized set of principles for the regulatory review of these studies in a global drug development program. These principles should also provide the flexibility to evaluate / discuss innovative approaches to clinical trial design throughout the development process. | Document, regulatory guideline concept | 2019 | ICH E20 2019 |
| Final Concept Paper ICH E6(R3): Guideline for Good Clinical Practice (step3 8.8. 2023) | The global harmonization regulatory guideline for GCP(ICH E6 R3). This revision includes risk-based approach to the design and conduct of clinical trial, increased reliance on electronic systems and non-traditional interventional clinical trials, such as pragmatic clinical trials, decentralized clinical trials, as well as those trials that incorporate real world data sources. | Document, regulatory guideline concept | 2019 | ICH E6(R3) 2019 |
| Adaptive designs in clinical trials: from scientific advice to marketing authorisation to the European Medicine Agency | The journal article assesses scientific advice letters containing questions related to adaptive clinical trials in phases II or III, issued by EMA. It also examines relevant regulatory aspects. | Document, journal article, research paper | 2018 | Collignon et al. 2018 |
| Reflection paper on methodological issues in confirmatory clinical trials planned with an adaptive design | The document outlines some general considerations for studies incorporating interim analyses. A set of minimal requirements is outlined that must be fulfilled whenever confirmatory clinical trials are planned with an adaptive design. Later sections comment on specific design modifications. Published by EMA. | Document, regulatory guide by health authority | 2007 | CHMP 2007 |
| Name | Explanation | Type | Year | Reference |
|---|---|---|---|---|
| Master Protocol Template (MPT) | A template of master protocol for platform trial, containing instruction text, common / suggested text, variable text and examples. It is downloadable as a word documents for readers' editing. It is developed within EU-PEARL project, based on TransCelerate Common Protocol Template CPT V8.0. | Document, project deliverable, guide, checklist, | 2023 | EU-PEARL template 1 2023 |
| Intervention Specific Appendix (ISA) template | A template of intervention specific appendix protocol for a platform trial, containing instruction text, common / suggested text, variable text and examples. It is downloadable as a word documents for readers' editing.This template is used with the Generic Master Protocol Template. It is developed within EU PEARL project, based on TransCelerate Common Protocol Template CPT V8.0. | Document, project deliverable, template | 2023 | EU-PEARL template 2 2023 |
| Guidance for supplementary information to the CTR Cover Letter associated with the Master protocol and ISA submission | A guidance for the cover letter outline including important points when submitting a platform trial application to a regulatory authority. This guideline applies to the companion protocol templates named Generic Master Protocol Template and Intervention Specific Appendix Template documents prepared in the EU-PEARL consortium. | Document, project deliverable, template | 2023 | EU-PEARL template 5 2023 |
| Recommended Ethical, Information Governance and Security Policies for IRPs | This document describes the Recommended Ethical, Information Governance and Security Policies for Integrated Research Platforms (IRPs) of the IMI EU-PEARL project.The deliverable identifies the documents required and the reasons why they are needed. It will also identify where possible existing templates, either within the EU-PEARL catalogue of products or elsewhere. | Document, project deliverable, template | 2023 | EU-PEARL D1.7 2023 |
| Recommended Ethical, Information Governance and Security Policies for IRPs | This deliverable of the EU-PEARL project identifies the documents required for ethical and information governance policy for Integrated Research Platform(IRPs) and the reasons why they are needed. It will also identify where possible existing templates, either within the EU-PEARL catalogue of products or elsewhere. | Document, project deliverable, guide, discussion | 2023 | EU-PEARL D1.7 2023 |
| European Certification Programme for IRP Platforms | This deliverable looks at how to make sure that the assessment and certification of Data research platform / Information and Communications Technology (ICT) systems are sustainable. | Document, project deliverable, guide, discussion | 2023 | EU-PEARL D1.9 2023 |
| Report on Methodological Tools for Intervention Selection | A problem unique to platform trials is deciding not just which investigational medicinal product should be added to the trial, but also the correct timing and procedures to avoid a negative impact on external validity and generalizability of the conclusion(s), as well as to minimize delays in the evaluation of ongoing investigational medicinal products. EU-PEARL framework suggests that the decision to include a new investigational medicinal product should be taken by independent experts within the IMP Selection Scientific Committee. | Document, project deliverable, discussion | 2023 | EU-PEARL D2.9 2023 |
| Report on Regulatory Issues of IRPs | Adaptive platform trial brings some new challenges due to their increased complexity, from an operational, logistical, as well as statistical perspective. Regulators, ethics committees as well as health technology assessment bodies are faced with assessing these challenges.This report summarises these challenges as well as potential solutions with a special focus on regulatory, ethical and HTA related aspects | Document, project deliverable, discussion | 2023 | EU PEARL D2.9 |
| Generic process for an EHR-based patient cohort for IRPs | In the platform trials with bigger and more complicated trial designs, recruiting participants remains a key determinant of research efficiency. This document describes a proposal for a generic process for gaining an estimate of patient numbers that matches the clinical study inclusion criteria at hospitals, based on the analysis of hospitals Electronic Health Records (EHR). This process can be used: 1 during the study feasibility assessment and site selection activities as part of study planning and 2. During the study participants recruitment, and as part of study execution by each clinical site in support of the investigator recruitment activities. | Document, project deliverable, guide, discussion | 2023 | EU-PEARL D3.1 2023 |
| Trial feasibility in federated hospital networks | This article describes the prototype tools and guidance documents that have been created to help building the hospital network as part of an integrated research platform which can conduct platform trials. | Document, project deliverable, guide, discussion | 2023 | EU-PEARL D3.7 2023 |
| TRD and PRD final master protocol for IRP. WP4 – Integrated Research Platform for Major Depressive Disorder (MDD) | EU-PEARL developed adaptive platform trial for treatment-resistant depression (TRD) and partially responsive depression (PRD). This allows new treatments to be added to the trial anytime they become available for testing, increases the speed by which we can select the best treatments and requires fewer participants overall. The present document describes the master design of this platform trial. | Document, project deliverable, protocol | 2023 | EU-PEARL D4.5 2023 |
| Operational Plan to implement TRD/PRD IRP and LNHS. WP4 – Integrated Research Platform for Major Depressive Disorder (MDD) | This report presents an overview of the stakeholder interaction and consultation such as Regulators, Ethics Committees, the Biopharmaceutical Industry, Clinicians/Academics, and Patient and Community Representatives.This has been sought by EU-PEARL for ‘Integrated Research Platform for Major Depressive Disorder (MDD). | Document, project deliverable, discussion | 2023 | EU-PEARL D4.6 2023 |
| TRD/PRD final report on Regulatory and Ethics Consultation. WP4 – Integrated Research Platform for Major Depressive Disorder (MDD) | The report present a draft of how EU-PEARL envisions an integrated research platform for depression. This draft has been discussed formally with the European Medicines Agency (EMA) experts to ensure that the trial yields speedy results on the benefit of any new drug tested and maintains the highest ethical and safety standards. | Document, project deliverable, discussion | 2023 | EU-PEARL D4.7 2023 |
| TB Final Master Protocol for IRP. WP5 – Integrated Research Platform for Tuberculosis (TB) | A major outcome of the EU-PEARL Tuberculosis (TB) workpackage is the development of TB master protocol outline to conduct platform trials in the context of an IRP with the final goal of expediting the development of a treatment regimen. EU-PEARL developed a template for the master protocol that provides guidance for the protocol author(s). | Document, project deliverable, protocol |
2023 |
EU PEARL D5.4 |
| TB Operational plan to implement TB. IRPWP5 – Integrated Research Platform for Tuberculosis (TB) | Alongside the master protocol, the EU-PEARL Tuberculosis (TB) workpackage developed, operational standards and a set of best practices for planning, conduct and reporting on IRPs. This report addresses the best practices among those crucial aspects of implementing an operational plan, particularly focusing on the Community Engagement (CE) in the context of a TB IRP. | Document, project deliverable, protocol | 2023 | EU PEARL D5.5 |
| TB Final Report on Regulatory and Ethics consultation. WP5 – Integrated Research Platform for Tuberculosis (TB) | This consensus document is the result of two very productive meetings held in 2022, which brought together researchers, academics, technical partners, TB drugs and regimens developers, trialists, regulators, guideline developers, programme managers, community representatives and nongovernmental organizations. | Document, project deliverable, protocol | 2023 | EU PEARL D5.6 |
| IRP- Integrated Sustainability and Business Plan adapted to TB | During the EUPEARL project several tools have been developed that may be exploited by other clinical trials . Tools are 1. A Master Protocol Template to conduct TB platform trials including a design proposal for TB adaptive trials, 2. TB Operational plan to implement TB IRP(a community engagement plan ) 3. Capacity Building and Assessment Handbook for sites to conduct TB drug development trial within the IRP environment and a systematic review to identify and assess host and bacterial biomarkers able to predict end of treatment outcomes. | Document, project deliverable, protocol | 2023 | EU PEARL D5.8 |
| NASH Final Master Protocol for an IRP. WP6 – Integrated research platform for non-alcoholic steatohepatitis (NASH) | One of the workpackage of EU-PEARL developed a master protocol to conduct platform trials ( phase 2 b) for NASH. | Document, project deliverable, handbook | 2023 | EU PEARL D6.3 |
| Operational Plan to implement the NASH IRP. WP6 – Integrated research platform for non-alcoholic steatohepatitis (NASH) | This document serves as a template Clinical Operations Plan (COP) to support the proposed NASH IRP. It provides specific details regarding where different sets of instructions can be found, who should take on specific activities, and what information needs to be recorded from the beginning to the end of the trial. | Document, project deliverable, guide | 2023 | EU PEARL D6.4 |
| NASH Final Report on Regulatory and Ethics Consultation. WP6 – Integrated research platform for non-alcoholic steatohepatitis (NASH) | This document presents the process of eliciting feedback from regulatory (EMA innovation taskforce) and ethics stakeholders for the master protocol for a NASH IRP. | Document, project deliverable, protocol | 2023 | EU PEARL D6.5 |
| Clinical research network report to support future NASH IRPs. WP6 – Integrated research platform for non-alcoholic steatohepatitis (NASH) | A key aspect for the success of platform trials is to establish a Clinical Research Network (CRN) comprising healthcare providers, researchers, and relevant stakeholders, in order to collaborate and conduct the trial. This deliverable describes the results of the survey to provide future directions and guidance on how to establish a CRN for a future NASH platform trial. | Document, project deliverable, survey discussion | 2023 | EU PEARL D6.6 |
| IRP Integrated Sustainability Business Plan adapted to NASH-PT. WP6 – Integrated research platform for non-alcoholic steatohepatitis (NASH) | This deliverable describes the value proposition of the NASH IRP, the lessons learned during the project duration that affect the options available to fund and set up the IRP, as well as, the proposed plan to implement such lessons in the near future. | Document, project deliverable, discussion | 2023 | EU PEARL D6.7 |
| Protocol for a Longitudinal Natural History Study (LNHS) in NF. WP7 – Integrated Research Platform for Neurofibromatosis (NF) | EU-PEARL established an Integrated Research Platforms (IRP) for Neurofibromatosis, including Neurofibromatosis type 1 (NF1). Patients for the treatment arms of the NF1-IRP will be selected from an underlying longitudinal natural history study (LNHS), forming the backbone of the IRP.This documents presents a details of LNHS. | Document, project deliverable, discussion | 2023 | EU PEARL D7.3 |
| NF Master Protocol for IRPs. WP7 – Integrated Research Platform for Neurofibromatosis (NF) | The platform trial for NF developed by EU-PEARL will consist of an observational and a treatment phase. In the observational phase, patients will not be treated with an investigational agent and will provide data on the natural course of their disease. If patients need treatment, and a drug is available, patients will be randomized to a treatment arm. The aim of this deliverable is to report the finalized master protocol for the platform trial in NF, as designed by the EU-PEARL team specifically working on this rare disease. | Document, project deliverable, protocol |
2023 |
EU PEARL D7.4 |
| Operational Plan for the implementation of the NF IRP. WP7 – Integrated Research Platform for Neurofibromatosis (NF) | The EU-PEARL project has designed two platform trials for NF, one for NF1 and one for all types of Schwannomatosis tumours. This report explains the steps needed to make these trials happen. | Document, project deliverable, protocol | 2023 | EU PEARL D7.5 |
| NF Specific Report on Regulatory and Ethics Consultation. WP7 – Integrated Research Platform for Neurofibromatosis (NF) | This report is the result of collaboration between the EU-PEARL clinicians, patients, industry, regulatory experts, and Erasmus Universitair Medisch Centrum Rotterdam (EMC) regulatory and ethics review. It also includes a briefing produced for an EMA Innovation Task Force meeting in close collaboration with the Global Coalition for Adaptive Research (GCAR), which is expected to play an essential role in the sustainability of the NF platform trials. | Document, project deliverable, protocol | 2023 | EU PEARL D7.6 |
| Clinical Network of NF Sites Report. WP7 – Integrated Research Platform for Neurofibromatosis (NF) | The aim of this deliverable is to present our approach in defining site selection criteria for the network of NF sites that could run the platform trials. The deliverable focuses on the network for the NF1 platform trial. At the time of writing this report, the NF2 and SWN protocols are not yet sufficiently defined to allow for meaningful decisions on site selection criteria. | Document, project deliverable, guide, discussion | 2023 | EU PEARL D7.7 |
| Accelerating clinical trial implementation in the context of the COVID-19 pandemic: challenges, lessons learned and recommendations from DisCoVeRy and the EU-SolidAct EU response group | This is a review paper discussed the European clinical trial for COVID including the EU platform trials | Document, journal article, discussion and commentary | 2023 | Diallo 2022 |
| Functional requirements for a federated network of patient data for cohort building, clinical trial feasibility and recruitment | This document explains the protocol feasibility and patient recruitment Information and Communication Technology (ICT) functions that should be implemented by ICT platforms and tools that reuse hospital Electronic Health Record (EHR) data, supporting an industry or public platform trial sponsor and contributing research organisations to undertake platform trials. | Document, project deliverable, guide, discussion | 2022 | EU PEARL D3.4 |
| Longitudinal Natural History Study(LNHS) / Readiness cohort Protocol for MDD platform. WP4 – Integrated Research Platform for Major Depressive Disorder (MDD) | One of the purposes of the EU-PEARL project, is to develop an adaptive platform trial on difficult-to-treat depression. This report provides the public information about an observational study on difficult-to-treat depression that is a part of this new framework of the platform design. LMHS arrow us to recruit large and continuous number of participants and promote a sustainable model for conducting clinical trials in this field. | Document, project deliverable, protocol (cohort study), discussion | 2022 | EU PEARL D4.4 |
| I-SPY COVID adaptive platform trial for COVID-19 acute respiratory failure: rationale, design and operations. | The Investigation of a Serial studies to Predict Your Therapeutic Response with Imaging And moLecular Analysis (ISPY COVID-19 trial) was designed and implemented in early 2020 to evaluate investigational agents rapidly and simultaneously on a phase 2 adaptive platform. This is a protocol paper for ISPY COVID. | Document, journal article, protocol paper, discussion | 2022 | Files et al. 2022 |
| Experiences of running a stratified medicine adaptive platform trial: Challenges and lessons learned from 10 years of the FOCUS4 trial in metastatic colorectal cancer. | FOCUS4 was the first molecularly stratified trial in metastatic colorectal cancer and it remains one of the first umbrella trial designs to be launched globally. This paper aims to describe lessons learned from the delivery of the trial over the last 10 years. | Document, journal article, review | 2022 | Brown et al. 2022 |
| Implementation of platform trials in the COVID-19 pandemic: A rapid review. | Between 2001 and 2019, 16 platform trials were initiated globally for COVID-19 pandemic. This review was conducted to quantify and describe platform trials used in COVID-19. | Document, journal article, review | 2022 | Vanderbeek 2022 |
| Guidelines for tailoring the informed consent process in clinical studies | These guidelines have been designed to provide information and evidence to assist with the development, or review of the consent process for use in clinical studies with human participants. Developed within i-CONSENT project. | Document, project deliverable, guide and checklist | 2021 | i-CONSENT 2021 |
| Lessons from COVID-19: the first year of the REMAP-CAP global adaptive platform trial | The video with slides discusses the accumulated experience from REMAP-CAP COVID-19 platform trial with a focus on design, Bayesian statistical model, data management, interpretation of results, funding, and challenges for publication and trial logistics. Hosted by NIH Collaboratory. | Video, presentation, discussion | 2021 | NIH Collaboratory 2021 |
| RECOVERY 1 year on: a rare success in the COVID-19 clinical trial landscape | For the first anniversary of RECOVERY in 2021, Peter Horby, co-lead on the RECOVERY platform trial was interviewed about the histoy and the future of RECOVERY. In a landscape marked by poorly designed and underpowered trials, RECOVERY stands out as a rare bright spot. It recruited 40000 patients into 13 treatment arms and demonstrates the benefits of the steroid dexamethasone and tocilizumab, while showing the lack of efficacy for azithromyscin, colchicine, convalesent plasma, hydroxychloroquine, and lopinavir-ritonavir. | Document, journal article, interview, discussion and comment | 2021 | Mullard. 2021 |
| Costs and staffing resource requirements for adaptive clinical trials: quantitative and qualitative results from the Costing Adaptive Trials project | This is a mock costing exercise amongst seven CTUs in the UK. This work sheds light on additional resources required to adequately support a high-quality adaptive trial. The percentage increase in costs for supporting an adaptive trial was generally modest and should not be a barrier to adaptive designs being cost-effective to use in practice. | Document, journal article, commentary | 2021 | Wilson et al. 2021 |
| Capacity Building and Assessment Handbook for site readiness. WP5 – Integrated Research Platform for Tuberculosis (TB) | This handbook represents an innovative approach that will help to prepare sites for future IRPs for TB, discussed within one of the working package of the EU-PEARL project. | Document, project deliverable, handbook | 2021 | EU PEARL D5.2 |
| The Precision Interventions for Severe and/or Exacerbation-Prone (PrecISE) Asthma Network: An overview of Network organization, procedures, and interventions | This paper describe the Precision Interventions for Severe and/or Exacerbation-Prone Asthma (PrecISE) Network, a groundbreaking collaborative effort of asthma researchers and biostatisticians from around the United States. The PrecISE Network was designed to conduct phase II/proof-of-concept clinical trials of precision interventions in the population with severe asthma. Using an innovative adaptive platform trial design, the PrecISE Network will evaluate up to 6 interventions simultaneously in biomarker-defined subgroups of subjects. | Document, journal article, commentary | 2020 | Georas et al. 2021 |
| Master protocol content development guide | A step-by-step tool to develop an engagement strategy for engaging all relevant stakeholders early in the master protocol development process, developed by CTTI. | Document, project outcome, guide | 2020 | CTTI 2020b |
| Adaptive platform trials: scalable from breast cancer to COVID | This video with attached slides summarizes how the adaptive platform I-SPY trials has been developed for breast cancer and how it is replicated as a COVID trial. It depicts the life cycle of an adaptive platform trial with rationale, design, operations, infrastructure, intervention selection, data collection, added value, and publications. Hosted by NIH Collaboratory. | Video, presentation, discussion | 2020 | NIH Collaboratory 2020 |
| Good Participatory Practice (GPP) for COVID-19 clinical trials: A toolbox | Good Participatory Practice (GPP) is a principle-based approach to effectively engage stakeholders in the design and conduct of prevention and treatment trials.WHO adapted GPP - Emerging Pathogens (GPP-EP) for COVID clinical trial in 2020. This tool includes some key themes of GPP and additional GPP resources. | Document, guide by health authority, checklist | 2020 | WHO 2022 |
| Clinical ICF - Study information and informed consent form | An informed consent form template harmonised for clinical studies and further research. It is meant to cover all information required by the General Data Protection Regulation (GDPR) within a traditional (non-electronic) informed consent document to be signed by patients or healthy volunteers before participating in a clinical study; it does not address country specific requirements. Developed within DO->IT project. | Document, project deliverable, template | 2019 | Norwegian Institute of Public Health and Boehringer Ingelheim 2019 |
| Mind the gap? The platform trial as a working environment | In this commentary journal article, trial management and data management teams of STAMPEDE and FOCUS4 trials summarise the operational challenges and personal experience of individual researchers, to enable the broader clinical trials community to learn from their experiences. | Document, journal article, commentary | 2019 | Morrell et al. 2019 |
| This is a platform alteration: a trial management perspective on the operational aspects of adaptive and platform and umbrella protocols | The journal article provides experiences gathered in FOCUS4 and STAMPEDE adaptive platform trials from a trial management perspective, highlighting the challenges and successes encountered in running adaptive platform trials. | Document, journal article, methodology | 2019 | Schiavone et al. 2019 |
| Flexible trial design in practice - stopping arms for lack-of-benefit and adding research arms mid-trial in STAMPEDE: a multi-arm multi-stage randomized controlled trial | The journal article describes methodological and practical experience arising with stopping recruitment to research arms following a pre-planned intermediate analysis and adding a new research arm during STAMPEDE, an adaptive platform oncology trial. It also includes criteria for assessing potential new intervention arms. | Document, journal article, methodology | 2012 | Sydes et al. 2012 |
| Name | Explanation | Type | Year | Reference |
|---|---|---|---|---|
| HECT-Highly efficient clinical trial simulation | An online-application developed by MTEC (Medical Technology Evidence Knowledge) Science Inc., that allows users to perform simulations of Bayesian platform adaptive trial to evaluate the pros and cons of candidate trial designs. | Webpage, statistical software | unknown | MTEC |
| EU-PEARL Webinar on Non-Concurrent Controls in Platform Trials | EU-PEARL Webinar on Non-Concurrent Controls in Platform Trials | Video, presentation, discussion | 2023 | EU PEARL video 2023 |
| Integrated Platform for Designing Clinical Trials | Clinical Trial Design Software for calculating sample size for basket and platform trials developed by the research teams in the University of Texas MD Anderson Cancer Center. | Website, software | 2023 | MD anderson cancer center. 2023 |
| NCC: An R-package for analysis and simulation of platform trials with non-concurrent controls | R-package NCC (https://CRAN.R-project.org/package=NCC) is for the design and analysis of platform trials using non-concurrent controls. NCC allows for simulating platform trials and evaluating the properties of analysis methods that make use of non-concurrent controls in a variety of settings. We describe the main NCC functions and show how to use the package to simulate and analyse platform trials by means of specific examples. | Document, journal article, methodology, simulation | 2023 | Krotka et al, 2023 |
| Statistical Analysis Plan (SAP) template | A template for a statistical plan for a platform trial. Developed within EU-PEARL based on TransCelerate Statistical Analysis Plan SAP V3.0 | Document, project deliverable, template | 2023 | EU PEARL template 3 2023 |
| Data Monitoring Committee (DMC) charter template | A template of Data Monitoring Committee Charter for platform trial. Developed within EU-PEARL. | Document, project deliverable, template | 2023 | EU PEARL template 4 2023 |
| Framework for ensuring and Benchmarking Acceptable Data Quality Standards for Platform Trials |
This deliverable covers the development of an 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 (e.g., from electronic health record –EHR – systems) is of a satisfactory quality. | Document, project deliverable, guide, discussion | 2023 | EU PEARL D1.10 |
| Estimands and Complex Innovative Designs | While the estimand framework is relevant to any study in which a treatment effect is estimated, experience is lacking with regards to its application to complex designs. This article discussed possible ways of implementing the estimand framework for different types of complex innovative designs. | Document, journal article, commentary | 2022 | Collignon et al. 2022 |
| Experiences of the Data Monitoring Committee for the RECOVERY trial, a large-scale adaptive platform randomised trial of treatments for patients hospitalised with COVID-19. | This paper summarises the experiences of the Data Monitoring Committee (DMC) for the Randomised Evaluation of COVID therapy trial (RECOVERY), a large-scale randomised adaptive platform clinical trial of treatments for hospitalised patients with COVID-19. | Document, journal article, commentary. | 2022 | Sandercock et al. 2022 |
| Systematic review of available software for multi-arm multi-stage and platform clinical trial design | This systematic research reviews existing software for the design of platform trials. Developed within EU-PEARL project. | Document, journal article, systematic review | 2021 | Meyer et al. 2021 |
| Statistical consideration when adding new arms to ongoing clinical trials: the potentials and the caveats | The journal article reviews the impact on statistical inference of adding arms. It also covers changing the control arm and how patient eligibility would complicate the trial design and analysis, the more generic statistical literature, and statistical considerations for different phases and funders. | Document,journal article, commentary | 2021 | Lee et al. 2021 |
| Data, data all around | This news article from the Lancet Digital Health highlights the complex routine data collection machinery behind the RECOVERY trial, simplifying site operations. | Document, journal article, news | 2021 | McCall 2021 |
| Statistical simulation in master protocols: components & communications considerations |
This tool provides early adopters with an overview of components of statistical simulation and tips for how to organize presentations of simulated trials to key stakeholder groups. Developed by CTTI. |
Document, project outcome, guide | 2020 | CTTI 2020c |
| Decision rules for identifying combination therapies in open-entry, randomized controlled platform trial | The definition of error rates is not straightforward as classical error rate concepts are not applicable. This article discusses a set of error rates and operating characteristics and then uses these to compare a set of design parameters under a range of simulation assumptions for an open-entry, exploratory platform trial design comparing combination therapies to the respective monotherapies and standard-of-care. | Document, journal article, commentary | 2020 | Meyer et al. 2020b |
| Adaptive design clinical trials for drugs and biologics - guidance for industry | This FDA document provides guidance to sponsors and applicants submitting investigational new drug applications, new drug applications, biologics licensing applications, or supplemental applications on the appropriate use of adaptive designs for clinical trials to provide evidence of the effectiveness and safety of a drug or biologic. | Document, regulatory guide by health authority | 2019 | FDA 2019b |
| Individualized screening trial of innovative glioblastoma therapy (INSIGhT): a Bayesian adaptive platform trial to develop precision medicines for patients with glioblastoma | This journal article presents the Bayesian, adaptive, platform trial INSIGhT to develop precision medicine approaches in glioblastoma. Detailed information on the statistical model, simulations, and randomization algorithm is provided with expected benefits and challenges. | Document, journal article, methodology | 2019 | Alexander et al. 2019 |
| Changing platforms without stopping the train: experiences of data management and data management systems when adapting platform protocols by adding and closing comparisons | The journal article shares experiences from FOCUS4 and STAMPEDE trials from the operational aspects of running these adaptive trials, focusing on data management. Adding and closing intervention arms, number of case report form changes, database amendments and database growth are discussed. | Document, journal article, methodology | 2019 | Hague et al. 2019 |
| The adaptive platform trial - the statistical efficiencies | The video provides statistical and methodological aspects of adaptive platform trials. It also covers case studies. Prepared by Berry Consultants. | Video, presentation by private company | 2015 | Berry Consultants 2015 |
| Name | Explanation | Type | Year | Reference |
|---|---|---|---|---|
| How to Use and Interpret the Results of a Platform Trial Users’ Guide to the Medical Literature | This Users’ Guide is intended to help clinicians and readers understand articles reporting on interventions evaluated using platform trial designs. | Document, journal article, guide, discussion and comment | 2020 | Park et al. 2020 |
| The Adaptive designs CONSORT Extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design | This CONSORT extension is meant to provide reporting guidance for any randomised trial that was designed using an adaptive design. | Document, website and journal article, statement and checklist | 2020 | Dimairo et al. 2020 |
| An overview of platform trials with a checklist for clinical readers | The objective of this journal review is to outline key considerations for general clinical readers when critically evaluating publications on platform trials and for researchers when designing these types of clinical trials. Supplementary tables provide example use of the checklist for platform trials. | Document, journal article, review and checklist | 2020 | Park et al. 2020a |
| CONSORT Consolidated Standards of Reporting Trials | A group of scientists and editors developed the CONSORT (Consolidated Standards of Reporting Trials) statement to improve the quality of reporting of RCTs in 2001. The statement consists of a checklist and flow diagram that authors can use for reporting an RCT. The website provides CONSORT 2010 Statement: updated guidelines for reporting parallel group randomised trials, extention of statement , checklist and elaboration paper. | Document, website checklist, published paper, template | 2010 | CONSORT 2010 |
| Name | Explanation | Type | Year | Reference |
|---|---|---|---|---|
| RAMPART (Renal Adjuvant MultiPle Arm Randomised Tria): An international investigator-led phase III multi arm multi stage multi-centre randomised controlled platform trial of adjuvant therapy in patients with resected primary renal cell carcinoma (RCC) at high or intermediate risk of relapse | RAMPART(Renal Adjuvant MultiPle Arm Randomised Trial) is an international investigator-led phase III multi arm multi stage multi-centre randomised controlled platform trial of adjuvant therapy in patients with resected primary renal cell carcinoma (RCC) at high or intermediate risk of relapse | Document, trial website, protocol, video, presentation, training | 2022 | RAMPART 2022 |
| A multinational, phase 2, randomised, adaptive protocol to evaluate immunogenicity and reactogenicity of diferent COVID-19 vaccines in adults ≥75 already vaccinated against SARS-CoV-2 (EU-COVAT-1-AGED): a trial conducted within the VACCELERATE network | VACCELERATE is a clinical research network for the coordination and conduct of COVID-19 vaccine trials. The network is comprised of academic institutions from all over Europe: The consortium is led by the University Hospital Cologne, Germany. This is a protocol paper for EU-COVAT-1-AGED, a trial to test different vaccine immunogenicity and reactogenicity in adults ≥75. This is an adaptive platform conducted within the VACCELERATE network | Document, journal article, protocol paper | 2022 | Neuhann 2022 |
| Motor Neuron Disease Systematic Multi-Arm Adaptive Randomised Trial (MND-SMART): a multi-arm, multi-stage, adaptive, platform, phase III randomised, double-blind, placebo-controlled trial of repurposed drugs in motor neuron disease. | Motor Neuron Disease Systematic Multi-Arm Adaptive Randomised Trial (MND-SMART) aims to evaluate the efficacy of drugs efficiently and definitively in a multi-arm, multi-stage, adaptive trial. The first two drugs selected for evaluation in MND-SMART are trazodone and memantine. | Document, journal article, protocol paper | 2022 | Wong et al. 2022 |
| Helping alleviate the longer-term consequences of COVID-19 (HEAL-COVID) | A national platform trial jointly Sponsored by Cambridge University Hospitals NHS Foundation Trust and The University of Cambridge. It aims to evaluate the impact of treatments on longer-term morbidity, mortality, re-hospitalisation, symptom burden and quality of life associated with COVID-19. | Document, trial website, protocol and consent form | 2021 | Cambridge University Hospitals NHS Foundation Trust 2021 |
| Seamless phase I/IIa platform for the rapid evaluation of candidates for COVID-19 treatment (AGILE) | AGILE is a multicentre, multi-arm, multi-dose, multi-stage open-label, adaptive, seamless phase I/II Bayesian randomised platform trial to determine the optimal dose, activity and safety of multiple candidate agents for the treatment of COVID-19. Sponsored by University of Liverpool. | Document and video, trial website, protocol and manual | 2021 | University of Liverpool 2021 |
| The UPMC OPTIMISE-C19 (OPtimizing Treatment and Impact of Monoclonal antIbodieS through Evaluation for COVID-19) trial: a structured summary of a study protocol for an open-label, pragmatic, comparative effectiveness platform trial with response-adaptive randomization | A protocol for OPTIMISE-C19 (OPtimizing Treatment and Impact of Monoclonal antIbodieS through Evaluation for COVID-19) trial, an Open-label, pragmatic, comparative effectiveness platform trial with response-adaptive randomization. The primary objective is to evaluate the comparative effectiveness of COVID-19 specific monoclonal antibodies (mABs) with US FDA Emergency Use Authorization (EUA), alongside UPMC Healtlh System efforts to increase patient access to these mABs. | Document, journal article, Protocol | 2021 | Huang et al. 2021 |
| PANORAMIC (platform adaptive trial of novel antivirals for early treatment of COVID 19 in the community) trial | PANORAMIC is a NIRH funded, national priority adaptive platform trial in UK, designed as a ‘platform clinical trial for the early treatment of COVID 19 community. | Ddocument, Trial website, video, protocol, ICF | 2021 | PANORAMIC |
| The COVID-19 Outpatient Pragmatic Platform Study (COPPS): Study design of a multi-center pragmatic platform trial. | This is the protocol of The COVID-19 Outpatient Pragmatic Protocol Study (COPPS), a flexible phase 2, multi-site, randomized, blinded trial based at Stanford University designed to overcome these issues by simultaneously evaluating multiple COVID-19 treatments in the outpatient setting in one common platform with shared controls. | Document, journal article, protocol paper | 2021 | Bunning 2021 |
| Accelerating COVID-19 therapeutic interventions and vaccines (ACTIV) trials |
A public-private partnership in the United States led by the National Institutes of Health (NIH) and coordinated by the Foundation for the National Institutes of Health (FNIH) to develop a research strategy for prioritizing and speeding development of the most promising COVID-19 vaccines and treatments. Some trials including STRIVE platform are ongoing under this project. |
Document, trial website, protocol | 2020 | NIH 2020 STRIVE 2022 |
| Randomised evaluation of COVID-19 therapy (RECOVERY) | An adaptive platform clinical trial design to accelerate the process of identifying effective treatments for COVID-19 patients who enter the United Kingdom’s National Health Service hospitals. Sponsored by University of Oxford. | Document and video, trial website, protocol, statistical plan, consent form, manual, patient engagement material, and presentation | 2020 | University of Oxford 2020a |
| Platform randomised trial of interventions against COVID-19 In older people (PRINCIPLE) | This study is a platform, randomised controlled trial in primary care that can rapidly test low-risk treatments for people at higher risk of complications from COVID-19. The intent is to establish an on-going trial infrastructure within a master protocol. Sponsored by University of Oxford. | Document, trial website. protocol, guidance, and patient information sheet | 2020 | University of Oxford 2020b |
| An open-label, multicentre, randomised, adaptive platform trial of the safety and efficacy of several therapies, including antiviral therapies, versus control in mild/moderate cases of COVID-19 (ANTICOV) | ANTICOV is an open-label, randomised, comparative, adaptive platform trial that will test the safety and efficacy of treatments in 2,000 to 3,000 mild-to-moderate COVID-19 patients. ANTICOV aims to identify early treatments that can prevent progression of COVID-19 to severe disease and potentially limit transmission. Sponsored by Drug for Neglected Diseases Initiative and others. | Document and video, trial website, protocol, patient information, informed consent form, and standard operating procedure | 2020 | Drugs for Neglected Diseases Initiative et al. 2020 |
| EU-SolidAct – European DisCoVeRy for Solidarity: An Adaptive Pandemic and Emerging Infection Platform Trial. | The EU-SolidAct trial is a part of EU-response, a pan-European research project involved with rapid and coordinated investigation of new and repurposed medication to treat Covid-19 during the ongoing pandemic. EU-SolidAct is an Adaptive Platform Trial sponsored by Oslo University Hospital, Norway. The master protocol is developed for evaluating treatments in hospitalized patients with COVID-19, the disease caused by the COVID 19. | Document, trial website, tutorial video, protocol and consent form, standard operating procedure | 2020 | EU -SolidAct |
| A Multicenter, Prospective, Adaptive, Double-blind, Randomized, Placebo-controlled Study to Evaluate the Effect of Interferon Lambda 1A, Fluvoxamina + Budesonida, Fluoxetina + Budesonida in Mild COVID-19 and High Risk of Complications(TOGERTHER) trial | The TOGETHER project is an international collaboration sponsored by Mcmaster University and PUC minas. The TOGETHER Clinical Trial was initiated in June of 2020, as an international collaboration in response to the worsening pandemic of SARS-CoV-2 / COVID-19. Utilizing an adaptive platform trial design, the international researchers and teams in Brazil, Canada, Australia, and the United States sought to identify evidence-informed therapeutics, through the re-purposing of existing medications. To identify effective repurposed therapies to prevent the disease progression of COVID-19.This international trial is financially supported by private sector philanthropic agencies. | Trial website, protocol papers, statistical plan. | 2020 | TOGETHER |
| Protocol for the DisCoVeRy trial: multicentre, adaptive, randomised trial of the safety and efficacy of treatments for COVID-19 in hospitalised adults | This is a protocol paper for Discovery trial, a multi-arm randomised controlled trial for COVID-19, developed by the French national institute for Health and Medical Research (Inserm). The objective of the trial is to evaluate the clinical efficacy and safety of different investigational re-purposed therapeutics relative to Standard of care in patients hospitalised with COVID-19. | Document, journal article, protocol paper | 2020 | Ader 2020 |
| A randomised, embedded, multi-factorial, adaptive platform trial for community-acquired pneumonia (REMAP-CAP) | REMAP-CAP is a randomised, embedded, multifactorial, adaptive platform trial for community-acquired pneumonia. In addition, REMAP-CAP provides and adaptive research platform for evaluation of multiple treatment modalities in the event of a respiratory pandemic resulting in critical illness. Sponsored by University Medical Center Utrecht. | Document, video, and podcast, trial website, protocol, statistical plan, and statistical model | 2015 | University Medical Center Utrecht 2015 |
| Molecular selection of therapy in colorectal cancer: a molecularly stratified randomised controlled trial programme (FOCUS4) | FOCUS4 is an adaptive, molecularly stratified trial which aims to improve the efficiency with which new novel agents can be tested in the colorectal cancer setting. It has a multi-arm, multi-stage design with a master protocol. Sponsored by University College London. | Document, trial website, protocol and patient information sheet | 2013 | University College London 2013 |
| Investigation of serial studies to predict your therapeutic response with imaging and molecular analysis (I-SPY) trials | Adaptive platform phase 2 trials family with a Bayesian design to investigate treatments for breast cancer or COVID-19. Sponsored by QuantumLeap Healthcare Collaborative. | Document and video, trial website, presentation | 2010 | QuantumLeap Healthcare Collaborative 2010 |
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