Platform trials are adaptive clinical trials in which patients with a single disease are randomly allocated to different treatments based on an algorithm to investigate whether various agents have clinical benefit. Adaptive platform trials, which can study multiple therapeutic interventions in a disease in a perpetual manner, offer the potential to improve the efficiency of drug development. Currently there are three adaptive platform clinical trials within ACTIV-4 that aim to prevent, treat, and address COVID-19-associated coagulopathy (CAC), or clotting, as well as help understand the effects of CAC across three patient populations: inpatient, outpatient, and convalescent. Introduction by Himisha Beltran, MD (Harvard: Dana-Farber Cancer Institute) Laura Esserman, MD, MBA. Author Correction: Adaptive platform trials: definition, design, conduct and reporting considerations. Cooperation and coordination between different EU projects is essential to tackle COVID-19. Well conducted, adaptive platform trials utilising a multi-arm multi-stage (MAMS) approach provide a solution to overcome limitations of traditional designs. Adaptive Platform Trials The Clinical Trial of the Future Case Study is included in the Harvard Business Review Case Study. Characteristics of Adaptive Trials • Clarity of goals – E.g., proof of concept vs. identification of dose to carry forward vs. confirmation of benefit – A statistically significant p value is not a goal • Frequent “looks” at the data and data-driven modification of the trial • Adaptive “by design” Platform trials referred to as Multi-Arm, Multi-Stage (MAMS) design trials evaluate several interventions against a common control group and can be perpetual. In July 2017, Dr. Brian M. Alexander, president and CEO of the AGILE Research Foundation, was preparing to launch a new type of clinical trial-an adaptive platform trial-to study potential therapies for glioblastoma (GBM), an aggressive form of brain cancer. Only a handful of the almost 4,000 trials on COVID-19 registered to date are following an adaptive, platform approach, therefore showing this is still a work in progress, and that is why the remit of EU-PEARL is so relevant.” Ms Van Dessel adds, “In a way, Solidarity trial is a perfect case study, because it demonstrates that it can be done. The broad objective of this REMAP is, over time, to determine and continuously update the optimal set of treatments for community-acquired pneumonia. The A case centers on Dr. Brian Alexander's efforts to launch an adaptive platform trial for studying potential therapies for glioblastoma (GBM), an aggressive form of brain cancer. This is the part II of the article that describes confirmatory adaptive platform trial (CAPT) design for COVID-19 treatment development. Unlike traditional RCTs, adaptive platform trials maintaine d several “treatment arms” to simultaneously and dynamically study the effects of multiple unique drugs for a given disease Alexander believed that adaptive platform trials had potential to usher in a new generation of effective treatments for GBM. Adaptive platform trials: definition, design, conduct and reporting considerations. The multi-arm element allows multiple different treatments to be investigated simultaneously against a shared, standard-of-care control arm. • Proposals with the … Bayesian adaptive platform trials hold the promise of addressing clinical uncertainties and informing treatment. 7:55 AM – 8:45 AM. Adaptive platform trials: definition, design, conduct and reporting considerations. Pancreatic Cancer Action Network partners with FibroGen to bring new experimental treatment arm to adaptive clinical trial, Precision Promise. Adaptive platform trials: definition, design, conduct and reporting considerations Abstract. Moreover, regulatory authorities appear to be increasingly amenable to platform trial designs. 14,353 Patient randomisations. The adaptive design of the protocol allows different blood thinners to be started, stopped or combined during the study in response to emerging trial data. 4. … 7,977 Total patients. Case Description of Adaptive Platform Trials: The Clinical Trial of the Future? The guidance describes important principles for designing, conducting, and reporting the results from an adaptive clinical trial. What are platform trials? Given the elusiveness of effective treatments, there is a strong case for platform trials when multiple potential agents are available for study. 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. This approach accelerates the timeline for testing different agents without compromising safety. The use of “response adaptive randomization” allocates more … Adaptive Platform Trials • Master Protocol • Focus is on the Disease – What is the best treatment for a unique patient with this disease? They also used statistical techniques to allow more patients to access promising therapies. Widely hailed as the future of phase II drug development, the adaptive I-SPY 2 platform trial has set a new benchmark for efficiency, through innovation in trial design and clinical operations. These designs help to further accept additions or exclusions of new therapies or patient populations during the clinical trial. author = "{The Adaptive Platform Trials Coalition} and Angus, {Derek C.} and Alexander, {Brian M.} and Scott Berry and Meredith Buxton and Roger Lewis and Melissa Paoloni and Webb, {Steven A.R.} Adaptive platform trials have the potential to evaluate treatments quickly (to determine early superiority or futility) and the flexibility to add or remove treatments during the ongoing study. I-SPY COVID TRIAL: An Adaptive Platform Trial to Reduce Mortality and Ventilator Requirements for Critically Ill Patients See the Full EUA Prescribing Information for complete dosage, administration, and preparation instructions. Remdesivir is available as a concentrated solution. Product #: 620001. Session 2: Keynote II: Adaptive Platform Trials: Scalable from Breast Cancer to COVID. For the past three years, Alexander had been working closely with a group of like-minded oncologists, statisticians, and clinical trial strategists to design an adaptive platform trial for GBM in the hopes of identifying effective therapies more quickly. By mid-2017, Alexander and his colleagues had completed a master protocol for the trial. Our ctDNA-guided adaptive platform trials will accelerate clinical development toward further precision oncology in the field of adjuvant therapy. Using modeling and response adaptive randomization, they allow for the evaluation of multiple treatments across different management domains, and progressive improvement in patient outcomes throughout the course of the trial. and Steven Arnold and Anna Barker and Berry, {Donald A.} What is an adaptive clinical trial? The multi-arm element allows multiple different treatments to be investigated simultaneously against a shared, standard-of-care control arm. To determine the most effective antithrombotic strategy for increasing the number of days free of organ support and reducing death. Such trials involve making adaptations to accruing data and conducting frequent interim analyses. Master protocols and adaptive platform trials (APTs) have been proposed as attractive solutions to efficiently address multiple therapeutic and biomarker hypotheses. I-SPY COVID TRIAL: An Adaptive Platform Trial to Reduce Mortality and Ventilator Requirements for Critically Ill Patients Study HIC# : 2000028816 Last Updated : 07/15/2021 ... clinical trial platform … An International Multi-Centre Randomised Adaptive Platform Clinical Trial to Assess the Clinical, Virological and Immunological Outcomes in Patients with SARS-CoV-2 Infection (COVID-19). Multiple interventions can be evaluated at the same time, and study arms can be staggered. The upfront fixed cost for developing a trial infrastructure is most commonly only amortized over the relatively short life of a single clinical trial. Additional copies are available from: Office of Communications, Division of Drug Information Adaptive platform trials have attracted major interests, particularly in the field of oncology, for their ability to evaluate multiple interventions using a common control and the flexibility of adding new interventions during the trial. In this phase 2 platform design, agents will be identified with a signal suggesting a big impact on reducing mortality and the need for, as well as duration, of mechanical ventilation. Therefore, it is necessary to touch HBR fundamentals before starting the Adaptive Platform Trials The Clinical Trial of the Future case analysis. The need for efficient trials 4 The need for efficient trials Collier et al. This case provides an update to the (A) case, which introduces students to adaptive platform trials, an ambitious, more efficient type of clinical trial that increases access to therapies. Read More > Cooperation and coordination between different EU projects is essential to tackle COVID-19. , Brown et al. 7,068 Patients with suspected or proven COVID-19. To familiarize yourself with the platform and concept of adaptive learning, you will be invited to try a course from Area9’s ready-to-use curated collection. Adaptive platform trials, by contrast, facilitated simultaneously studying multiple therapies for a given disease and promised a number of efficiency improvements. We describe the methodological foundation of adaptive … Official Study Title: Adaptive Platform Treatment Trial for Outpatients With COVID-19 (Adapt Out COVID or ACTIV-2) Drug studies often look at the effect one or two drugs have on a medical condition, and involve one company. 5 A platform trial is a type of adaptive trial designed to evaluate multiple treatments efficiently. • Approach 2: Platform trial where the 2ndcompound is added to the platform • Assumptions: –Compound 2 is expected to be available about 25-30 months after the first one –Platform is more difficult to get started, approx. Incorporating adaptive elements into platform trials enables optimized assessment of multiple therapies and quicker decisions about which products are promising and merit further evaluation and which do not. Adaptive Designs for Clinical Trials of Drugs and Biologics . The right time. • Typical Innovations – Multiple Interventions, staggered entry – Adaptations to accruing data – requent interim analyses (dont wait for the end of trial!) Pulmonary REMAP-CAP is a randomised, embedded, multifactorial, adaptive platform trial for community-acquired pneumonia. They are adaptive in that they incorporate rules for … The I-SPY TRIALs and Adaptive Platform Trials: Scalable from Breast Cancer to COVID" I nvestigation of S erial studies to P redict Y our T herapeutic R esponse with biomarker I maging, integration and A daptive L earning. Nat Rev Drug Discov. A Bayesian adaptive design is proposed for a clinical trial in Duchenne muscular dystrophy. A Randomised, Embedded, Multi-factorial, Adaptive Platform Trial for Community-Acquired Pneumonia. The Adaptive Platform Trials Coalition. The limitations to traditional RCTs are that they can be slow to answer questions and … The goal of this project is to rapidly screen promising agents, in the setting of an adaptive platform trial, for treatment of critically ill COVID-19 patients. and Talisa et al. I-SPY Platform. The ‘A’ and the ‘P’—it was designed to be an adaptive platform,” says Anthony Gordon, chair in anesthesia and critical care at Imperial College and the UK chief investigator on the trial. The I-SPY Trials For Patients. Platform trial design strategies can be applied to the problem of finding the best treatment strategy for patients suffering from posttraumatic hemorrhagic shock. Cystic fibrosis (CF) is a genetic disease typically characterized by progressive lung damage and premature mortality. Confirmatory Adaptive Platform Trial (CAPT) Design Both ACTT and CATCO can be formulated according to the confirmatory adaptive platform (CAPT) design with … Currently there are three adaptive platform clinical trials within ACTIV-4 that aim to prevent, treat, and address COVID-19-associated coagulopathy (CAC), or clotting, as well as help understand the effects of CAC across three patient populations: inpatient, outpatient, and convalescent. Bayesian adaptive platform trials hold the promise of addressing clinical uncertainties and informing treatment. Adaptive Platform Trials Randomized controlled trials (RCTs) are the gold-standard method for generating evidence to show benefits of new therapies or interventions. Case Study . For this reason, RECOVER shares a joint coordination mechanism with a new, ambitious EU-funded research initiative, EU-RESPONSE. 1. An adaptive clinical trial is a clinical trial that evaluates a medical device or treatment by observing participant outcomes (and possibly other measures, such as side-effects) on a prescribed schedule, and modifying parameters of the trial protocol in accord with those observations. Adopting cloud leads businesses to connect their employees with customers, vendors, and tools to serve the customer expectations and adapt to changing markets and business conditions amid crises. A platform trial is an adaptive clinical trial in which multiple treatments for the same disease can be tested simultaneously. Coordination of European COVID-19 Adaptive Platform Trials. Using modeling and response adaptive randomization, they allow for the evaluation of multiple treatments across different management domains, and progressive improvement in patient outcomes throughout the course of the trial. Researchers, clinicians, policymakers and patients are increasingly interested in questions about therapeutic interventions that are difficult or costly to answer with traditional, free-standing, parallel-group randomized controlled trials (RCTs) Year: 2019. Well conducted, adaptive platform trials utilising a multi-arm multi-stage (MAMS) approach provide a solution to overcome limitations of traditional designs. The ‘A’ and the ‘P’—it was designed to be an adaptive platform,” says Anthony Gordon, chair in anesthesia and critical care at Imperial College and the UK chief investigator on the trial. The right patient. … Researchers, clinicians, policymakers and patients are increasingly interested in questions about therapeutic interventions that are difficult or costly to answer with traditional, free-standing, parallel-group randomized controlled trials (RCTs) Year: 2019. They also used statistical techniques to allow more patients access to promising therapies. The A case centers on Dr. Brian Alexander's efforts to launch an adaptive platform trial for studying potential therapies for glioblastoma (GBM), an aggressive form of brain cancer. This study will use a study design known as a REMAP, a Randomised, Embedded, Multifactorial, Adaptive Platform trial. Randomised controlled trials (RCTs) are the gold-standard method for generating evidence to show benefits of new therapies or interventions. Several Adaptive Platform Trials are now funded in various disease areas (see Angus et al. Case Study . The purpose of this study is to evaluate the effect of a range of interventions to improve outcome of patients admitted to intensive care with community-acquired pneumonia. Case Description of Adaptive Platform Trials: The Clinical Trial of the Future? For this reason, RECOVER shares a joint coordination mechanism with a new, ambitious EU-funded research initiative, EU-RESPONSE. Researchers, clinicians, policymakers and patients are increasingly interested in questions about therapeutic... Introduction. master protocols for adaptive platform trials to increase the clinical trial bandwidth across the country and rapidly scale up and maximize the number of host-directed trials that are conducted and which complement other ongoing trials. adaptive platform trials focus on a broader set of disease states and test multiple therapies simultaneously and sequentially.22,27,28 They are thus an experimental platform, rather than a series of experiments. and Bonten, {Marc J.M.} CMAJ, 2009; 180:277–8. Platform trials, also referred to as multi-arm, multi-stage (MAMS) design trials [ 7, 8, 9, 10 ], are trials that evaluate several interventions against a … 2019 Oct;18(10):808. doi: 10.1038/s41573-019-0045-0. Adaptive and platform studies differ (a study can be adaptive while testing a single therapy) but are highly complementary. In this phase 2 platform design, agents will be identified with a signal suggesting a big impact on reducing mortality and the need for, as well as duration, of mechanical ventilation. ACTIV-4A is a randomized, open label, adaptive platform trial to compare the effectiveness of antithrombotic strategies for prevention of adverse outcomes in COVID-19 positive inpatients. Adaptive platform trials (APTs) are a newer alternative to traditionally designed clinical trials. Platform trial design strategies can be applied to the problem of finding the best treatment strategy for patients suffering from posttraumatic hemorrhagic shock. Use of adaptive features in trial design ‐ Response adaptive randomization ‐ Bayesian borrowing 5. Primary Objectives. Platform trials are an emerging concept that is gaining in popularity, as it has the capability of saving costs through smart trial design. and Mary Brophy and Christopher Butler and Cloughesy, {Timothy F.} and Derde, {Lennie P.G.} Rather than testing individual interventions in a single homogeneous disease state and terminating when that task is complete, adaptive platform trials focus on a broader set of disease states and test multiple therapies simultaneously and sequentially (22, 27, 28). for a discussion). Awareness of these limitations has led to growing interest in novel trial designs, particularly those that use ‘master protocols’, which can be classified as basket, umbrella and adaptive platform trials (APTs) 2. Addition of docetaxel, zoledronic acid, or both to first-line long-term hormone therapy in prostate cancer (STAMPEDE): survival results from an adaptive, multiarm, multistage, platform randomised controlled trial Adaptive design, as defined by the U.S. FDA, is “a clinical trial design that allows for prospectively planned modifications to one or more aspects of the design based on accumulating data from subjects in the trial.” 1 Adaptive design characteristics include modifying an ongoing clinical trial in accordance with predetermined rules, based on data from interim analyses. Data from the agents tested in adaptive platform trials can quickly demonstrate whether a new therapy is better, worse or equivalent than standard of care treatments for this challenging disease. The trial was designed to demonstrate treatment efficacy on an ambulatory‐based clinical endpoint and to identify early success on a biomarker (dystrophin protein levels) that can serve as a basis for accelerated approval in the United States. In response, researchers have proposed new RCT designs such as adaptive platform trials (APTs), which are able to study multiple interventions in a disease or condition in a perpetual manner, with interventions entering and leaving the platform on the basis of a predefined decision algorithm. Now.™ They also used statistical techniques to allow more patients to access promising therapies. “Adaptive platform clinical trials can be a faster, less expensive, more efficient way to learn which treatments do and do not work for patients.” PanCAN’s own adaptive platform clinical trial, Precision Promise SM, will open at 14 Clinical Trial Consortium sites across the United States in fall 2019 through early 2020. – Response Adaptive Randomization (RAR) The goal of this project is to rapidly screen promising agents, in the setting of an adaptive platform trial, for treatment of critically ill COVID-19 patients. Adaptive Platform Trials. Trial patients are enrolled in a continuous manner via a common master protocol, with interventions entering and leaving the platform on the basis of a predefined decision algorithm. 48 Current or completed interventions in 14 Domains 327 APTs allow researchers to test multiple treatments for a medical condition at the same time. The (B) case provides an update and explores the strategies that Alexander is considering for sustainably financing this trial. Introduction. Guidance for Industry . This case provides an update to the (A) case, which introduces students to adaptive platform trials, an ambitious, more efficient type of clinical trial that increases access to therapies. This enables the coordination and complementarity of the two large European Adaptive Platform Trials and will set a precedent for future clinical trials, establishing structures and processes that can serve as a blueprint for coordination in other disease areas. The Indacaterol to Help Achieve New COPD Treatment Excellence (INHANCE) trial was an adaptive two-stage (i.e., phase 2–3), confirmatory, randomized clinical trial of … An adaptive platform is well suited to studying COVID-19 interventions. Participants are randomly assigned to either usual care or an intervention, where an intervention consists of the intervention active agent plus usual care. The A case centers on Dr. Brian Alexander's efforts to launch an adaptive platform trial for studying potential therapies for glioblastoma (GBM), an aggressive form of brain cancer. There is a need for more efficient evaluation of … The NSW Adaptive Platform Trials aim to deliver: rapid and efficient assessments of COVID-19 models of care and interventions; improved understanding of the natural history of the disease, the biological response and therapeutics options to deliver quality evidence for decision making at the health system and clinical levels Official Study Title: Adaptive Platform Treatment Trial for Outpatients With COVID-19 (Adapt Out COVID or ACTIV-2) Drug studies often look at the effect one or two drugs have on a medical condition, and involve one company. 12,808 Patient randomisations with suspected or proven COVID-19. randomized, placebo-controlled, adaptive platform, Proof-of-Concept clinical trial of multiple interventions for the secondary prevention of Alzheimer’s Dementia in subjects from Preclinical to Prodromal stages of Alzheimer’s Disease Scott Berry Barcelona Global Assembly May 2016 22 Suggestions for implementation 1. I-SPY 2 Trial Resources Funding & Support. I-SPY | The right drug. Coordination of European COVID-19 Adaptive Platform Trials. Adaptive platform trials, by contrast, facilitated simultaneously studying multiple therapies for a given disease and promised a number of efficiency improvements. In this interview, Allyson Gage, Chief Medical Officer at Cohen Veterans Bioscience (CVB) will discuss her perspectives on platform trial design. and Esserman, {Laura J.} They are thus an experimental platform, rather than a series of experiments. In a simple setting, we have explored the feasibility, efficiency, and flexibility of two subgroups of platform trials: multi-arm multi-stage and adaptive randomization trials. Adaptive platform trials, by contrast, facilitated simultaneously studying multiple therapies for a given disease and promised a number of efficiency improvements. In July 2017, Dr. Brian M. Alexander, president and CEO of the AGILE Research Foundation, was preparing to launch a new type of clinical trial-an adaptive platform trial-to study potential therapies for glioblastoma (GBM), an aggressive form of brain cancer. Examples of adaptive trials include traditional group-sequential trials, as well as trials incorporating reestimation of sample size or using variable randomization proportions (response-adaptive randomization). Adaptive platform trials, by contrast, facilitated simultaneously studying multiple therapies for a given disease and promised a number of efficiency improvements. They also used statistical techniques to allow more patients access to promising therapies. Manax is leading PanCAN’s efforts to launch Precision Promise. Adaptive platform trials focus on a specific disease, asking: What is the best treatment for a unique patient with this disease? 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