Master Protocols to Study Multiple Therapies, Multiple Diseases, or Both

Woodcock, Janet; LaVange, Lisa M.

doi:10.1056/nejmra1510062

Cited by 771 publications

(729 citation statements)

References 29 publications

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“…A master protocol trial uses a common system for patient selection, logistics, templates, and data management [22]. Histologic and hematologic specimens of patients enrolled in master protocol trials are also measured and analyzed using a common basic system (e.g., next generating sequencer and immunohistochemistry) to collect coherent molecular marker data.…”

Section: Master Protocol Trialmentioning

confidence: 99%

“…The common definitions of each trial type based on literature [22,23] are shown in Table 1. However, as pointed out by Renfro and Sargent [23], the definitions of each trial type are not standardized [10,20,[35], [36], [37]], with possible overlaps between them that should be noted.…”

Section: Basket Umbrella and Platform Trialsmentioning

confidence: 99%

“…For example, the NCI-MATCH trial, which will be mentioned in a later section, is a type that has aspects of both basket and umbrella trials. In this article, we will organize the trial types by definitions given in recent works [[21], [22], [23]]. …”

Section: Basket Umbrella and Platform Trialsmentioning

confidence: 99%

“…This article begins with an overview of the history from biomarker-based trial design to master protocol trial, and subsequently summarizes clinical trials using master protocols in oncology based on recent general theories of master protocol design [[21], [22], [23]]. We introduce several examples of master protocol trials along with their classifications according to previous studies.…”

Section: Introductionmentioning

confidence: 99%

See 3 more Smart Citations

Master protocol trials in oncology: Review and new trial designs

Hirakawa

Asano

Sato

et al. 2018

Contemporary Clinical Trials Communications

103

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In oncology, next generation sequencing and comprehensive genomic profiling have enabled the detailed classification of tumors using molecular biology. However, it is unrealistic to conduct phase I–III trials according to each sub-population based on patient molecular subtypes. Common protocols that assess the combination of several molecular markers and their targeted therapies by means of multiple sub-studies are required. These protocols are called “master protocols,” and are drawing attention as a next-generation clinical trial design. Recently, several reviews of clinical trials based on the master protocol design have been published, but their definitions of these such trials, including basket, umbrella, and platform trials, were not consistent. Concurrently, the acceleration of the development of new statistical designs for master protocol trials has been underway. This article provides an overview of recent reviews for master protocols, including their statistical design methodologies in Oncology. We also introduce several examples of previous and on-going master protocol trials along with their classifications by some recent studies.

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Section: Master Protocol Trialmentioning

confidence: 99%

Section: Basket Umbrella and Platform Trialsmentioning

confidence: 99%

Section: Basket Umbrella and Platform Trialsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Master protocol trials in oncology: Review and new trial designs

Hirakawa

Asano

Sato

et al. 2018

Contemporary Clinical Trials Communications

103

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“…The use of “basket” or “umbrella” trials, or more commonly called “platform” trials involves assessing the effects of multiple interventions on one or more conditions, using modern adaptive designs and statistical approaches, including Bayesian analyses (Table 1). 22, 23 A common feature of basket, umbrella and platform trials is the use of a Master Trial Protocol which defines overarching clinical trial elements for the various individual trials conducted within the platform with relative minor trial design differences depending on unique drug characteristics. The master trial protocol enables sharing of trial documents and procedures across trials supporting trial consistency and efficiency.…”

Section: Advances In Clinical Trial Designs To Foster Drug Developmentmentioning

confidence: 99%

New clinical trial designs for establishing drug efficacy and safety in a precision medicine era

Heerspink

List

Perkovic

2018

Diabetes Obesity Metabolism

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Despite advances in pharmacotherapy, diabetic kidney disease (DKD) remains associated with a high burden of micro‐ and macrovascular complications often leading to premature mortality. New therapies are highly desirable to mitigate the burden of this disease. However, there are a number of barriers that hamper drug development in DKD. These include, amongst others, the lengthy and complex clinical trials required to prove drug efficacy and safety, inefficiencies in clinical trial conduct, and the high costs associated with these development programs. In this review a number of aspects are discussed, aiming to identify opportunities to transform and innovate drug development for DKD. Many clinical trials in DKD, as well as in other areas, face difficulties in timely and efficient enrolment of participants. To address this issue a network of sites should be created that are continuously recruiting individuals with DKD and collecting crucial information that can be used to understand prognosis and prognostic factors, and more importantly to serve as a pool of participants for recruitment to randomized trials. Second, the current clinical endpoints are late events in the progression of DKD. Endpoints based on lesser declines in estimated glomerular filtration rate (eGFR) or changes in albuminuria can shorten follow‐up and/or lead to smaller and cheaper trials. Enrichment by enrolling clinical trial populations based on biomarker profiles is another approach that may facilitate clinical trial efficiency and conduct. Biomarkers can be used to individualize treatment by targeting populations more likely to respond leading to smaller and more efficient trials. Finally, using new trial design such as basket, umbrella or more broadly platform trials to assess a number of therapies simultaneously offers the potential to transform the drug development process in DKD. There are a number of opportunities to transform development approaches for new therapies for DKD. Platform trials along with appropriate biomarker‐based enrichment strategies offer the possibility to foster drug development in a precision medicine era.

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Modernizing Clinical Trials for Patients With Cancer

Sharpless

Doroshow

2019

JAMA

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Clinical trials involve evaluating and validating new therapies in humans and represent the fundamental means of making progress in cancer care. The oncology community has made significant improvements in treating most cancers and, in some cases, has developed cures, such as for testicular cancer, Hodgkin disease, and acute lymphocytic leukemia. In an even larger number of cancers, researchers and clinicians have succeeded in making cancer a chronic disease that people die with rather than of. Each of these therapeutic discoveries represents the results of clinical trials.Many of these advances were made possible in large part through the Clinical Trials Program at the National Cancer Institute (NCI). This program began in 1955, 1 and since then has changed substantially in its approach to the design, coordination, and implementation of clinical trials. The past 10 years, in particular, have seen a transformational reworking of the NCI's clinical trials infrastructure. 2 The NCI National Clinical Trials Network (NCTN) now includes more than 3000 study sites across the United States and Canada and each year enrolls more than 20 000 children and adults with all types of cancer in clinical trials. 3 The NCTN portfolio includes studies across the cancer research continuum, from first-in-human trials of new agents, to evaluations of new diagnostic and therapeutic techniques, to trials evaluating cancer prevention and symptom management. The infrastructure provides a broad range of support services, including centralized institutional review boards; clinical trials monitoring, data acquisition, and management systems; tissue banks; and quality-control centers for imaging and radiation therapy.

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Master Protocols to Study Multiple Therapies, Multiple Diseases, or Both

Cited by 771 publications

References 29 publications

Master protocol trials in oncology: Review and new trial designs

Master protocol trials in oncology: Review and new trial designs

New clinical trial designs for establishing drug efficacy and safety in a precision medicine era

Modernizing Clinical Trials for Patients With Cancer

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