ReviewPloS one2016
Biomarker-Guided Adaptive Trial Designs in Phase II and Phase III: A Methodological Review.
Review in PloS one, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 60 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
60 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Long-term effects on clinical event, mental health, and related outcomes of CPAP for obstructive sleep apnea: a systematic review.Journal of clinical sleep medicine : JCSM : official publication of the American Academy of Sleep Medicine · 2024Pooled it
- Design and analysis of umbrella trials: Where do we stand?Frontiers in medicine · 2022Pooled it
- Serum exocrine pancreas enzymes are biomarkers of immunotherapy response in new-onset type 1 diabetes.Frontiers in endocrinology · 2024Trial
- Hyperforin as a Multifunctional Specialized Metabolite of Natural Origin for Chronic Diseases: From Molecular Mechanisms to Clinical Translation.Current issues in molecular biology · 2026Review
- Operationalizing Alzheimer's disease trials in the era of targeted therapies.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2026Review
- Reprogramming Neuroinflammation: Mitochondrial Targets and Immune Checkpoint Inhibitors in Alzheimer's Disease.Molecular neurobiology · 2026Review
- Five tenets for advancing evidence-based precision medicine.Nature medicine · 2026Review
- Exploratory Cytokine and Bone-Marker Patterns in a Proteoglycan-Induced Spondyloarthritis Mouse Model: Th1/Th2 Strain Comparison and TLR2/3/4 Knockout Readouts.International journal of molecular sciences · 2026Article
- Nitric Oxide Signaling in Cardiovascular Physiology and Pathology: Mechanisms, Dysregulation, and Therapeutic Frontiers.International journal of molecular sciences · 2026Review
- The roles of biomarkers in Alzheimer's disease clinical trials.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026Review
- Ozone therapy in femoral head necrosis: a perspective on biomarker-guided treatment rationalization and mechanistic exploration.Frontiers in medicine · 2026Article
- Neurovascular Dysfunction and Glymphatic Impairment: An Unexplored Therapeutic Frontier in Neurodegeneration.International journal of molecular sciences · 2025Review
- Adaptive Biomarker-Based Design for Early Phase Clinical Trials.Statistics in medicine · 2025Article
- Biomarker-guided adaptive enrichment design with threshold detection for clinical trials with time-to-event outcome.Journal of biopharmaceutical statistics · 2025Article
- Interpreting results from biomarker-guided clinical trials: avoiding "negative" or "failed" terminology.BMC medicine · 2025Article
- Characterization of the Biological Variability of the Angiome Biomarkers over Time in Healthy Participants.Cancer epidemiology, biomarkers & prevention : a publication of the American Association for Cancer Research, cosponsored by the American Society of Preventive Oncology · 2025Article
- The promise of PD1/PDL1 targeted immunotherapy in locally advanced cervical cancer: a game-changer for patients outcome?Frontiers in immunology · 2025Review
- Two-stage Adaptive Enrichment Designs with Survival Outcomes and Adjustment for Misclassification in Predictive Biomarkers.Statistics in biopharmaceutical research · 2025Article
- The PERMIT guidelines for designing and implementing all stages of personalised medicine research.Scientific reports · 2024Article
- Testing for a treatment effect in a selected subgroup.Statistical methods in medical research · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
backgroundPersonalized medicine is a growing area of research which aims to tailor the treatment given to a patient according to one or more personal characteristics. These characteristics can be demographic such as age or gender, or biological such as a genetic or other biomarker. Prior to utilizing a patient's biomarker information in clinical practice, robust testing in terms of analytical validity, clinical validity and clinical utility is necessary. A number of clinical trial designs have been proposed for testing a biomarker's clinical utility, including Phase II and Phase III clinical trials which aim to test the effectiveness of a biomarker-guided approach to treatment; these designs can be broadly classified into adaptive and non-adaptive. While adaptive designs allow planned modifications based on accumulating information during a trial, non-adaptive designs are typically simpler but less flexible. METHODS AND
findingsWe have undertaken a comprehensive review of biomarker-guided adaptive trial designs proposed in the past decade. We have identified eight distinct biomarker-guided adaptive designs and nine variations from 107 studies. Substantial variability has been observed in terms of how trial designs are described and particularly in the terminology used by different authors. We have graphically displayed the current biomarker-guided adaptive trial designs and summarised the characteristics of each design.
conclusionsOur in-depth overview provides future researchers with clarity in definition, methodology and terminology for biomarker-guided adaptive trial designs.
Indexed as
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.