Journal article
Optimal Design of Clinical Trials Involving Persons with Disorders of Consciousness
Neurocritical care, Vol.40(1), pp.74-80
02/2024
DOI: 10.1007/s12028-023-01813-2
PMID: 37535178
Abstract
Background: Limited data exist regarding the optimal clinical trial design for studies involving persons with disorders of consciousness (DoC), and only a few therapies have been tested in high-quality clinical trials. To address this, the Curing Coma Campaign Clinical Trial Working Group performed a gap analysis on the current state of clinical trials in DoC to identify the optimal clinical design for studies involving persons with DoC. Methods: The Curing Coma Campaign Clinical Trial Working Group was divided into three subgroups to (1) review clinical trials involving persons with DoC, (2) identify unique challenges in the design of clinical trials involving persons with DoC, and (3) recommend optimal clinical trial designs for DoC. Results: There were 3055 studies screened, and 66 were included in this review. Several knowledge gaps and unique challenges were identified. There is a lack of high-quality clinical trials, and most data regarding patients with DoC are based on observational studies focusing on patients with traumatic brain injury and cardiac arrest. There is a lack of a structured long-term outcome assessment with significant heterogeneity in the methodology, definitions of outcomes, and conduct of studies, especially for long-term follow-up. Another major barrier to conducting clinical trials is the lack of resources, especially in low-income countries. Based on the available data, we recommend incorporating trial designs that use master protocols, sequential multiple assessment randomized trials, and comparative effectiveness research. Adaptive platform trials using a multiarm, multistage approach offer substantial advantages and should make use of biomarkers to assess treatment responses to increase trial efficiency. Finally, sound infrastructure and international collaboration are essential to facilitate the conduct of trials in patients with DoC. Conclusions: Conduct of trials in patients with DoC should make use of master protocols and adaptive design and establish international registries incorporating standardized assessment tools. This will allow the establishment of evidence-based practice recommendations and decrease variations in care.
Details
- Title: Subtitle
- Optimal Design of Clinical Trials Involving Persons with Disorders of Consciousness
- Creators
- Sung-Min Cho - Johns Hopkins UniversityChiara Robba - Ospedale Policlinico San MartinoMichael N Diringer - Washington University in St. LouisDaniel F Hanley - Johns Hopkins MedicineJ. Claude Hemphill - University of California, San FranciscoJanneke Horn - University of AmsterdamAriane Lewis - New York UniversitySarah L Livesay - Rush UniversityDavid Menon - University of CambridgeTarek Sharshar - Université Paris CitéRobert D Stevens - Johns Hopkins MedicineJames Torner - University of IowaPaul M Vespa - University of California, Los AngelesWendy C Ziai - Johns Hopkins UniversityMarcus Spann - Johns Hopkins UniversityRaimund Helbok - Innsbruck Medical UniversityJose I Suarez - Johns Hopkins University
- Resource Type
- Journal article
- Publication Details
- Neurocritical care, Vol.40(1), pp.74-80
- DOI
- 10.1007/s12028-023-01813-2
- PMID
- 37535178
- NLM abbreviation
- Neurocrit Care
- ISSN
- 1541-6933
- eISSN
- 1556-0961
- Publisher
- Springer
- Grant note
- National Heart, Lung, and Blood Institute: 1K23HL157610
S-MC is funded by National Heart, Lung, and Blood Institute grant 1K23HL157610.
- Language
- English
- Date published
- 02/2024
- Academic Unit
- Neurology; Epidemiology; Injury Prevention Research Center; Neurosurgery
- Record Identifier
- 9984800201602771
Metrics
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