James D Guest1, John D Steeves2, M J Mulcahey3, Linda A T Jones4, Frank Rockhold5, Rϋediger Rupp6, John L K Kramer2, Steven Kirshblum7,8, Andrew Blight9, Daniel Lammertse10. 1. The Miami Project to Cure Paralysis, Department of Neurological Surgery, Miller School of Medicine, Miami, FL, USA. 2. International Collaboration on Repair Discoveries, University of British Columbia, Vancouver, BC, Canada. 3. Center for Outcomes and Measurement, College of Rehabilitation Sciences, Thomas Jefferson University, Philadelphia, PA, USA. Maryjane.mulcahey@jefferson.edu. 4. University of Colorado, Denver, CO, USA. 5. Duke Clinical Research Institute, Duke University Medical Center, Durham, NC, USA. 6. Spinal Cord Center, Heidelberg University Hospital, Heidelberg, Germany. 7. Kessler Institute for Rehabilitation, West Orange, NJ, USA. 8. Rutgers New Jersey Medical School, Newark, NJ, USA. 9. Acorda Therapeutics, Inc., Ardsley, NY, USA. 10. Craig Hospital, Englewood, CO, USA.
Abstract
STUDY DESIGN: Narrative review. PURPOSE: To provide an overview of adaptive trial designs, and describe how adaptive methods can address persistent challenges encountered by randomized controlled trials of people with spinal cord injury (SCI). RESULTS: With few exceptions, adaptive methodologies have not been incorporated into clinical trial designs of people with SCI. Adaptive methods provide an opportunity to address high study costs, slow recruitment, and excessive amount of time needed to carry out the trial. The availability of existing SCI registries are well poised to support modeling and simulation, both of which are used extensively in adaptive trial designs. Eight initiatives for immediate advancement of adaptive methods in SCI were identified. CONCLUSION: Although successfully applied in other fields, adaptive clinical trial designs in SCI clinical trial programs have been narrow in scope and few in number. Immediate application of several adaptive methods offers opportunity to improve efficiency of SCI trials. Concerted effort is needed by all stakeholders to advance adaptive clinical trial design methodology in SCI.
STUDY DESIGN: Narrative review. PURPOSE: To provide an overview of adaptive trial designs, and describe how adaptive methods can address persistent challenges encountered by randomized controlled trials of people with spinal cord injury (SCI). RESULTS: With few exceptions, adaptive methodologies have not been incorporated into clinical trial designs of people with SCI. Adaptive methods provide an opportunity to address high study costs, slow recruitment, and excessive amount of time needed to carry out the trial. The availability of existing SCI registries are well poised to support modeling and simulation, both of which are used extensively in adaptive trial designs. Eight initiatives for immediate advancement of adaptive methods in SCI were identified. CONCLUSION: Although successfully applied in other fields, adaptive clinical trial designs in SCI clinical trial programs have been narrow in scope and few in number. Immediate application of several adaptive methods offers opportunity to improve efficiency of SCI trials. Concerted effort is needed by all stakeholders to advance adaptive clinical trial design methodology in SCI.
Authors: Andrew R Blight; Jane Hsieh; Armin Curt; James W Fawcett; James D Guest; Naomi Kleitman; Shekar N Kurpad; Brian K Kwon; Daniel P Lammertse; Norbert Weidner; John D Steeves Journal: Spinal Cord Date: 2019-04-08 Impact factor: 2.772
Authors: J D Steeves; D Lammertse; A Curt; J W Fawcett; M H Tuszynski; J F Ditunno; P H Ellaway; M G Fehlings; J D Guest; N Kleitman; P F Bartlett; A R Blight; V Dietz; B H Dobkin; R Grossman; D Short; M Nakamura; W P Coleman; M Gaviria; A Privat Journal: Spinal Cord Date: 2006-12-19 Impact factor: 2.772
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