Abstract
Youth-onset type 2 diabetes (T2D) is increasingly recognized as a disorder with substantial risk for long-term metabolic, cardiovascular, and renal morbidity and mortality, as well as individual and societal burden. Recent studies suggest that the disorder differs from adult-onset T2D in a variety of ways and that there is an urgent need for an expanded set of treatment options. However, demographic, economic, and social challenges limit the number of eligible candidates for clinical trials in youth-onset T2D, and a growing number trials mandated by regulatory agencies have created a circumstance in which too many trials are chasing too few eligible participants. A solution to this situation will require novel approaches to clinical trial design incorporating collaboration among clinical investigators, pharmaceutical sponsors, and regulatory agencies. If successful, this approach could also serve as a model for clinical trials in other rare and understudied pediatric disorders.
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Conflicts of Interest
Philip Zeitler is a consultant and has participated in the design and monitoring of clinical trials for Daiichi-Sankyo, Bristol-Myers Squibb, and Merck.
Hubert S. Chou is an employee of Daichii-Sankyo Pharma Development and has stock in the company.
Kenneth C. Copeland is a consultant and has participated in the design and monitoring of clinical trials for Daiichi-Sankyo.
Mitchell Geffner is a consultant and has participated in the design and oversight of clinical trials for Daichii-Sankyo. Dr. Geffner is a consultant for Ipsen and on the advisory board for Merck-Serono, Novo Nordisk, Pfizer Inc, and Endo pharmaceuticals. He receives research support from Eli Lilly, Inc, Genentech, Inc, Novo Nordisk, Pfizer, Inc, and Versartis.
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This article does not contain any studies with human or animal subjects performed by any of the authors.
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This article is part of the Topical Collection on Pediatric Type 2 Diabetes
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Zeitler, P., Chou, H.S., Copeland, K.C. et al. Clinical Trials in Youth-Onset Type 2 Diabetes: Needs, Barriers, and Options. Curr Diab Rep 15, 28 (2015). https://doi.org/10.1007/s11892-015-0597-2
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DOI: https://doi.org/10.1007/s11892-015-0597-2