Clinical Trials in Pancreatitis: Opportunities and Challenges in the Design and Conduct of Patient-Focused Clinical Trials in Recurrent Acute and Chronic Pancreatitis

Abstract

Recurrent acute pancreatitis and chronic pancreatitis represent high morbidity diseases, which are frequently associated with chronic abdominal pain, pancreatic insufficiencies, and reduced quality of life. Currently, there are no therapies to reverse or delay disease progression, and clinical trials are needed to investigate potential interventions that would address this important gap. This conference report provides details regarding information shared during a National Institute of Diabetes and Digestive and Kidney Diseases-sponsored workshop on Clinical Trials in Pan-creatitis that sought to clearly delineate the current gaps and opportunities related to the design and conduct of patient-focused trials in recurrent acute pancreatitis and chronic pancreatitis. Key stakeholders including representatives from patient advocacy organizations, physician investigators (including clinical trialists), the US Food and Drug Administration, and the National Institutes of Health convened to discuss challenges and opportunities with particular emphasis on lessons learned from trials in participants with other painful conditions, as well as the value of incorporating the patient perspective throughout all stages of trials.

Interventions for Pancreatitis—New Approaches, Knowledge Gaps, and Research Opportunities

“…As with all clinical trials in pancreatitis, the development and implementation of a core outcome set are desirable to reduce heterogeneity and produce higher-quality evidence. 31 In the absence of such guidance, investigators are encouraged to adopt standard definitions from nutrition societies. For example, the Global Leadership Initiative on Malnutrition criteria were developed by an international consortium and represent a consensus for the diagnosis of malnutrition.…”

“…Several important considerations must be accounted for when approaching study design for nutrition studies in pancreatitis. As with all clinical trials in pancreatitis, the development and implementation of a core outcome set are desirable to reduce heterogeneity and produce higher-quality evidence 31 . In the absence of such guidance, investigators are encouraged to adopt standard definitions from nutrition societies.…”

Short-Term Soy Bread Intervention Leads to a Dose-Response Increase in Urinary Isoflavone Metabolites and Satiety in Chronic Pancreatitis

“…Moreover, complementary food processing techniques can enhance bioavailability of bioactive compounds or utilize food structures to modulate their delivery to localized sites along the gastrointestinal tract [38,60]. While nutritional intervention studies represent a great opportunity in CP, there are several challenges, including the lack of standardized outcome assessments to determine clinical efficacy [61]. Nevertheless, considering the absence of approved medical therapies for CP and the significant nutritional abnormalities, this is a key opportunity for future investigations.…”