Common Study Designs of Nutrition Clinical Trials: Review of the Basic Elements and the Pros and Cons

Abstract

Introduction: Nutrition Clinical Trials (NCTs) are pivotal in establishing causal links between nutritional interventions and chronic diseases. This review comprehensively examines prevalent clinical trial designs, emphasizing their strengths and limitations. The goal is to provide insights into the selection and optimization of these designs for dietary intervention studies. Methods: Various study designs in NCTs are explored, including quasi-experimental designs, double-blind randomized placebo-controlled trials for nutrient/functional foods supplementation, community-based lifestyle interventions, pragmatic nutrition interventions, and field trial projects. The characteristics, advantages, and challenges of each design are discussed. Real examples are presented to illustrate how these designs can be tailored and optimized for dietary intervention studies. Results: Parallel randomized clinical trials are acknowledged as the gold standard, despite requiring substantial sample sizes and having inherent limitations. Cross-over NCTs emerge as valuable for assessing temporary treatment effects while mitigating potential confounders and interpatient variability. However, they may not be suitable for acute diseases and progressive disorders, and attrition rates can be higher. Multi-arm randomized designs offer increased study power with a lower sample size but necessitate more intricate design, analysis, and result reporting. Conclusion: In conclusion, each study design in NCTs comes with its set of strengths and limitations. The selection of an appropriate design should consider determinants and common considerations to provide robust evidence for establishing cause-and-effect associations or assessing the safety and efficacy of food products in nutrition research. This comprehensive understanding aids researchers in making informed choices when planning and conducting nutrition clinical trials.