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Pivotal trial characteristics and types of endpoints used to support Food and Drug Administration rare disease drug approvals between 2013 and 2022.
Clin Trials
January 2025
Rare Diseases Team, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA.
Background/aims: Rare disease drug development faces unique challenges, such as genotypic and phenotypic heterogeneity within small patient populations and a lack of established outcome measures for conditions without previously successful drug development programs. These challenges complicate the process of selecting the appropriate trial endpoints and conducting clinical trials in rare diseases. In this descriptive study, we examined novel drug approvals for non-oncologic rare diseases by the U.
View Article and Find Full Text PDFAdaptive promising zone design for sequential parallel comparison design with continuous outcomes.
Clin Trials
January 2025
Department of Biostatistics, University of Florida, Gainesville, FL, USA.
Introduction: The sequential parallel comparison design has emerged as a valuable tool in clinical trials with high placebo response rates. To further enhance its efficiency and effectiveness, adaptive strategies, such as sample size adjustment and allocation ratio modification can be employed.
Methods: We compared the performance of Jennison and Turnbull's method and the Promising Zone approach for sample size adjustment in a two-phase sequential parallel comparison design study.
Background/aims: Certain sociodemographic groups are routinely underrepresented in clinical trials, limiting generalisability. Here, we describe the extent to which enriched enrolment approaches yielded a diverse trial population enriched for older age in a randomised controlled trial of a blood-based multi-cancer early detection test (NCT05611632).
Methods: Participants aged 50-77 years were recruited from eight Cancer Alliance regions in England.
Winery By-Products and Effects on Atherothrombotic Markers: Focus on Platelet-Activating Factor.
Front Biosci (Landmark Ed)
January 2025
Department of Nutrition and Dietetics, School of Health Sciences and Education, Harokopio University, 17676 Athens, Greece.
Platelet aggregation and inflammation play a crucial role in atherothrombosis. Wine contains micro-constituents of proper quality and quantity that exert cardioprotective actions, partly through inhibiting platelet-activating factor (PAF), a potent inflammatory and thrombotic lipid mediator. However, wine cannot be consumed extensively due to the presence of ethanol.
View Article and Find Full Text PDFEfficacy of General Magnetic Therapy and Motor-Evoked Potential-Guided Precision Magnetic Therapy in Patients with Overactive Bladder: A Randomized Controlled Trial.
Br J Hosp Med (Lond)
January 2025
Department of Urology, The Second Affiliated Hospital of Zhejiang Chinese Medicine University, Hangzhou, Zhejiang, China.
Overactive bladder (OAB) is a prevalent chronic condition affecting approximately 12% of adults, with incidence increasing with age. While pharmacological and behavioural therapies are standard treatments, their efficacy is often limited by side effects and poor adherence. This study aimed to compare the therapeutic effects of precision magnetic stimulation guided by motor-evoked potential with general magnetic therapy in patients with OAB.
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