کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
6150793 1596430 2016 6 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Design of a multi-arm randomized clinical trial with no control arm
ترجمه فارسی عنوان
طراحی یک کارآزمایی بالینی تصادفی چند طرفه بدون بازوی کنترل
موضوعات مرتبط
علوم پزشکی و سلامت پزشکی و دندانپزشکی پزشکی و دندانپزشکی (عمومی)
چکیده انگلیسی

BackgroundClinical trial designs that include multiple treatments are currently limited to those that perform pairwise comparisons of each investigational treatment to a single control. However, there are settings, such as the recent Ebola outbreak, in which no treatment has been demonstrated to be effective; and therefore, no standard of care exists which would serve as an appropriate control.Methods/designFor illustrative purposes, we focused on the care of patients presenting in austere settings with critically ill ‘sepsis-like’ syndromes. Our approach involves a novel algorithm for comparing mortality among arms without requiring a single fixed control. The algorithm allows poorly-performing arms to be dropped during interim analyses. Consequently, the study may be completed earlier than planned. We used simulation to determine operating characteristics for the trial and to estimate the required sample size.ResultsWe present a potential study design targeting a minimal effect size of a 23% relative reduction in mortality between any pair of arms. Using estimated power and spurious significance rates from the simulated scenarios, we show that such a trial would require 2550 participants. Over a range of scenarios, our study has 80 to 99% power to select the optimal treatment. Using a fixed control design, if the control arm is least efficacious, 640 subjects would be enrolled into the least efficacious arm, while our algorithm would enroll between 170 and 430. This simulation method can be easily extended to other settings or other binary outcomes.ConclusionEarly dropping of arms is efficient and ethical when conducting clinical trials with multiple arms.

ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Contemporary Clinical Trials - Volume 46, January 2016, Pages 12–17