COBALT: A Confirmatory Trial of Obeticholic Acid in Primary Biliary Cholangitis With Placebo and External Controls.

Kris V KowdleyGideon M HirschfieldCharles CoombsElizabeth S MalechaLeona BessonovaJing LiNuvan RathnayakaGeorge MellsDavid E JonesPalak J TrivediBettina E HansenRachel Smith James Wason Shaun Kuan Wei Hiu Dorcas N Kareithi Andrew L Mason Christopher L Bowlus Kate MullerMarco CarboneMarina C Berenguer Piotr MilkiewiczFemi AdekunleAlejandra Villamil

Published in: The American journal of gastroenterology (2024)

Functional unblinding and treatment crossover, particularly in the placebo arm, confounded the ITT estimate of outcomes associated with OCA in the RCT. Comparison with the real-world EC showed that OCA treatment significantly reduced the risk of negative clinical outcomes. These analyses demonstrate the value of EC data in confirmatory trials and suggest that treatment with OCA improves clinical outcomes in patients with PBC.

Keyphrases

clinical trial
phase iii
metabolic syndrome
double blind
open label
machine learning
electronic health record
phase ii
smoking cessation
data analysis
glycemic control