Results 1 to 10 of about 510,312 (368)

A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint [PDF]

Pilot and Feasibility Studies, 2018
Background It has been argued that true endpoints (or ‘hard’ endpoints) for clinical trials, which are meaningful to clinicians, researchers and patients alike, are limited to those that measure health status, survival and cost.
M. J. Campbell, G. A. Lancaster, S. M. Eldridge   +2 more
doaj   +6 more sources

Progression-free survival as a surrogate endpoint in myeloma clinical trials: an evolving paradigm [PDF]

Blood Cancer Journal
Measurement of overall survival (OS) remains the gold standard for interpreting the impact of new therapies for multiple myeloma in phase 3 trials. However, as outcomes have improved, it is increasingly challenging to use OS as the primary endpoint if ...
Charlotte Pawlyn, Fredrik H. Schjesvold, David A. Cairns, L. J. Wei, Faith Davies, Omar Nadeem, Haifaa Abdulhaq, Maria-Victoria Mateos, Jacob Laubach, Katja Weisel, Heinz Ludwig, S. Vincent Rajkumar, Pieter Sonneveld, Graham Jackson, Gareth Morgan, Paul G. Richardson   +15 more
doaj   +3 more sources

Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials. [PDF]

Hum Gene Ther, 2023
Duchenne muscular dystrophy (DMD) is a serious, rare genetic disease, affecting primarily boys. It is caused by mutations in the DMD gene and is characterized by progressive muscle degeneration that results in loss of function and early death due to ...
Chamberlain JS, Robb M, Braun S, Brown KJ, Danos O, Ganot A, Gonzalez-Alegre P, Hunter N, McDonald C, Morris C, Tobolowsky M, Wagner KR, Ziolkowski O, Duan D.   +13 more
europepmc   +2 more sources

eGFR slope as a surrogate endpoint for clinical study in early stage of chronic kidney disease: from The Japan Chronic Kidney Disease Database. [PDF]

Clin Exp Nephrol, 2023
Background In clinical trials targeting early chronic kidney disease (CKD), eGFR slope has been proposed as a surrogate endpoint for predicting end-stage kidney disease (ESKD).
Itano S, Kanda E, Nagasu H, Nangaku M, Kashihara N.   +4 more
europepmc   +2 more sources

An evaluation of culture results during treatment for tuberculosis as surrogate endpoints for treatment failure and relapse. [PDF]

PLoS ONE, 2013
It is widely acknowledged that new regimens are urgently needed for the treatment of tuberculosis. The primary endpoint in the Phase III trials is a composite outcome of failure at the end of treatment or relapse after stopping treatment. Such trials are
Patrick P J Phillips, Katherine Fielding, Andrew J Nunn   +2 more
doaj   +5 more sources

Considerations for the Feasibility of Neutralizing Antibodies as a Surrogate Endpoint for COVID-19 Vaccines. [PDF]

Front Immunol, 2022
To effectively control and prevent the pandemic of coronavirus disease 2019 (COVID-19), suitable vaccines have been researched and developed rapidly.
Liu J, Mao Q, Wu X, He Q, Bian L, Bai Y, Wang Z, Wang Q, Zhang J, Liang Z, Xu M.   +10 more
europepmc   +2 more sources

Using Bayesian Evidence Synthesis Methods to Incorporate Real-World Evidence in Surrogate Endpoint Evaluation. [PDF]

Med Decis Making, 2023
Objective Traditionally, validation of surrogate endpoints has been carried out using randomized controlled trial (RCT) data. However, RCT data may be too limited to validate surrogate endpoints.
Wheaton L, Papanikos A, Thomas A, Bujkiewicz S.   +3 more
europepmc   +3 more sources

Development of a framework and decision tool for the evaluation of health technologies based on surrogate endpoint evidence. [PDF]

Health Econ, 2022
In the drive toward faster patient access to treatments, health technology assessment (HTA) agencies and payers are increasingly faced with reliance on evidence based on surrogate endpoints, increasing decision uncertainty.
Ciani O, Grigore B, Taylor RS.
europepmc   +2 more sources

Application of the iBox prognostication system as a surrogate endpoint in the TRANSFORM randomised controlled trial: proof-of-concept study. [PDF]

BMJ Open, 2021
Objectives Development of pharmaceutical agents in transplantation is currently limited by long waits for hard endpoints. We applied a validated integrative risk-prognostication system integrative Box (iBox) as a surrogate endpoint to the TRANSFORM Study,
Aubert O, Divard G, Pascual J, Oppenheimer F, Sommerer C, Citterio F, Tedesco H, Chadban S, Henry M, Vincenti F, Srinivas T, Watarai Y, Legendre C, Bernhardt P, Loupy A.   +14 more
europepmc   +2 more sources

Validation of the Surrogate Threshold Effect for Change in Bone Mineral Density as a Surrogate Endpoint for Fracture Outcomes: The FNIH-ASBMR SABRE Project. [PDF]

J Bone Miner Res, 2022
The surrogate threshold effect (STE) is defined as the minimum treatment effect on a surrogate that is reliably predictive of a treatment effect on the clinical outcome. It provides a framework for implementing a clinical trial with a surrogate endpoint.
Eastell R, Vittinghoff E, Lui LY, McCulloch CE, Pavo I, Chines A, Khosla S, Cauley JA, Mitlak B, Bauer DC, Bouxsein M, Black DM.   +11 more
europepmc   +2 more sources