Do single‐arm trials have a role in drug development plans incorporating randomised trials?

MJ Grayling, AP Mander - Pharmaceutical statistics, 2016 - Wiley Online Library
Often, single‐arm trials are used in phase II to gather the first evidence of an oncological
drug's efficacy, with drug activity determined through tumour response using the RECIST …

A phase II study of the orally administered negative enantiomer of gossypol (AT-101), a BH3 mimetic, in patients with advanced adrenal cortical carcinoma

H Xie, J Yin, MH Shah, ME Menefee, KC Bible… - Investigational new …, 2019 - Springer
Background Adrenal cortical carcinoma (ACC) is a rare cancer with treatment options of
limited efficacy, and poor prognosis if metastatic. AT-101 is a more potent inhibitor of B cell …

Adaptive clinical trials in tuberculosis: applications, challenges and solutions

GR Davies, PPJ Phillips, T Jaki - The International Journal of …, 2015 - ingentaconnect.com
Drug development for tuberculosis (TB) faces numerous practical obstacles, including the
need for combination treatment with at least three drugs, reliance on possibly …

Mouse tumour models to guide drug development and identify resistance mechanisms

M Das Thakur, NK Pryer, M Singh - The Journal of pathology, 2014 - Wiley Online Library
We need improved, translatable and predictive tumour models for the evaluation of
response and the evolution of resistance to targeted therapeutics. We provide a review of …

Lung cancer clinical trials with a seamless phase II/III design: systematic review

D Palermos, TN Sergentanis, M Gavriatopoulou… - Journal of Clinical …, 2022 - mdpi.com
Current lung cancer clinical research focuses on biomarkers and personalized treatment
strategies. Adaptive clinical trial designs have gained significant ground due to their …

[图书][B] Statistical remedies for medical researchers

PF Thall - 2020 - Springer
Many new medical treatments are developed using powerful technologies, such as
microarrays, gene sequencing, or cytometric machines that can rapidly identify dozens of …

[HTML][HTML] Correlation of single arm versus randomised phase 2 oncology trial characteristics with phase 3 outcome

JG Monzon, AE Hay, GT McDonald, JL Pater… - European Journal of …, 2015 - Elsevier
Background and aim The primary aim of this study was to determine whether randomised
phase 2 (RP2) trials predict phase 3 trial outcome better than single arm phase 2 (SAP2) …

A novel prognostic nomogram for predicting survival in diffuse pleural mesothelioma

Y Aksoy, A Chou, M Mahjoub, A Sheen, L Sioson… - Pathology, 2023 - Elsevier
Recent advances in the management of diffuse pleural mesothelioma (DPM) have
increased interest in prognostication and risk stratification on the basis that maximum benefit …

Maintenance efficacy designs in psychiatry: randomized discontinuation trials–enriched but not better

SN Ghaemi, HP Selker - Journal of Clinical and Translational …, 2017 - cambridge.org
IntroductionAlthough classical randomized clinical trials (RCTs) are the gold standard for
proof of drug efficacy, randomized discontinuation trials (RDTs), sometimes called …

Comparison of single-arm vs. randomized phase II clinical trials: a Bayesian approach

V Sambucini - Journal of biopharmaceutical statistics, 2015 - Taylor & Francis
Single-arm studies are typically used in phase II of clinical trials, whose main objective is to
determine whether a new treatment warrants further testing in a randomized phase III trial …