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Articles by Khadija Rerhou
[2610.28265] Composite Variable Strategies for Continuous Endpoints
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Challenges and Lessons Learned in Autologous Chimeric Antigen Receptor T-Cell Therapy Development from a Statistical Perspective - Therapeutic Innovation & Regulatory Science
Abstract Chimeric antigen receptor (CAR) T-cell therapy is a human gene therapy product where T cells from a patient are genetically modified to enable them to recognize desired target antigen(s) more effectively. In recent years, promising antitumor activity has been seen with autologous CAR T cells. Since 2017, six CAR T-cell therapies for the treatment of hematological malignancies have been approved by the Food and Drug Administration (FDA).
Development of consensus-driven SPIRIT and CONSORT extensions for early phase dose-finding trials: the DEFINE study
Solovyeva, O. et al. (2023) Development of consensus-driven SPIRIT and CONSORT extensions for early phase dose-finding trials: the DEFINE study. BMC Medicine, 21, 246. (doi: 10.1186/s12916-023-02937-0) Text 302304.pdf - Published Version Available under License Creative Commons Attribution. 1MB Abstract Background: Early phase dose-finding (EPDF) trials are crucial for the development of a new intervention and influence whether it should be investigated in further trials.
Development of consensus-driven SPIRIT and CONSORT extensions for early phase dose-finding trials: the DEFINE study - BMC Medicine
Timeline and key parts of the study To drive delivery of the project, an international Executive Committee (EC) of multidisciplinary experts was formed, and an Independent Expert Panel (IEP) provided oversight and quality control assurances (see Additional file A1). Development of CONSORT-DEFINE commenced in March 2021, followed by SPIRIT-DEFINE in January 2022.
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