Adaptive phase I–II clinical trial designs identifying optimal biological doses for targeted agents and immunotherapies.
Targeted agents and immunotherapies have revolutionized cancer treatment, offering promising options for various cancer types. Unlike traditional therapies the principle of "more is better" is not always applicable to these new therapies due to their unique biomedical mechanisms. As a result, variou...
| Published in: | Clinical Trials Vol. 21; no. 3; pp. 298 - 308 |
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| Main Authors: | , , , , , |
| Format: | review tables/charts Journal Article |
| Published: |
Sage Publications, Ltd.
Jun2024
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| Online Access: | View this record in EBSCOhost |
| fields | @attributes: recordID: 1 pdfLink: plink: https://search.ebscohost.com/login.aspx?direct=true&db=ccm&AN=177518667&site=ehost-live header: @attributes: shortDbName: ccm uiTerm: 177518667 longDbName: CINAHL Complete uiTag: AN controlInfo: bkinfo: dissinfo: jinfo: jid: 17407745 AB6 jtl: Clinical Trials issn: 17407745 maglogo: N pubinfo: dt: Jun2024 vid: 21 iid: 3 pid: 33180 pub: Sage Publications, Ltd. place: <Blank> artinfo: ui: 177518667 174696432 177518667 177518667 10.1177/17407745231220661 177518667 ppf: 298 ppct: 10 formats: tig: atl: Adaptive phase I–II clinical trial designs identifying optimal biological doses for targeted agents and immunotherapies. aug: au: Zang, Yong Guo, Beibei Qiu, Yingjie Liu, Hao Opyrchal, Mateusz Lu, Xiongbin affil: Department of Biostatistics and Health Data Science, School of Medicine, Indiana University, Indianapolis, IN, USA sug: subj: Clinical Trials Immunotherapy Dosage Forms Adaptation, Physiological Drug Efficacy Patient Safety Drug Toxicity Survival Analysis Quality of Life Dose-Response Relationship, Drug ab: Targeted agents and immunotherapies have revolutionized cancer treatment, offering promising options for various cancer types. Unlike traditional therapies the principle of "more is better" is not always applicable to these new therapies due to their unique biomedical mechanisms. As a result, various phase I–II clinical trial designs have been proposed to identify the optimal biological dose that maximizes the therapeutic effect of targeted therapies and immunotherapies by jointly monitoring both efficacy and toxicity outcomes. This review article examines several innovative phase I–II clinical trial designs that utilize accumulated efficacy and toxicity outcomes to adaptively determine doses for subsequent patients and identify the optimal biological dose, maximizing the overall therapeutic effect. Specifically, we highlight three categories of phase I–II designs: efficacy-driven, utility-based, and designs incorporating multiple efficacy endpoints. For each design, we review the dose–outcome model, the definition of the optimal biological dose, the dose-finding algorithm, and the software for trial implementation. To illustrate the concepts, we also present two real phase I–II trial examples utilizing the EffTox and ISO designs. Finally, we provide a classification tree to summarize the designs discussed in this article. pubtype: Academic Journal doctype: review tables/charts Journal Article ougenre: Article language: English refInfo: holdings: @attributes: islocal: N |
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