Patient-reported measures of tinnitus for individuals with neurofibromatosis type 2-related schwannomatosis: Recommendations for clinical trials
DISCUSSION: Response Evaluation in Neurofibromatosis and Schwannomatosis currently recommends the Tinnitus Functional Index for the assessment of tinnitus in neurofibromatosis type 2 clinical trials.PMID:38321701 | PMC:PMC10865763 | DOI:10.1177/17407745231217279 (Source: Clinical Trials)
Source: Clinical Trials - February 7, 2024 Category: Research Authors: Heather L Thompson Jane Grabowski Barbara Franklin Kimberley S Koetsier D Bradley Welling Source Type: research
Patient-reported measures of tinnitus for individuals with neurofibromatosis type 2-related schwannomatosis: Recommendations for clinical trials
DISCUSSION: Response Evaluation in Neurofibromatosis and Schwannomatosis currently recommends the Tinnitus Functional Index for the assessment of tinnitus in neurofibromatosis type 2 clinical trials.PMID:38321701 | PMC:PMC10865763 | DOI:10.1177/17407745231217279 (Source: Clinical Trials)
Source: Clinical Trials - February 7, 2024 Category: Research Authors: Heather L Thompson Jane Grabowski Barbara Franklin Kimberley S Koetsier D Bradley Welling Source Type: research
New strategies for confirmatory testing of secondary hypotheses on combined data from multiple trials
CONCLUSIONS: Inference on combined data across trials is a useful approach for generating pivotal evidence of efficacy for important but sample size-intense secondary endpoints. It requires careful preparation and regulatory discussion.PMID:38311901 | DOI:10.1177/17407745231214382 (Source: Clinical Trials)
Source: Clinical Trials - February 5, 2024 Category: Research Authors: Marc Vandemeulebroecke Dieter A H äring Eva Hua Xiaoling Wei Dong Xi Source Type: research
The use of linked administrative data in Australian randomised controlled trials: A scoping review
CONCLUSIONS: As linked administrative data are not intended for research purposes, detailed knowledge of the data sets is required by researchers, and the time delay in receiving the data is viewed as a barrier to its use. The lack of access to primary care data sets is viewed as a barrier to administrative data use; however, work to expand the number of healthcare data sets that can be linked has made it easier for researchers to access and use these data, which may have implications on how randomised controlled trials will be run in future.PMID:38305216 | DOI:10.1177/17407745231225618 (Source: Clinical Trials)
Source: Clinical Trials - February 2, 2024 Category: Research Authors: Salma Fahridin Neeru Agarwal Karen Bracken Stephen Law Rachael L Morton Source Type: research
Is inadequate risk stratification diluting hazard ratio estimates in randomized clinical trials?
Clin Trials. 2024 Feb 2:17407745231222448. doi: 10.1177/17407745231222448. Online ahead of print.ABSTRACTIn randomized clinical trials, analyses of time-to-event data without risk stratification, or with stratification based on pre-selected factors revealed at the end of the trial to be at most weakly associated with risk, are quite common. We caution that such analyses are likely delivering hazard ratio estimates that unwittingly dilute the evidence of benefit for the test relative to the control treatment. To make our case, first, we use a hypothetical scenario to contrast risk-unstratified and risk-stratified hazard rat...
Source: Clinical Trials - February 2, 2024 Category: Research Authors: Devan V Mehrotra Rachel Marceau West Source Type: research
Research encouraging off-label use of quetiapine: A systematic meta-epidemiological analysis
CONCLUSION: Many small, post-approval studies suggested the promise of quetiapine for numerous off-label indications. These findings generally went unconfirmed in large, blinded, randomized trials years after first being published. The imbalance of exploratory and confirmatory studies likely encourages ineffective off-label treatment.PMID:38284364 | DOI:10.1177/17407745231225470 (Source: Clinical Trials)
Source: Clinical Trials - January 29, 2024 Category: Research Authors: Peter Grabitz Lana Saksone Susanne Gabriele Schorr Johannes Schwietering Merlin Bittlinger Jonathan Kimmelman Source Type: research
Statistical and practical considerations in planning and conduct of dose-optimization trials
This article provides a comprehensive review of various design strategies for dose-optimization trials, including phase 1/2 and 2/3 designs, and highlights their respective advantages and disadvantages. In addition, practical considerations for selecting an appropriate design and planning and executing the trial are discussed. The article also presents freely available software tools that can be utilized for designing and implementing dose-optimization trials. The approaches and their implementation are illustrated through real-world examples.PMID:38243399 | DOI:10.1177/17407745231207085 (Source: Clinical Trials)
Source: Clinical Trials - January 20, 2024 Category: Research Authors: Ying Yuan Heng Zhou Suyu Liu Source Type: research
Adaptive Bayesian information borrowing methods for finding and optimizing subgroup-specific doses
Clin Trials. 2024 Jan 19:17407745231212193. doi: 10.1177/17407745231212193. Online ahead of print.ABSTRACTIn precision oncology, integrating multiple cancer patient subgroups into a single master protocol allows for the simultaneous assessment of treatment effects in these subgroups and promotes the sharing of information between them, ultimately reducing sample sizes and costs and enhancing scientific validity. However, the safety and efficacy of these therapies may vary across different subgroups, resulting in heterogeneous outcomes. Therefore, identifying subgroup-specific optimal doses in early-phase clinical trials is...
Source: Clinical Trials - January 20, 2024 Category: Research Authors: Jingyi Zhang Ruitao Lin Xin Chen Fangrong Yan Source Type: research
The symbolic two-step method applied to cancer care delivery research: Safeguarding against designing an underpowered cluster randomized trial with a continuous outcome by accounting for the imprecision in the within- and between-center variation
CONCLUSIONS: Accounting for imprecision in the estimates of the components required for sample size estimation using the symbolic two-step method in the design of a cluster randomized trial yields conservative estimates of power.PMID:38243404 | DOI:10.1177/17407745231219680 (Source: Clinical Trials)
Source: Clinical Trials - January 20, 2024 Category: Research Authors: David Zahrieh Blaize W Kandler Jennifer Le-Rademacher Source Type: research
A Bayesian adaptive design approach for stepped-wedge cluster randomized trials
CONCLUSION: This study presents the incorporation of Bayesian adaptive strategies into stepped-wedge cluster randomized trials design. The proposed approach provides the flexibility to stop the trial early if substantial evidence of efficacy or futility is observed, improving the flexibility and efficiency of stepped-wedge cluster randomized trials.PMID:38240270 | DOI:10.1177/17407745231221438 (Source: Clinical Trials)
Source: Clinical Trials - January 19, 2024 Category: Research Authors: Jijia Wang Jing Cao Chul Ahn Song Zhang Source Type: research
Adaptive phase I-II clinical trial designs identifying optimal biological doses for targeted agents and immunotherapies
Clin Trials. 2024 Jan 11:17407745231220661. doi: 10.1177/17407745231220661. Online ahead of print.ABSTRACTTargeted 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 ...
Source: Clinical Trials - January 11, 2024 Category: Research Authors: Yong Zang Beibei Guo Yingjie Qiu Hao Liu Mateusz Opyrchal Xiongbin Lu Source Type: research
Hierarchical Bayesian modeling of heterogeneous outcome variance in cluster randomized trials
CONCLUSION: We proposed new hierarchical Bayesian variance models to accommodate cluster-specific variances in cluster randomized trials. The newly developed methods inform the understanding of how an intervention strategy is implemented and disseminated differently across clusters and can help improve future trial design.PMID:38197388 | DOI:10.1177/17407745231222018 (Source: Clinical Trials)
Source: Clinical Trials - January 10, 2024 Category: Research Authors: Guangyu Tong Jiaqi Tong Yi Jiang Denise Esserman Michael O Harhay Joshua L Warren Source Type: research
Optimizing the doses of cancer drugs after usual dose finding
Clin Trials. 2023 Dec 27:17407745231213882. doi: 10.1177/17407745231213882. Online ahead of print.ABSTRACTSince the middle of the 20th century, oncology's dose-finding paradigm has been oriented toward identifying a drug's maximum tolerated dose, which is then carried forward into phase 2 and 3 trials and clinical practice. For most modern precision medicines, however, maximum tolerated dose is far greater than the minimum dose needed to achieve maximal benefit, leading to unnecessary side effects. Regulatory change may decrease maximum tolerated dose's predominance by enforcing dose optimization of new drugs. Dozens of al...
Source: Clinical Trials - December 27, 2023 Category: Research Authors: Garth W Strohbehn Walter M Stadler Philip S Boonstra Mark J Ratain Source Type: research
A review of patient recruitment in randomised controlled trials of preoperative exercise
CONCLUSION: The recruitment of participants to preoperative exercise randomised controlled trials is challenging, but patient retention appears to be less of a problem. Future trials investigating the effectiveness of a preoperative exercise programme following cancer surgery should consider the time taken to recruit patients. Strategies associated with improved recruitment should be investigated in future studies.PMID:38149306 | DOI:10.1177/17407745231219270 (Source: Clinical Trials)
Source: Clinical Trials - December 27, 2023 Category: Research Authors: Daniel Steffens Michael Solomon Jane Young Paula R Beckenkamp Jenna Bartyn Cherry Koh Mark Hancock Source Type: research
Optimizing the doses of cancer drugs after usual dose finding
Clin Trials. 2023 Dec 27:17407745231213882. doi: 10.1177/17407745231213882. Online ahead of print.ABSTRACTSince the middle of the 20th century, oncology's dose-finding paradigm has been oriented toward identifying a drug's maximum tolerated dose, which is then carried forward into phase 2 and 3 trials and clinical practice. For most modern precision medicines, however, maximum tolerated dose is far greater than the minimum dose needed to achieve maximal benefit, leading to unnecessary side effects. Regulatory change may decrease maximum tolerated dose's predominance by enforcing dose optimization of new drugs. Dozens of al...
Source: Clinical Trials - December 27, 2023 Category: Research Authors: Garth W Strohbehn Walter M Stadler Philip S Boonstra Mark J Ratain Source Type: research
