Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in neurofibromatosis 1 clinical trials
CONCLUSION: The Response Evaluation in Neurofibromatosis and Schwannomatosis patient-reported outcome working group currently recommends the FACE-Q Craniofacial Module Appearance Distress scale to evaluate patient-reported changes in appearance concerns in clinical trials for neurofibromatosis 1-related plexiform or cutaneous neurofibromas. Additional research is needed to validate this measure in people with neurofibromatosis 1, including older adults and those with tumors in various body locations, and explore the effects of nontumor manifestations on appearance concerns in people with neurofibromatosis 1 and schwannomat...
Source: Clinical Trials - December 23, 2023 Category: Research Authors: Vanessa L Merker Heather L Thompson Pamela L Wolters Frank D Buono Cynthia M Hingtgen Tena Rosser Belinda Barton Carolina Barnett Taylor Smith Diana Haberkamp Miranda L McManus Andrea Baldwin Irene P Moss Claas R öhl Staci Martin Source Type: research
A pilot recruitment strategy to enhance ethical and equitable access to Covid-19 pediatric vaccine trials
CONCLUSION: Future use of this approach to enhance equitable access to research while avoiding exploitation should test different methods to build trust and communicate with underserved communities more effectively.PMID:38140914 | DOI:10.1177/17407745231217299 (Source: Clinical Trials)
Source: Clinical Trials - December 23, 2023 Category: Research Authors: William J Muller Ravi Jhaveri Taylor Heald-Sargent Michelle L Macy Nia Heard-Garris Seema Shah Erin Paquette Source Type: research
Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in neurofibromatosis 1 clinical trials
CONCLUSION: The Response Evaluation in Neurofibromatosis and Schwannomatosis patient-reported outcome working group currently recommends the FACE-Q Craniofacial Module Appearance Distress scale to evaluate patient-reported changes in appearance concerns in clinical trials for neurofibromatosis 1-related plexiform or cutaneous neurofibromas. Additional research is needed to validate this measure in people with neurofibromatosis 1, including older adults and those with tumors in various body locations, and explore the effects of nontumor manifestations on appearance concerns in people with neurofibromatosis 1 and schwannomat...
Source: Clinical Trials - December 23, 2023 Category: Research Authors: Vanessa L Merker Heather L Thompson Pamela L Wolters Frank D Buono Cynthia M Hingtgen Tena Rosser Belinda Barton Carolina Barnett Taylor Smith Diana Haberkamp Miranda L McManus Andrea Baldwin Irene P Moss Claas R öhl Staci Martin Source Type: research
A pilot recruitment strategy to enhance ethical and equitable access to Covid-19 pediatric vaccine trials
CONCLUSION: Future use of this approach to enhance equitable access to research while avoiding exploitation should test different methods to build trust and communicate with underserved communities more effectively.PMID:38140914 | DOI:10.1177/17407745231217299 (Source: Clinical Trials)
Source: Clinical Trials - December 23, 2023 Category: Research Authors: William J Muller Ravi Jhaveri Taylor Heald-Sargent Michelle L Macy Nia Heard-Garris Seema Shah Erin Paquette Source Type: research
Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in neurofibromatosis 1 clinical trials
CONCLUSION: The Response Evaluation in Neurofibromatosis and Schwannomatosis patient-reported outcome working group currently recommends the FACE-Q Craniofacial Module Appearance Distress scale to evaluate patient-reported changes in appearance concerns in clinical trials for neurofibromatosis 1-related plexiform or cutaneous neurofibromas. Additional research is needed to validate this measure in people with neurofibromatosis 1, including older adults and those with tumors in various body locations, and explore the effects of nontumor manifestations on appearance concerns in people with neurofibromatosis 1 and schwannomat...
Source: Clinical Trials - December 23, 2023 Category: Research Authors: Vanessa L Merker Heather L Thompson Pamela L Wolters Frank D Buono Cynthia M Hingtgen Tena Rosser Belinda Barton Carolina Barnett Taylor Smith Diana Haberkamp Miranda L McManus Andrea Baldwin Irene P Moss Claas R öhl Staci Martin Source Type: research
A pilot recruitment strategy to enhance ethical and equitable access to Covid-19 pediatric vaccine trials
CONCLUSION: Future use of this approach to enhance equitable access to research while avoiding exploitation should test different methods to build trust and communicate with underserved communities more effectively.PMID:38140914 | DOI:10.1177/17407745231217299 (Source: Clinical Trials)
Source: Clinical Trials - December 23, 2023 Category: Research Authors: William J Muller Ravi Jhaveri Taylor Heald-Sargent Michelle L Macy Nia Heard-Garris Seema Shah Erin Paquette Source Type: research
Algorithmic benchmark modulation: A novel method to develop success rates for clinical studies
CONCLUSION: The Probability of Technical Success modulation algorithm addresses several concerns generally associated with assurance calculations or benchmark without modulation: selection biases, situations where little relevant prior data are available and the difficulty to model many factors affecting study outcomes. As opposed to using industry benchmarks, the Probability of Technical Success modulation algorithm allows to accommodate project-specific considerations.PMID:38126256 | DOI:10.1177/17407745231207858 (Source: Clinical Trials)
Source: Clinical Trials - December 21, 2023 Category: Research Authors: Bart Ja Willigers Sridevi Nagarajan Serban Ghiorghui Patrick Darken Simon Lennard Source Type: research
Algorithmic benchmark modulation: A novel method to develop success rates for clinical studies
CONCLUSION: The Probability of Technical Success modulation algorithm addresses several concerns generally associated with assurance calculations or benchmark without modulation: selection biases, situations where little relevant prior data are available and the difficulty to model many factors affecting study outcomes. As opposed to using industry benchmarks, the Probability of Technical Success modulation algorithm allows to accommodate project-specific considerations.PMID:38126256 | DOI:10.1177/17407745231207858 (Source: Clinical Trials)
Source: Clinical Trials - December 21, 2023 Category: Research Authors: Bart Ja Willigers Sridevi Nagarajan Serban Ghiorghui Patrick Darken Simon Lennard Source Type: research
Risk-benefit trade-offs and precision utilities in phase I-II clinical trials
CONCLUSIONS: Herein, we first describe why it is essential to consider clinical practice when designing a clinical trial and outline a stepwise process for doing this. We then review a precision phase I-II design based on utilities tailored to prognostic subgroups that characterize efficacy-toxicity risk-benefit trade-offs. The design chooses each patient's dose to optimize their expected utility and allows patients in different prognostic subgroups to have different optimal doses. We illustrate the design with a dose-finding trial of a new therapeutic agent for metastatic clear cell renal cell carcinoma.PMID:38111231 | DO...
Source: Clinical Trials - December 19, 2023 Category: Research Authors: Pavlos Msaouel Juhee Lee Peter F Thall Source Type: research
Risk-benefit trade-offs and precision utilities in phase I-II clinical trials
CONCLUSIONS: Herein, we first describe why it is essential to consider clinical practice when designing a clinical trial and outline a stepwise process for doing this. We then review a precision phase I-II design based on utilities tailored to prognostic subgroups that characterize efficacy-toxicity risk-benefit trade-offs. The design chooses each patient's dose to optimize their expected utility and allows patients in different prognostic subgroups to have different optimal doses. We illustrate the design with a dose-finding trial of a new therapeutic agent for metastatic clear cell renal cell carcinoma.PMID:38111231 | DO...
Source: Clinical Trials - December 19, 2023 Category: Research Authors: Pavlos Msaouel Juhee Lee Peter F Thall Source Type: research
Risk-benefit trade-offs and precision utilities in phase I-II clinical trials
CONCLUSIONS: Herein, we first describe why it is essential to consider clinical practice when designing a clinical trial and outline a stepwise process for doing this. We then review a precision phase I-II design based on utilities tailored to prognostic subgroups that characterize efficacy-toxicity risk-benefit trade-offs. The design chooses each patient's dose to optimize their expected utility and allows patients in different prognostic subgroups to have different optimal doses. We illustrate the design with a dose-finding trial of a new therapeutic agent for metastatic clear cell renal cell carcinoma.PMID:38111231 | DO...
Source: Clinical Trials - December 19, 2023 Category: Research Authors: Pavlos Msaouel Juhee Lee Peter F Thall Source Type: research
Adherence to key recommendations for design and analysis of stepped-wedge cluster randomized trials: A review of trials published 2016-2022
CONCLUSIONS: Future methods development should prioritize methods for SW-CRTs with binary or time-to-event outcomes, small numbers of clusters, continuous recruitment designs, multivariate outcomes, or time-dependent repeated measures. Trialists, journal editors, and peer reviewers should be aware that SW-CRTs have additional methodological requirements over parallel arm designs including the need to account for period effects as well as complex intracluster correlations.PMID:37990575 | DOI:10.1177/17407745231208397 (Source: Clinical Trials)
Source: Clinical Trials - November 22, 2023 Category: Research Authors: Pascale Nevins Mary Ryan Kendra Davis-Plourde Yongdong Ouyang Jules Antoine Pereira Macedo Can Meng Guangyu Tong Xueqi Wang Luis Ortiz-Reyes Agn ès Caille Fan Li Monica Taljaard Source Type: research
Adherence to key recommendations for design and analysis of stepped-wedge cluster randomized trials: A review of trials published 2016-2022
CONCLUSIONS: Future methods development should prioritize methods for SW-CRTs with binary or time-to-event outcomes, small numbers of clusters, continuous recruitment designs, multivariate outcomes, or time-dependent repeated measures. Trialists, journal editors, and peer reviewers should be aware that SW-CRTs have additional methodological requirements over parallel arm designs including the need to account for period effects as well as complex intracluster correlations.PMID:37990575 | DOI:10.1177/17407745231208397 (Source: Clinical Trials)
Source: Clinical Trials - November 22, 2023 Category: Research Authors: Pascale Nevins Mary Ryan Kendra Davis-Plourde Yongdong Ouyang Jules Antoine Pereira Macedo Can Meng Guangyu Tong Xueqi Wang Luis Ortiz-Reyes Agn ès Caille Fan Li Monica Taljaard Source Type: research
Adherence to key recommendations for design and analysis of stepped-wedge cluster randomized trials: A review of trials published 2016-2022
CONCLUSIONS: Future methods development should prioritize methods for SW-CRTs with binary or time-to-event outcomes, small numbers of clusters, continuous recruitment designs, multivariate outcomes, or time-dependent repeated measures. Trialists, journal editors, and peer reviewers should be aware that SW-CRTs have additional methodological requirements over parallel arm designs including the need to account for period effects as well as complex intracluster correlations.PMID:37990575 | DOI:10.1177/17407745231208397 (Source: Clinical Trials)
Source: Clinical Trials - November 22, 2023 Category: Research Authors: Pascale Nevins Mary Ryan Kendra Davis-Plourde Yongdong Ouyang Jules Antoine Pereira Macedo Can Meng Guangyu Tong Xueqi Wang Luis Ortiz-Reyes Agn ès Caille Fan Li Monica Taljaard Source Type: research
Adherence to key recommendations for design and analysis of stepped-wedge cluster randomized trials: A review of trials published 2016-2022
CONCLUSIONS: Future methods development should prioritize methods for SW-CRTs with binary or time-to-event outcomes, small numbers of clusters, continuous recruitment designs, multivariate outcomes, or time-dependent repeated measures. Trialists, journal editors, and peer reviewers should be aware that SW-CRTs have additional methodological requirements over parallel arm designs including the need to account for period effects as well as complex intracluster correlations.PMID:37990575 | DOI:10.1177/17407745231208397 (Source: Clinical Trials)
Source: Clinical Trials - November 22, 2023 Category: Research Authors: Pascale Nevins Mary Ryan Kendra Davis-Plourde Yongdong Ouyang Jules Antoine Pereira Macedo Can Meng Guangyu Tong Xueqi Wang Luis Ortiz-Reyes Agn ès Caille Fan Li Monica Taljaard Source Type: research
