Design of a clinical trial using generalized pairwise comparisons to test a less intensive treatment regimen
CONCLUSION: Generalized pairwise comparisons allow for simultaneous assessment of efficacy and safety, with priority given to the former. The sample size required would be of the order of 300 patients, as compared with more than 700 patients for a non-inferiority trial using a margin of 4% against the less intensive treatment regimen for the absolute difference in event-free survival at 2 years, as considered here.PMID:37877379 | DOI:10.1177/17407745231206465 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Micka ël De Backer Manju Sengar Vikram Mathews Samuel Salvaggio Vaiva Deltuvaite-Thomas Jean-Christophe Chi êm Everardo D Saad Marc Buyse Source Type: research
Estimating counterfactual placebo HIV incidence in HIV prevention trials without placebo arms based on markers of HIV exposure
CONCLUSION: Careful assessment of the underlying assumptions, study of their violation, evaluation of the approach in trials with placebo arms, and advancement of improved exposure markers are needed before the HIV exposure marker approach can be relied upon in practice.PMID:37877356 | DOI:10.1177/17407745231203327 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Yifan Zhu Fei Gao David V Glidden Deborah Donnell Holly Janes Source Type: research
Development and pilot validation of a novel disfigurement severity scale for plexiform neurofibromas in children with neurofibromatosis type 1
CONCLUSION: This study demonstrates that our observer-rated disfigurement severity score for plexiform neurofibroma was feasible, reliable, and documented improvement in disfigurement in participants with plexiform neurofibroma shrinkage. Prospective studies in larger samples are needed to validate this scale further.PMID:37877369 | DOI:10.1177/17407745231206402 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Liny John Gurbani Singh Eva Dombi Pamela L Wolters Staci Martin Andrea Baldwin Seth M Steinberg Jessica Bernstein Patricia Whitcomb Dominique C Pichard Anne Dufek Andy Gillespie Kara Heisey Miriam Bornhorst Michael J Fisher Brian D Weiss AeRang Kim Brigit Source Type: research
Do recruitment SWAT interventions have an impact on participant retention in randomised controlled trials? A systematic review
CONCLUSION: Assessment of the impacts of recruitment strategies, evaluated in a SWAT, on retention of participants in the host trial remains limited. Assessment of the impact of recruitment interventions on retention is recommended to minimise future research costs and waste.PMID:37877371 | DOI:10.1177/17407745231206283 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Catherine E Arundel Laura Clark Source Type: research
Statistical rules for safety monitoring in clinical trials
CONCLUSIONS: Because the goal is to minimize the number of patients exposed to and experiencing toxicities from an unsafe therapy, we recommend using the Pocock or Beta-Binomial, weak prior methods for constructing safety stopping rules. At the design stage, the operating characteristics of candidate rules should be evaluated under various possible toxicity rates in order to guide the choice of rule(s) for a given trial; our R package facilitates this evaluation.PMID:37877375 | DOI:10.1177/17407745231203391 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Michael J Martens Brent R Logan Source Type: research
Design of a clinical trial using generalized pairwise comparisons to test a less intensive treatment regimen
CONCLUSION: Generalized pairwise comparisons allow for simultaneous assessment of efficacy and safety, with priority given to the former. The sample size required would be of the order of 300 patients, as compared with more than 700 patients for a non-inferiority trial using a margin of 4% against the less intensive treatment regimen for the absolute difference in event-free survival at 2 years, as considered here.PMID:37877379 | DOI:10.1177/17407745231206465 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Micka ël De Backer Manju Sengar Vikram Mathews Samuel Salvaggio Vaiva Deltuvaite-Thomas Jean-Christophe Chi êm Everardo D Saad Marc Buyse Source Type: research
Estimating counterfactual placebo HIV incidence in HIV prevention trials without placebo arms based on markers of HIV exposure
CONCLUSION: Careful assessment of the underlying assumptions, study of their violation, evaluation of the approach in trials with placebo arms, and advancement of improved exposure markers are needed before the HIV exposure marker approach can be relied upon in practice.PMID:37877356 | DOI:10.1177/17407745231203327 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Yifan Zhu Fei Gao David V Glidden Deborah Donnell Holly Janes Source Type: research
Development and pilot validation of a novel disfigurement severity scale for plexiform neurofibromas in children with neurofibromatosis type 1
CONCLUSION: This study demonstrates that our observer-rated disfigurement severity score for plexiform neurofibroma was feasible, reliable, and documented improvement in disfigurement in participants with plexiform neurofibroma shrinkage. Prospective studies in larger samples are needed to validate this scale further.PMID:37877369 | DOI:10.1177/17407745231206402 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Liny John Gurbani Singh Eva Dombi Pamela L Wolters Staci Martin Andrea Baldwin Seth M Steinberg Jessica Bernstein Patricia Whitcomb Dominique C Pichard Anne Dufek Andy Gillespie Kara Heisey Miriam Bornhorst Michael J Fisher Brian D Weiss AeRang Kim Brigit Source Type: research
Do recruitment SWAT interventions have an impact on participant retention in randomised controlled trials? A systematic review
CONCLUSION: Assessment of the impacts of recruitment strategies, evaluated in a SWAT, on retention of participants in the host trial remains limited. Assessment of the impact of recruitment interventions on retention is recommended to minimise future research costs and waste.PMID:37877371 | DOI:10.1177/17407745231206283 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Catherine E Arundel Laura Clark Source Type: research
Statistical rules for safety monitoring in clinical trials
CONCLUSIONS: Because the goal is to minimize the number of patients exposed to and experiencing toxicities from an unsafe therapy, we recommend using the Pocock or Beta-Binomial, weak prior methods for constructing safety stopping rules. At the design stage, the operating characteristics of candidate rules should be evaluated under various possible toxicity rates in order to guide the choice of rule(s) for a given trial; our R package facilitates this evaluation.PMID:37877375 | DOI:10.1177/17407745231203391 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Michael J Martens Brent R Logan Source Type: research
Design of a clinical trial using generalized pairwise comparisons to test a less intensive treatment regimen
CONCLUSION: Generalized pairwise comparisons allow for simultaneous assessment of efficacy and safety, with priority given to the former. The sample size required would be of the order of 300 patients, as compared with more than 700 patients for a non-inferiority trial using a margin of 4% against the less intensive treatment regimen for the absolute difference in event-free survival at 2 years, as considered here.PMID:37877379 | DOI:10.1177/17407745231206465 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Micka ël De Backer Manju Sengar Vikram Mathews Samuel Salvaggio Vaiva Deltuvaite-Thomas Jean-Christophe Chi êm Everardo D Saad Marc Buyse Source Type: research
Estimating counterfactual placebo HIV incidence in HIV prevention trials without placebo arms based on markers of HIV exposure
CONCLUSION: Careful assessment of the underlying assumptions, study of their violation, evaluation of the approach in trials with placebo arms, and advancement of improved exposure markers are needed before the HIV exposure marker approach can be relied upon in practice.PMID:37877356 | DOI:10.1177/17407745231203327 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Yifan Zhu Fei Gao David V Glidden Deborah Donnell Holly Janes Source Type: research
Development and pilot validation of a novel disfigurement severity scale for plexiform neurofibromas in children with neurofibromatosis type 1
CONCLUSION: This study demonstrates that our observer-rated disfigurement severity score for plexiform neurofibroma was feasible, reliable, and documented improvement in disfigurement in participants with plexiform neurofibroma shrinkage. Prospective studies in larger samples are needed to validate this scale further.PMID:37877369 | DOI:10.1177/17407745231206402 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Liny John Gurbani Singh Eva Dombi Pamela L Wolters Staci Martin Andrea Baldwin Seth M Steinberg Jessica Bernstein Patricia Whitcomb Dominique C Pichard Anne Dufek Andy Gillespie Kara Heisey Miriam Bornhorst Michael J Fisher Brian D Weiss AeRang Kim Brigit Source Type: research
Do recruitment SWAT interventions have an impact on participant retention in randomised controlled trials? A systematic review
CONCLUSION: Assessment of the impacts of recruitment strategies, evaluated in a SWAT, on retention of participants in the host trial remains limited. Assessment of the impact of recruitment interventions on retention is recommended to minimise future research costs and waste.PMID:37877371 | DOI:10.1177/17407745231206283 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Catherine E Arundel Laura Clark Source Type: research
Statistical rules for safety monitoring in clinical trials
CONCLUSIONS: Because the goal is to minimize the number of patients exposed to and experiencing toxicities from an unsafe therapy, we recommend using the Pocock or Beta-Binomial, weak prior methods for constructing safety stopping rules. At the design stage, the operating characteristics of candidate rules should be evaluated under various possible toxicity rates in order to guide the choice of rule(s) for a given trial; our R package facilitates this evaluation.PMID:37877375 | DOI:10.1177/17407745231203391 (Source: Clinical Trials)
Source: Clinical Trials - October 25, 2023 Category: Research Authors: Michael J Martens Brent R Logan Source Type: research
