(unported, v3.0) License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 127–138 Open Access Journal of Clinical Trials Dovepress
{"title":"Pediatric clinical trials: a US perspective","authors":"M. Oelstrom, Margo L Hoover-Regan","doi":"10.2147/OAJCT.S48506","DOIUrl":"https://doi.org/10.2147/OAJCT.S48506","url":null,"abstract":"(unported, v3.0) License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 127–138 Open Access Journal of Clinical Trials Dovepress","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"6 1","pages":"127-138"},"PeriodicalIF":1.2,"publicationDate":"2014-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/OAJCT.S48506","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68414327","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
J. Enns, P. Zahradka, Randolph P Guzman, Alanna Baldwin, Brendon Foot, Carla G. Taylor
License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 117–125 Open Access Journal of Clinical Trials Dovepress
许可证。许可的完整条款可在http://creativecommons.org/licenses/by-nc/3.0/上获得。允许非商业用途的工作，没有任何进一步的许可，从多芬医学出版社有限公司，只要工作适当署名。超出许可范围的许可由多芬医疗新闻有限公司管理。有关如何申请许可的信息可在以下网站找到:http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 117-125 Dovepress
{"title":"Randomized controlled trial to evaluate the effect of canola oil on blood vessel function in peripheral arterial disease: rationale and design of the Canola-PAD Study","authors":"J. Enns, P. Zahradka, Randolph P Guzman, Alanna Baldwin, Brendon Foot, Carla G. Taylor","doi":"10.2147/OAJCT.S70576","DOIUrl":"https://doi.org/10.2147/OAJCT.S70576","url":null,"abstract":"License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 117–125 Open Access Journal of Clinical Trials Dovepress","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"6 1","pages":"117-125"},"PeriodicalIF":1.2,"publicationDate":"2014-10-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/OAJCT.S70576","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68415352","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
D. Colombo, G. Bellia, D. Vassellatti, E. Zagni, S. Sgarbi, S. Rizzoli
Only recently has medical research begun to understand the importance of taking sex into account, recognizing that symptoms and responses to medical treatment may be very different between males and females. However, the analyses provided by the pharmaceutical industry to regulatory authorities often do not present safety and efficacy data by sex. Novartis has started a gender-medicine project called MetaGeM, which includes nine observational studies sponsored by Novartis Farma, Italy; conducted in Italy between 2002 and 2013 in a range of different clinical areas. The MetaGeM project aims to analyze and describe by means of post hoc analyses and meta-analyses, clinical outcomes, therapeutic approaches, and safety data of these studies, by sex: PSYCHAE; GENDER ATTENTION in psoriasis; Synergy in psoriatic arthritis; ICEBERG in HBsAg carriers; SURF and CETRA in liverand renal transplanted patients, respectively; DEEP in Parkinson’s disease; and EVOLUTION and AXEPT in Alzheimer’s disease. The present paper describes the methodology of the MetaGeM project.
{"title":"A gender-medicine post hoc analysis (MetaGeM) project to test sex differences in previous observational studies in different diseases: methodology","authors":"D. Colombo, G. Bellia, D. Vassellatti, E. Zagni, S. Sgarbi, S. Rizzoli","doi":"10.2147/OAJCT.S67914","DOIUrl":"https://doi.org/10.2147/OAJCT.S67914","url":null,"abstract":"Only recently has medical research begun to understand the importance of taking sex into account, recognizing that symptoms and responses to medical treatment may be very different between males and females. However, the analyses provided by the pharmaceutical industry to regulatory authorities often do not present safety and efficacy data by sex. Novartis has started a gender-medicine project called MetaGeM, which includes nine observational studies sponsored by Novartis Farma, Italy; conducted in Italy between 2002 and 2013 in a range of different clinical areas. The MetaGeM project aims to analyze and describe by means of post hoc analyses and meta-analyses, clinical outcomes, therapeutic approaches, and safety data of these studies, by sex: PSYCHAE; GENDER ATTENTION in psoriasis; Synergy in psoriatic arthritis; ICEBERG in HBsAg carriers; SURF and CETRA in liverand renal transplanted patients, respectively; DEEP in Parkinson’s disease; and EVOLUTION and AXEPT in Alzheimer’s disease. The present paper describes the methodology of the MetaGeM project.","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"6 1","pages":"111-116"},"PeriodicalIF":1.2,"publicationDate":"2014-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/OAJCT.S67914","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68415116","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
: Clinical trials are one of the key study designs in the evolving field of comparative effectiveness research. Evaluating the effectiveness of interventions in real-world settings is complex and demands a rethinking of the traditional clinical trial approach as well as transformation of the clinical trial landscape. Novel strategies and refinement of existing approaches have been proposed to generate evidence that can guide health care stakeholders in their decision process. The purpose of this review is to discuss clinical trial design approaches in the era of comparative effectiveness research. We will focus on aspects relevant to the type of clinical trial, study population and recruitment, randomization process, outcome measures, and data collection.
{"title":"Clinical trial design in the era of comparative effectiveness research","authors":"Anke C. Winter, G. Colditz","doi":"10.2147/OAJCT.S39758","DOIUrl":"https://doi.org/10.2147/OAJCT.S39758","url":null,"abstract":": Clinical trials are one of the key study designs in the evolving field of comparative effectiveness research. Evaluating the effectiveness of interventions in real-world settings is complex and demands a rethinking of the traditional clinical trial approach as well as transformation of the clinical trial landscape. Novel strategies and refinement of existing approaches have been proposed to generate evidence that can guide health care stakeholders in their decision process. The purpose of this review is to discuss clinical trial design approaches in the era of comparative effectiveness research. We will focus on aspects relevant to the type of clinical trial, study population and recruitment, randomization process, outcome measures, and data collection.","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"6 1","pages":"101-110"},"PeriodicalIF":1.2,"publicationDate":"2014-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/OAJCT.S39758","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68414058","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
J. Moskowitz, A. Carrico, M. Cohn, Larissa G. Duncan, Cori Bussolari, Kristin Layous, J. Hult, A. Brousset, P. Cotten, Stephanie Maurer, Martha Pietrucha, M. Acree, J. Wrubel, Mallory O. Johnson, F. Hecht, S. Folkman
License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 85–100 Open Access Journal of Clinical Trials Dovepress
许可证。许可的完整条款可在http://creativecommons.org/licenses/by-nc/3.0/上获得。允许非商业用途的工作，没有任何进一步的许可，从多芬医学出版社有限公司，只要工作适当署名。超出许可范围的许可由多芬医疗新闻有限公司管理。有关如何请求许可的信息可在以下网站找到:http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 85-100 Open Access Journal of Clinical Trials Dovepress
{"title":"Randomized controlled trial of a positive affect intervention to reduce stress in people newly diagnosed with HIV; protocol and design for the IRISS study","authors":"J. Moskowitz, A. Carrico, M. Cohn, Larissa G. Duncan, Cori Bussolari, Kristin Layous, J. Hult, A. Brousset, P. Cotten, Stephanie Maurer, Martha Pietrucha, M. Acree, J. Wrubel, Mallory O. Johnson, F. Hecht, S. Folkman","doi":"10.2147/OAJCT.S64645","DOIUrl":"https://doi.org/10.2147/OAJCT.S64645","url":null,"abstract":"License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 85–100 Open Access Journal of Clinical Trials Dovepress","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"6 1","pages":"85-100"},"PeriodicalIF":1.2,"publicationDate":"2014-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/OAJCT.S64645","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68415085","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Shourav. Yathindranath, A. Kureishi, Simranjit Singh, Spencer Yeow, G. Geng, K. Wai, Audrey Ho, Elvira Zenaida Lansang, Ken J Lee
License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 75–84 Open Access Journal of Clinical Trials Dovepress
许可证。许可的完整条款可在http://creativecommons.org/licenses/by-nc/3.0/上获得。允许非商业用途的工作，没有任何进一步的许可，从多芬医学出版社有限公司，只要工作适当署名。超出许可范围的许可由多芬医疗新闻有限公司管理。有关如何申请许可的信息可在以下网站找到:http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 75-84 Open Access Journal of Clinical Trials Dovepress
{"title":"Evolution of the clinical trial landscape in Asia Pacific","authors":"Shourav. Yathindranath, A. Kureishi, Simranjit Singh, Spencer Yeow, G. Geng, K. Wai, Audrey Ho, Elvira Zenaida Lansang, Ken J Lee","doi":"10.2147/OAJCT.S57060","DOIUrl":"https://doi.org/10.2147/OAJCT.S57060","url":null,"abstract":"License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 75–84 Open Access Journal of Clinical Trials Dovepress","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"6 1","pages":"75-84"},"PeriodicalIF":1.2,"publicationDate":"2014-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/OAJCT.S57060","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68414862","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Sagita Dewi, Veronica Kristiansen, S. lindkær-Jensen, S. Larsen
Background: The most commonly used escalation methods in dose-finding studies have obvious weaknesses, and the Bayesian approach is difficult for clinicians to understand and to apply. The study aims were to introduce and assess the performance of clinically based response surface pathway (RSP) design for dose-finding studies, exemplified by one between-patient study, one within-patient study, and simulation studies. Methods: The between-patient study consisted of 15 women suffering from stage IV breast cancer, while the within-patient study consisted of seven female dogs with metastatic mammary cancer. The studies were conducted to determine the maximum tolerated dose (MTD) of a new anticancer agent named BP-C1 using three-level RSP designs. Adjustment of the dose from one design level to the next was based on a k -adjustment factor estimated to ensure coverage of the entire predefined dose window. Patient sequences with an equal number of patients as design levels were included in the between-patient design, whereas the same patients were included in all the design levels in the within-patient design. Results: Four of the five patient sequences in the between-patient study and all seven dogs in the within-patient study reached the upper limit of the dose windows without any increase in toxicity. The MTD of BP-C1 was thus found to be higher than the predefined cumulative dose window for both patient groups. In all three scenarios, the RSP design estimated MTDs better than the traditional 3 + 3 design; however, the toxicity rates were found to be higher when the target MTD was under the starting dose. Conclusion: The RSP designs do not need an assumed statistical model, and may be useful in estimating MTD, using a minimal sample size. The k -adjustment factor ensures complete dose window coverage and the design utilizes more information by allowing multinomial outcomes. Phase I studies are conducted sequentially, allocating dose levels to patients based on the observed toxicity effect from previous patients or treatment periods.
{"title":"Between- and within-patient n -level response surface pathway design in dose-finding studies","authors":"Sagita Dewi, Veronica Kristiansen, S. lindkær-Jensen, S. Larsen","doi":"10.2147/OAJCT.S57955","DOIUrl":"https://doi.org/10.2147/OAJCT.S57955","url":null,"abstract":"Background: The most commonly used escalation methods in dose-finding studies have obvious weaknesses, and the Bayesian approach is difficult for clinicians to understand and to apply. The study aims were to introduce and assess the performance of clinically based response surface pathway (RSP) design for dose-finding studies, exemplified by one between-patient study, one within-patient study, and simulation studies. Methods: The between-patient study consisted of 15 women suffering from stage IV breast cancer, while the within-patient study consisted of seven female dogs with metastatic mammary cancer. The studies were conducted to determine the maximum tolerated dose (MTD) of a new anticancer agent named BP-C1 using three-level RSP designs. Adjustment of the dose from one design level to the next was based on a k -adjustment factor estimated to ensure coverage of the entire predefined dose window. Patient sequences with an equal number of patients as design levels were included in the between-patient design, whereas the same patients were included in all the design levels in the within-patient design. Results: Four of the five patient sequences in the between-patient study and all seven dogs in the within-patient study reached the upper limit of the dose windows without any increase in toxicity. The MTD of BP-C1 was thus found to be higher than the predefined cumulative dose window for both patient groups. In all three scenarios, the RSP design estimated MTDs better than the traditional 3 + 3 design; however, the toxicity rates were found to be higher when the target MTD was under the starting dose. Conclusion: The RSP designs do not need an assumed statistical model, and may be useful in estimating MTD, using a minimal sample size. The k -adjustment factor ensures complete dose window coverage and the design utilizes more information by allowing multinomial outcomes. Phase I studies are conducted sequentially, allocating dose levels to patients based on the observed toxicity effect from previous patients or treatment periods.","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"6 1","pages":"63-74"},"PeriodicalIF":1.2,"publicationDate":"2014-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/OAJCT.S57955","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68414935","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
L. Ngo, A. Haeberle, J. Dyck-Jones, D. Gelmont, L. Yel
License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 55–61 Open Access Journal of Clinical Trials Dovepress
许可证。许可的完整条款可在http://creativecommons.org/licenses/by-nc/3.0/上获得。允许非商业用途的工作，没有任何进一步的许可，从多芬医学出版社有限公司，只要工作适当署名。超出许可范围的许可由多芬医疗新闻有限公司管理。有关如何申请许可的信息可在以下网站找到:http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 55-61 Open Access Journal of Clinical Trials Dovepress
{"title":"Safety and tolerability of an intravenously administered alpha1-proteinase inhibitor at an increased infusion rate: a novel, randomized, placebo-masked, infusion rate-controlled, crossover study in healthy adults","authors":"L. Ngo, A. Haeberle, J. Dyck-Jones, D. Gelmont, L. Yel","doi":"10.2147/OAJCT.S62754","DOIUrl":"https://doi.org/10.2147/OAJCT.S62754","url":null,"abstract":"License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 55–61 Open Access Journal of Clinical Trials Dovepress","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"6 1","pages":"55-61"},"PeriodicalIF":1.2,"publicationDate":"2014-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/OAJCT.S62754","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68414778","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
J. Pascual, T. Srinivas, S. Chadban, F. Citterio, F. Oppenheimer, H. Tedesco, M. Henry, C. Legendre, Y. Watarai, C. Sommerer, P. Lee, J. Hexham, Gaohong Dong, P. Bernhardt, F. Vincenti
Two well defined, modifiable risk factors for kidney allograft failure are acute rejection and poor graft function at one year post-transplant. Regulatory bodies and expert panels in the USA and Europe have recognized that both acute rejection and one-year graft function should be assessed when evaluating immunosuppressive regimens. TRANSFORM (Clinicaltrials.gov NCT01950819) is one of the first trials to adopt this approach and the first that applies a novel combined clinically meaningful endpoint to take the first step towards changing the paradigm for immunosuppression in kidney transplant patients. Everolimus with reduced-exposure calcineurin inhibitor (CNI) therapy is a strategy designed to reduce the risk of chronic nephrotoxicity and other dose-dependent complications associated with CNI therapy. In TRANSFORM, de novo kidney transplant patients are randomized to everolimus with reduced-exposure CNI, or mycophenolic acid with standard-exposure CNI, both with induction therapy and maintenance steroids. The primary endpoint is a composite of treated biopsy-proven acute rejection or estimated glomerular filtration rate ,50 mL/min/1.73 m 2 at month 12 post-transplant, which is expected to be sensitive both to the effects of acute and chronic allograft rejection and nephrotoxic side effects of immunosuppressive therapies. The construct of this endpoint allows the integration of a continuous outcome (graft function) with a logistic outcome (rejection). The trial uses a randomized, multicenter, open-label, two-arm design. After completion of a 2-year core study, patients enter a further 3-year prospective observational study. By capturing follow-up to 5 years, TRANSFORM will provide substantial data on the incidence of graft loss, graft dysfunction, cancer, cardiovascular events, and other patient-relevant outcomes. TRANSFORM will determine whether de novo CNI reduction with an everolimus-based regimen achieves short-term outcomes compared with standard CNI. As the largest clinical trial undertaken to date in kidney transplantation, recruiting more than 2,000 patients, and with extended follow-up to 5 years, TRANSFORM will provide critical data required to help maximize long-term outcomes.
{"title":"TrAnsFOrM: a novel study design to evaluate the effect of everolimus on long-term outcomes after kidney transplantation","authors":"J. Pascual, T. Srinivas, S. Chadban, F. Citterio, F. Oppenheimer, H. Tedesco, M. Henry, C. Legendre, Y. Watarai, C. Sommerer, P. Lee, J. Hexham, Gaohong Dong, P. Bernhardt, F. Vincenti","doi":"10.2147/OAJCT.S63058","DOIUrl":"https://doi.org/10.2147/OAJCT.S63058","url":null,"abstract":"Two well defined, modifiable risk factors for kidney allograft failure are acute rejection and poor graft function at one year post-transplant. Regulatory bodies and expert panels in the USA and Europe have recognized that both acute rejection and one-year graft function should be assessed when evaluating immunosuppressive regimens. TRANSFORM (Clinicaltrials.gov NCT01950819) is one of the first trials to adopt this approach and the first that applies a novel combined clinically meaningful endpoint to take the first step towards changing the paradigm for immunosuppression in kidney transplant patients. Everolimus with reduced-exposure calcineurin inhibitor (CNI) therapy is a strategy designed to reduce the risk of chronic nephrotoxicity and other dose-dependent complications associated with CNI therapy. In TRANSFORM, de novo kidney transplant patients are randomized to everolimus with reduced-exposure CNI, or mycophenolic acid with standard-exposure CNI, both with induction therapy and maintenance steroids. The primary endpoint is a composite of treated biopsy-proven acute rejection or estimated glomerular filtration rate ,50 mL/min/1.73 m 2 at month 12 post-transplant, which is expected to be sensitive both to the effects of acute and chronic allograft rejection and nephrotoxic side effects of immunosuppressive therapies. The construct of this endpoint allows the integration of a continuous outcome (graft function) with a logistic outcome (rejection). The trial uses a randomized, multicenter, open-label, two-arm design. After completion of a 2-year core study, patients enter a further 3-year prospective observational study. By capturing follow-up to 5 years, TRANSFORM will provide substantial data on the incidence of graft loss, graft dysfunction, cancer, cardiovascular events, and other patient-relevant outcomes. TRANSFORM will determine whether de novo CNI reduction with an everolimus-based regimen achieves short-term outcomes compared with standard CNI. As the largest clinical trial undertaken to date in kidney transplantation, recruiting more than 2,000 patients, and with extended follow-up to 5 years, TRANSFORM will provide critical data required to help maximize long-term outcomes.","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"6 1","pages":"45-54"},"PeriodicalIF":1.2,"publicationDate":"2014-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/OAJCT.S63058","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68414891","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Y. Sano, T. Fujii, T. Matsuda, Y. Oda, S. Kudo, M. Igarashi, H. Iishi, K. Kaneko, K. Hotta, N. Kobayashi, Yuichiro Yamaguchi, K. Kobayashi, H. Ishikawa, Y. Murakami, T. Shimoda, T. Fujimori, Y. Ajioka, H. Taniguchi, H. Ikematsu, K. Konishi, Yutaka Saito, S. Yoshida
License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 37–44 Open Access Journal of Clinical Trials Dovepress
许可证。许可的完整条款可在http://creativecommons.org/licenses/by-nc/3.0/上获得。允许非商业用途的工作，没有任何进一步的许可，从多芬医学出版社有限公司，只要工作适当署名。超出许可范围的许可由多芬医疗新闻有限公司管理。有关如何申请许可的信息可在以下网站找到:http://www.dovepress.com/permissions.php Open Access Journal of Clinical Trials 2014:6 37-44 Open Access Journal of Clinical Trials Dovepress
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