Diego Garcia-Borreguero , Jed Black , Christopher J. Earley , Stephany Fulda , Birgit Högl , Mauro Manconi , William Ondo , Thomas Roth , Claudia Trenkwalder , John W. Winkelman , on behalf of the International Restless Legs Syndrome Study Group (IRLSSG)
{"title":"重新思考不宁腿综合征的临床试验:路线图","authors":"Diego Garcia-Borreguero , Jed Black , Christopher J. Earley , Stephany Fulda , Birgit Högl , Mauro Manconi , William Ondo , Thomas Roth , Claudia Trenkwalder , John W. Winkelman , on behalf of the International Restless Legs Syndrome Study Group (IRLSSG)","doi":"10.1016/j.smrv.2024.101978","DOIUrl":null,"url":null,"abstract":"<div><p>The number of large clinical trials of restless legs syndrome (RLS) have decreased in recent years, this coincides with reduced interest in developing and testing novel pharmaceuticals. Therefore, the International Restless Legs Syndrome Study Group (IRLSSG) formed a task force of global experts to examine the causes of these trends and make recommendations to facilitate new clinical trials. In our article, we delve into potential complications linked to the diagnostic definition of RLS, identify subpopulations necessitating more attention, and highlight issues pertaining to endpoints and study frameworks. In particular, we recommend developing alternative scoring methods for more accurate RLS diagnosis, thereby improving clinical trial specificity. Furthermore, enhancing the precision of endpoints will increase study effect sizes and mitigate study costs. Suggestions to achieve this include developing online, real-time sleep diaries with high-frequency sampling of nightly sleep latency and the use of PLMs as surrogate markers. Furthermore, to reduce the placebo response, strategies should be adopted that include placebo run-in periods. As RLS is frequently a chronic condition, priority should be given to long-term studies, using a randomized, placebo-controlled, withdrawal design. Lastly, new populations should be investigated to develop targeted treatments such as mild RLS, pregnancy, hemodialysis, or iron-deficient anemia.</p></div>","PeriodicalId":49513,"journal":{"name":"Sleep Medicine Reviews","volume":"77 ","pages":"Article 101978"},"PeriodicalIF":11.2000,"publicationDate":"2024-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Rethinking clinical trials in restless legs syndrome: A roadmap\",\"authors\":\"Diego Garcia-Borreguero , Jed Black , Christopher J. Earley , Stephany Fulda , Birgit Högl , Mauro Manconi , William Ondo , Thomas Roth , Claudia Trenkwalder , John W. Winkelman , on behalf of the International Restless Legs Syndrome Study Group (IRLSSG)\",\"doi\":\"10.1016/j.smrv.2024.101978\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><p>The number of large clinical trials of restless legs syndrome (RLS) have decreased in recent years, this coincides with reduced interest in developing and testing novel pharmaceuticals. Therefore, the International Restless Legs Syndrome Study Group (IRLSSG) formed a task force of global experts to examine the causes of these trends and make recommendations to facilitate new clinical trials. In our article, we delve into potential complications linked to the diagnostic definition of RLS, identify subpopulations necessitating more attention, and highlight issues pertaining to endpoints and study frameworks. In particular, we recommend developing alternative scoring methods for more accurate RLS diagnosis, thereby improving clinical trial specificity. Furthermore, enhancing the precision of endpoints will increase study effect sizes and mitigate study costs. Suggestions to achieve this include developing online, real-time sleep diaries with high-frequency sampling of nightly sleep latency and the use of PLMs as surrogate markers. Furthermore, to reduce the placebo response, strategies should be adopted that include placebo run-in periods. As RLS is frequently a chronic condition, priority should be given to long-term studies, using a randomized, placebo-controlled, withdrawal design. Lastly, new populations should be investigated to develop targeted treatments such as mild RLS, pregnancy, hemodialysis, or iron-deficient anemia.</p></div>\",\"PeriodicalId\":49513,\"journal\":{\"name\":\"Sleep Medicine Reviews\",\"volume\":\"77 \",\"pages\":\"Article 101978\"},\"PeriodicalIF\":11.2000,\"publicationDate\":\"2024-07-18\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Sleep Medicine Reviews\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S1087079224000820\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Sleep Medicine Reviews","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1087079224000820","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
Rethinking clinical trials in restless legs syndrome: A roadmap
The number of large clinical trials of restless legs syndrome (RLS) have decreased in recent years, this coincides with reduced interest in developing and testing novel pharmaceuticals. Therefore, the International Restless Legs Syndrome Study Group (IRLSSG) formed a task force of global experts to examine the causes of these trends and make recommendations to facilitate new clinical trials. In our article, we delve into potential complications linked to the diagnostic definition of RLS, identify subpopulations necessitating more attention, and highlight issues pertaining to endpoints and study frameworks. In particular, we recommend developing alternative scoring methods for more accurate RLS diagnosis, thereby improving clinical trial specificity. Furthermore, enhancing the precision of endpoints will increase study effect sizes and mitigate study costs. Suggestions to achieve this include developing online, real-time sleep diaries with high-frequency sampling of nightly sleep latency and the use of PLMs as surrogate markers. Furthermore, to reduce the placebo response, strategies should be adopted that include placebo run-in periods. As RLS is frequently a chronic condition, priority should be given to long-term studies, using a randomized, placebo-controlled, withdrawal design. Lastly, new populations should be investigated to develop targeted treatments such as mild RLS, pregnancy, hemodialysis, or iron-deficient anemia.
期刊介绍:
Sleep Medicine Reviews offers global coverage of sleep disorders, exploring their origins, diagnosis, treatment, and implications for related conditions at both individual and public health levels.
Articles comprehensively review clinical information from peer-reviewed journals across various disciplines in sleep medicine, encompassing pulmonology, psychiatry, psychology, physiology, otolaryngology, pediatrics, geriatrics, cardiology, dentistry, nursing, neurology, and general medicine.
The journal features narrative reviews, systematic reviews, and editorials addressing areas of controversy, debate, and future research within the field.