% 0期刊文章%伦纳德·h . van den Berg %埃里克·索伦森% Eric A . Macklin Gary Gronseth % %一个罗伯特·h·Baloh Jinsy安德鲁斯% %迈克尔·贝纳塔尔%一个詹姆斯D·贝瑞%阿德里亚诺亚蔡%菲利普Corcia %一个天使爱美丽安琪拉其全称% k . Gubitz %一个凯瑟琳Lomen-Hoerth %克里斯托弗·j·麦克德莫特%埃里克·p·Pioro %一个Vincenzo Silani杰弗里·罗森菲尔德% %一个马丁·R·特纳%马库斯·韦伯%本杰明·里克斯布鲁克斯% Hiroshi Mitsumoto罗伯特·g·米勒% %的艾利房子肌萎缩性侧索硬化症临床试验指导集团% T修订艾利房子共识肌萎缩性侧索硬化症的临床试验设计和实现指南% D R 10.1212 / WNL 2019%。0000000000007242 % J首页神经病学% P e1610-e1623 % V 92% N 14% X目的修改1999艾利房子共识指南的设计和实现临床前研究和临床试验治疗肌萎缩性脊髓侧索硬化症(ALS)。方法达成共识委员会由140国际ALS的关键成员社区(肌萎缩性侧索硬化症研究人员,临床医生、病人的代表,研究经费,代表行业和监管机构)解决9需要在ALS的研究领域:(1)临床前研究;(2)生物和表型异质性;(3)结果评价;(4)疾病和症状的干预措施;(5)招聘和保留;(6)生物标志物;(7)临床试验阶段;(8)超越传统的试验设计;和(9)统计方面的考虑。 Assigned to 1 of 8 sections, committee members generated a draft set of guidelines based on a “background” of developing a (pre)clinical question and a “rationale” outlining the evidence and expert opinion. Following a 2-day, face-to-face workshop at the Airlie House Conference Center, a modified Delphi process was used to develop draft consensus research guidelines, which were subsequently reviewed and modified based on comments from the public. Statistical experts drafted a separate document of statistical considerations (section 9).Results In this report, we summarize 112 guidelines and their associated backgrounds and rationales. The full list of guidelines, the statistical considerations, and a glossary of terms can be found in data available from Dryad (appendices e-3–e-5, doi.org/10.5061/dryad.32q9q5d). The authors prioritized 15 guidelines with the greatest potential to improve ALS clinical research.Conclusion The revised Airlie House ALS Clinical Trials Consensus Guidelines should serve to improve clinical trial design and accelerate the development of effective treatments for patients with ALS.ALS=amyotrophic lateral sclerosis; NIV=noninvasive ventilation; PALS=people living with amyotrophic lateral sclerosis; QOL=quality of life %U //www.ez-admanager.com/content/neurology/92/14/e1610.full.pdf