PT -期刊文章AU -伦纳德h . van den Berg Gary Gronseth AU -埃里克·索伦森盟盟-埃里克·a·Macklin盟Jinsy安德鲁斯AU -罗伯特·h·Baloh AU -迈克尔·贝纳塔尔AU -詹姆斯d·贝瑞盟-阿德里亚诺亚蔡AU - Philippe Corcia盟安琪拉其全称盟-天使爱美丽k Gubitz AU -凯瑟琳Lomen-Hoerth AU -克里斯托弗·j·麦克德莫特AU -埃里克·p·Pioro AU -杰弗里·罗森菲尔德盟Vincenzo Silani AU -马丁·r·特纳AU -马库斯·韦伯盟-本杰明·里克斯布鲁克斯AU -罗伯特·g·米勒盟Hiroshi Mitsumoto AU -艾利房子肌萎缩性侧索硬化症临床试验指导集团TI -修订后的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- 10.1212 / WNL援助。0000000000007242 DP - 2019年4月02 TA -神经病首页学PG - e1610 e1623 VI - 92 IP - 14 4099 - //www.ez-admanager.com/content/92/14/e1610.short 4100 - //www.ez-admanager.com/content/92/14/e1610.full所以Neurology2019 4月02;92 AB -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; and (9) statistical considerations. 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