RT期刊文章SR电子T1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摩根富林明神经学神经学乔FD Lippincott Williams &威尔金斯SP e1610 OP e1623 10.1212 / WNL。首页92签证官0000000000007242是14 A1伦纳德h . van den Berg A1埃里克·索伦森A1加里Gronseth A1埃里克·a . Macklin A1 Jinsy安德鲁斯A1罗伯特·h·Baloh A1迈克尔·贝纳塔尔A1詹姆斯d·贝瑞A1阿德里亚诺亚蔡A1菲利普Corcia A1安吉拉其全称A1天使爱美丽k Gubitz A1凯瑟琳Lomen-Hoerth A1克里斯托弗·j·麦克德莫特A1埃里克·p·Pioro A1杰弗里·罗森菲尔德A1 Vincenzo Silani A1马丁·r·特纳A1马库斯·韦伯A1本杰明·里克斯布鲁克斯A1罗伯特·g·米勒A1 Hiroshi Mitsumoto A1的艾利房子肌萎缩性侧索硬化症临床试验指导集团1999年2019 UL //www.ez-admanager.com/content/92/14/e1610.abstract AB目的修改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)。首页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