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Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials.

Abstract：

OBJECTIVE:To revise the 1999 Airlie House consensus guidelines for the design and implementation of preclinical therapeutic studies and clinical trials in amyotrophic lateral sclerosis (ALS). METHODS:A consensus committee comprising 140 key members of the international ALS community (ALS researchers, clinicians, patient representatives, research funding representatives, industry, and regulatory agencies) addressed 9 areas of need within ALS research: (1) preclinical studies; (2) biological and phenotypic heterogeneity; (3) outcome measures; (4) disease-modifying and symptomatic interventions; (5) recruitment and retention; (6) biomarkers; (7) clinical trial phases; (8) beyond traditional trial designs; 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.

journal_name

Neurology

journal_title

Neurology

authors

van den Berg LH,Sorenson E,Gronseth G,Macklin EA,Andrews J,Baloh RH,Benatar M,Berry JD,Chio A,Corcia P,Genge A,Gubitz AK,Lomen-Hoerth C,McDermott CJ,Pioro EP,Rosenfeld J,Silani V,Turner MR,Weber M,Brooks BR,Miller

doi

10.1212/WNL.0000000000007242

subject

Has Abstract

pub_date

2019-04-02 00:00:00

pages

e1610-e1623

issue

eissn

0028-3878

issn

1526-632X

pii

WNL.0000000000007242

journal_volume

pub_type

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Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials.