1. Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials.
- Author
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van den Berg, Leonard, Sorenson, Eric, Gronseth, Gary, Macklin, Eric, Andrews, Jinsy, Baloh, Robert, Benatar, Michael, Berry, James, Chio, Adriano, Corcia, Philippe, Genge, Angela, Gubitz, Amelie, Lomen-Hoerth, Catherine, McDermott, Christopher, Pioro, Erik, Rosenfeld, Jeffrey, Silani, Vincenzo, Turner, Martin, Weber, Markus, Brooks, Benjamin, Miller, Robert, and Mitsumoto, Hiroshi
- Subjects
Amyotrophic Lateral Sclerosis ,Biomarkers ,Clinical Trials as Topic ,Delphi Technique ,Guidelines as Topic ,Humans ,Outcome Assessment ,Health Care ,Patient Selection ,Research Design ,Statistics as Topic - 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.
- Published
- 2019