Data from: Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials

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 nine areas of
need within ALS research: 1. Pre-clinical 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 one of eight sections, committee
members generated a draft set of recommendations based on a “background”
of developing a (pre)clinical question and a “rationale” outlining the
evidence and expert opinion. Following a two-day, face-to-face workshop at
the Airlie House Conference Center, a modified Delphi process was used to
develop draft consensus 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 paper we summarize 112 recommendations and their associated
backgrounds and rationales. The full list of recommendations on all main
topics as well as on statistical considerations are available (Appendices
e-3 and e-4). The authors prioritized 15 recommendations 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.