Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials
Articolo
Data di Pubblicazione:
2019
Citazione:
Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials / L.H. van den Berg, E. Sorenson, G. Gronseth, E.A. Macklin, J. Andrews, R.H. Baloh, M. Benatar, J.D. Berry, A. Chio, P. Corcia, A. Genge, A.K. Gubitz, C. Lomen-Hoerth, C.J. Mcdermott, E.P. Pioro, J. Rosenfeld, V. Silani, M.R. Turner, M. Weber, B.R. Brooks, R.G. Miller, H. Mitsumoto. - In: NEUROLOGY. - ISSN 0028-3878. - 92:14(2019 Apr 02), pp. e1610-e1623. [10.1212/WNL.0000000000007242]
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.
Tipologia IRIS:
01 - Articolo su periodico
Elenco autori:
L.H. van den Berg, E. Sorenson, G. Gronseth, E.A. Macklin, J. Andrews, R.H. Baloh, M. Benatar, J.D. Berry, A. Chio, P. Corcia, A. Genge, A.K. Gubitz, C. Lomen-Hoerth, C.J. Mcdermott, E.P. Pioro, J. Rosenfeld, V. Silani, M.R. Turner, M. Weber, B.R. Brooks, R.G. Miller, H. Mitsumoto
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