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

Leonard H. Van Den Berg, Eric James Sorenson, Gary Gronseth, Eric A. Macklin, Jinsy Andrews, Robert H. Baloh, Michael Benatar, James D. Berry, Adriano Chio, Philippe Corcia, Angela Genge, Amelie K. Gubitz, Catherine Lomen-Hoerth, Christopher J. Mcdermott, Erik P. Pioro, Jeffrey Rosenfeld, Vincenzo Silani, Martin R. Turner, Markus Weber, Benjamin Rix BrooksRobert G. Miller, Hiroshi Mitsumoto

Research output: Contribution to journalArticle

8 Citations (Scopus)

Abstract

ObjectiveTo revise the 1999 Airlie House consensus guidelines for the design and implementation of preclinical therapeutic studies and clinical trials in amyotrophic lateral sclerosis (ALS).MethodsA 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).ResultsIn 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.ConclusionThe 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.

Original languageEnglish (US)
Pages (from-to)E1610-E1623
JournalNeurology
Volume92
Issue number14
DOIs
StatePublished - Apr 2 2019

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Amyotrophic Lateral Sclerosis
Clinical Trials
Guidelines
Research
Committee Membership
Patient Advocacy
Expert Testimony
Industry
Biomarkers
Research Personnel
Outcome Assessment (Health Care)
Education
Therapeutics

ASJC Scopus subject areas

  • Clinical Neurology

Cite this

Van Den Berg, L. H., Sorenson, E. J., Gronseth, G., Macklin, E. A., Andrews, J., Baloh, R. H., ... Mitsumoto, H. (2019). Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials. Neurology, 92(14), E1610-E1623. https://doi.org/10.1212/WNL.0000000000007242

Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials. / Van Den Berg, Leonard H.; Sorenson, Eric James; Gronseth, Gary; Macklin, Eric A.; Andrews, Jinsy; Baloh, Robert H.; Benatar, Michael; Berry, James D.; Chio, Adriano; Corcia, Philippe; Genge, Angela; Gubitz, Amelie K.; Lomen-Hoerth, Catherine; Mcdermott, Christopher J.; Pioro, Erik P.; Rosenfeld, Jeffrey; Silani, Vincenzo; Turner, Martin R.; Weber, Markus; Brooks, Benjamin Rix; Miller, Robert G.; Mitsumoto, Hiroshi.

In: Neurology, Vol. 92, No. 14, 02.04.2019, p. E1610-E1623.

Research output: Contribution to journalArticle

Van Den Berg, LH, Sorenson, EJ, 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, RG & Mitsumoto, H 2019, 'Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials', Neurology, vol. 92, no. 14, pp. E1610-E1623. https://doi.org/10.1212/WNL.0000000000007242
Van Den Berg, Leonard H. ; Sorenson, Eric James ; Gronseth, Gary ; Macklin, Eric A. ; Andrews, Jinsy ; Baloh, Robert H. ; Benatar, Michael ; Berry, James D. ; Chio, Adriano ; Corcia, Philippe ; Genge, Angela ; Gubitz, Amelie K. ; Lomen-Hoerth, Catherine ; Mcdermott, Christopher J. ; Pioro, Erik P. ; Rosenfeld, Jeffrey ; Silani, Vincenzo ; Turner, Martin R. ; Weber, Markus ; Brooks, Benjamin Rix ; Miller, Robert G. ; Mitsumoto, Hiroshi. / Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials. In: Neurology. 2019 ; Vol. 92, No. 14. pp. E1610-E1623.
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AU - Van Den Berg, Leonard H.

AU - Sorenson, Eric James

AU - Gronseth, Gary

AU - Macklin, Eric A.

AU - Andrews, Jinsy

AU - Baloh, Robert H.

AU - Benatar, Michael

AU - Berry, James D.

AU - Chio, Adriano

AU - Corcia, Philippe

AU - Genge, Angela

AU - Gubitz, Amelie K.

AU - Lomen-Hoerth, Catherine

AU - Mcdermott, Christopher J.

AU - Pioro, Erik P.

AU - Rosenfeld, Jeffrey

AU - Silani, Vincenzo

AU - Turner, Martin R.

AU - Weber, Markus

AU - Brooks, Benjamin Rix

AU - Miller, Robert G.

AU - Mitsumoto, Hiroshi

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N2 - ObjectiveTo revise the 1999 Airlie House consensus guidelines for the design and implementation of preclinical therapeutic studies and clinical trials in amyotrophic lateral sclerosis (ALS).MethodsA 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).ResultsIn 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.ConclusionThe 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.

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