Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials
<br/><strong>Objective: </strong>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).<br/><strong>Methods: </strong>A consensus committ...
| Prif Awduron: | , , , , , , , , , , , , , , , , , , , , , |
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| Fformat: | Journal article |
| Iaith: | English |
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Wolters Kluwer Health
2019
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| _version_ | 1826275277024526336 |
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| author | Van Den Berg, L Sorenson, E Gronseth, G Macklin, E Andrews, J Baloh, R Benatar, M Berry, J Chio, A Corcia, P Genge, A Gubitz, A Lomen-Hoerth, C McDermott, C Pioro, E Rosenfeld, J Silani, V Turner, M Weber, M Brooks, B Miller, R Mitsumoto, H |
| author_facet | Van Den Berg, L Sorenson, E Gronseth, G Macklin, E Andrews, J Baloh, R Benatar, M Berry, J Chio, A Corcia, P Genge, A Gubitz, A Lomen-Hoerth, C McDermott, C Pioro, E Rosenfeld, J Silani, V Turner, M Weber, M Brooks, B Miller, R Mitsumoto, H |
| author_sort | Van Den Berg, L |
| collection | OXFORD |
| description | <br/><strong>Objective: </strong>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).<br/><strong>Methods: </strong>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).<br/><strong>Results: </strong>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.<br/><strong>Conclusion: </strong>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. |
| first_indexed | 2024-03-06T22:56:15Z |
| format | Journal article |
| id | oxford-uuid:607db8d4-7c06-4dd1-8bb4-c06539baaca3 |
| institution | University of Oxford |
| language | English |
| last_indexed | 2024-03-06T22:56:15Z |
| publishDate | 2019 |
| publisher | Wolters Kluwer Health |
| record_format | dspace |
| spelling | oxford-uuid:607db8d4-7c06-4dd1-8bb4-c06539baaca32022-03-26T17:53:43ZRevised Airlie House consensus guidelines for design and implementation of ALS clinical trialsJournal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:607db8d4-7c06-4dd1-8bb4-c06539baaca3EnglishSymplectic Elements at OxfordWolters Kluwer Health2019Van Den Berg, LSorenson, EGronseth, GMacklin, EAndrews, JBaloh, RBenatar, MBerry, JChio, ACorcia, PGenge, AGubitz, ALomen-Hoerth, CMcDermott, CPioro, ERosenfeld, JSilani, VTurner, MWeber, MBrooks, BMiller, RMitsumoto, H<br/><strong>Objective: </strong>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).<br/><strong>Methods: </strong>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).<br/><strong>Results: </strong>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.<br/><strong>Conclusion: </strong>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. |
| spellingShingle | Van Den Berg, L Sorenson, E Gronseth, G Macklin, E Andrews, J Baloh, R Benatar, M Berry, J Chio, A Corcia, P Genge, A Gubitz, A Lomen-Hoerth, C McDermott, C Pioro, E Rosenfeld, J Silani, V Turner, M Weber, M Brooks, B Miller, R Mitsumoto, H Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials |
| title | Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials |
| title_full | Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials |
| title_fullStr | Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials |
| title_full_unstemmed | Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials |
| title_short | Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials |
| title_sort | revised airlie house consensus guidelines for design and implementation of als clinical trials |
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