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

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, committe

SUBMITTER: van den Berg LH 

PROVIDER: S-EPMC6448453 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature