2026 Volume 43 Issue 3 Pages 363-365
An AMED–funded project entitled “Study to develop guidelines for clinical evaluation of amyotrophic lateral sclerosis therapeutics” has been ongoing in Japan since 2024 to facilitate multi–regional clinical trials in ALS. The guideline is being developed in collaboration with key stakeholders, including researchers, regulatory authorities, pharmaceutical companies, and patients with ALS. It addresses : (1) fundamental principles of clinical trial design ; (2) target populations ; (3) efficacy assessment ; (4) statistical considerations ; (5) biomarkers ; (6) safety assessment and adverse events ; (7) use of concomitant medications and therapies ; (8) participant data collection and secondary use of existing data ; (9) clinical trial sites ; and (10) clinical trials for familial ALS and related conditions. The guideline will be published in both Japanese and English in 2026.
