Phase 2B randomized controlled trial of NP001 in amyotrophic lateral sclerosis: Pre-specified and post hoc analyses.
Robert G MillerRongzhen ZhangPaige M BracciAri AzhirRichard BarohnRichard BedlackMichael BenatarJames D BerryMerit CudkowiczEdward J KasarskisHiroshi MitsumotoGeorgios ManousakisDavid WalkBjorn OskarssonJeremy ShefnerMichael S McGrathPublished in: Muscle & nerve (2022)
Although the phase 2B trial failed to meet its primary endpoints, post hoc analyses identified a subgroup whose decline in ALSFRS-R and VC scores were significantly slower than placebo. Further studies will be required to validate these findings.