ARO-HSD

Evidence outlook · lead readout

NCT04202354 · Non-alcoholic Steatohepatitis

The reported results come from study designs that frequently fail to replicate under controlled conditions — see the rationale below.

NCT04202354: This is a small early-phase dose-escalation study: only the healthy-volunteer cohorts were randomized, quadruple-blinded, and placebo-controlled (4 participants per ARO-HSD dose vs 16 pooled placebo), while the suspected-NASH cohorts were open-label with no comparator (6 per dose). The primary endpoint was a safety endpoint — the number of treatment-related adverse events — reported as descriptive counts with no statistical analysis, and the secondary endpoints were pharmacokinetic measurements. With a total of 50 participants and no efficacy endpoint (no histology, imaging, or clinical outcome in NASH), the trial is far too small and early-phase to provide evidence of clinical benefit.