Randomized Phase I Trials - One Size Fits All?
rct · Level II
Where this comes from
- Record sourced from PubMed, PMID 38320508.
- Also identified by DOI 10.1056/EVIDe2300282.
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Abstract
In this issue of <i>NEJM Evidence</i>, Gaudet et al. present the safety profile and pharmacodynamics of ARO-APOC3, a small interfering RNA therapeutic that inhibits apolipoprotein C-III (APOC3) mRNA expression in a phase I trial.<sup>1</sup> Assignment to treatment was based on fasting levels of triglycerides. The trial included two double-blinded cohorts with 52 randomly assigned healthy participants and 40 patients with hypertriglyceridemia assigned to escalating doses of ARO-APOC3 at 10, 25, 50, or 100 mg or placebo in a single- and/or repeat-dose (days 1 and 29) regimen. An open-label cohort of patients with chylomicronemia was treated with ARO-APOC3 at 50 mg.
Medical subject headings
- Triglycerides