clinical trial · NCT07491172
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
CardiovascularMetabolic DiseaseDyslipidemiasLipid DisorderHypertriglyceridemiaHeterozygous Familial Hypercholesterolemia (HeFH)Homozygous Familial Hypercholesterolemia (HoFH)Severe Hypertriglyceridemia (sHTG)CTX310
brief summary
This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.
started
Jun 21, 2024
primary completion
Jun 1, 2027
completion
Jun 1, 2028
last updated
Mar 24, 2026
detailed description
This is a phase 1, open-label, multi-center study of CTX310 in participants with refractory dyslipidemias. Subjects will receive a dose of CTX310 via intravenous (IV) infusion.
official title
A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Participants With Refractory Dyslipidemias
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol