NCT07491172

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 90 (estimated)
• Sponsor: CRISPR Therapeutics AG · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Cardiovascular
• Metabolic Disease
• Dyslipidemias
• Lipid Disorder
• Hypertriglyceridemia
• Heterozygous Familial Hypercholesterolemia (HeFH)
• Homozygous Familial Hypercholesterolemia (HoFH)
• Severe Hypertriglyceridemia (sHTG)
• Mixed Hyperlipemia
• Hypercholesterolaemia

Interventions

Type	Name	Description
DRUG	CTX310	CTX310 is a lipid nanoparticle (LNP) formulation of clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) components for in vivo editing of the target gene angiopoietin-like 3 (ANGPTL3).

Timeline

• Start date: 2024-06-21
• Primary completion: 2027-06-01
• Completion: 2028-06-01
• First posted: 2026-03-24
• Last updated: 2026-03-24

Locations

18 sites across 4 countries: United States, Australia, New Zealand, United Kingdom

Regulatory

• FDA-regulated drug study