Actively Recruiting
Phase 1 Study to Evaluate the Safety of CTX310, a CRISPR-Based Treatment, for Adults 18 to 75 Years Old with Hard-to-Treat Dyslipidemias
Led by CRISPR Therapeutics AG · Updated on 2026-07-23
90
Participants Needed
19
Research Sites
52 weeks
Total Duration
AI-Summary
What this Trial Is About
Researchers are studying CTX310, a new gene-editing treatment, in adults aged 18 to 75 with dyslipidemias that do not respond to current therapies. This early-phase, open-label trial aims to evaluate the safety and tolerability of CTX310, a lipid nanoparticle formulation designed to edit the ANGPTL3 gene involved in lipid regulation. Participants will receive a single intravenous infusion of CTX310. The study is conducted across multiple centers and follows an ascending dose design to assess safety at different dose levels. The treatment period includes monitoring for up to 12 months after infusion to evaluate safety, pharmacokinetics, pharmacodynamics, and preliminary effects on lipid levels. During the study, participants will have regular assessments including laboratory tests to monitor lipid levels and organ function, safety evaluations, and pharmacological measurements. Researchers will track adverse events and treatment effects for up to one year following the infusion. Participants must adhere to study visits and contraception requirements during this time to ensure accurate monitoring and safety.
CONDITIONS
Brief Title
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
Research Team
C
Clinical Trials
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