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.
Age of ≥18 and ≤75 years at the time of signing the informed consent.
Able to provide written informed consent.
Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg/dL - and LDL-C ≥70 mg/dL in participants with ASCVD, or LDL-C ≥70 or 100mg/dL in participants with or without ASCVD respectively, or TG ≥500 mg/dL.
Refractory to the maximal intensity or MTD of standard of care lines of lipid-lowering therapies available through routine clinical care, for at least 12 weeks prior to screening
Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.
Abnormal or compromised function of kidney, heart, blood or liver.
Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1.
Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran).
Single group
CTX310
DrugCTX310 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).
To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments
Incidence of dose-limiting toxicities and frequency of adverse events
To assess the preliminary efficacy of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Percentage change from baseline in lipid values (LDL-C, non-HDL-C, Trigs, apoB and HDL-C)
To further characterize the safety of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Frequency and severity of adverse events (AE), including treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESIs), clinically significant laboratory abnormalities, and clinically significant abnormal vital signs.
To assess the pharmacokinetics (PK) of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Levels of CTX310 in blood over time
To assess the pharmacodynamic (PD) response of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Percentage change from baseline of ANGPTL3
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