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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
90
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).
Research Site 10
Jacksonville, Florida, United States
NOT_YET_RECRUITINGResearch Site 17
Orlando, Florida, United States
NOT_YET_RECRUITINGResearch Site 11
Port Orange, Florida, United States
NOT_YET_RECRUITINGResearch Site 14
Durham, North Carolina, United States
NOT_YET_RECRUITINGResearch Site 13
High Point, North Carolina, United States
NOT_YET_RECRUITINGResearch Site 9
Cleveland, Ohio, United States
NOT_YET_RECRUITINGResearch Site 12
Portland, Oregon, United States
NOT_YET_RECRUITINGResearch Site 1
Adelaide, Australia
RECRUITINGResearch Site 15
Brisbane, Australia
NOT_YET_RECRUITINGResearch Site 16
Brisbane, Australia
NOT_YET_RECRUITING...and 8 more locations
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
Time frame: From CTX310 infusion up to 12 months
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)
Time frame: Over 12 months, compared to baseline
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.
Time frame: From CTX310 infusion up to 12 months
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
Time frame: From CTX310 infusion up to 12 months
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
Time frame: Over 12 months, compared to baseline
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