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CS-121 APOC3 Base Editing in Severe Hypertriglyceridemia

NCT07657780 · Status: NOT_YET_RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2026-06-18

No results posted yet for this study

Summary

This is an open-label, single-arm, dose-escalation IIT clinical trial to evaluate the safety, tolerability, pharmacodynamics (PD), and pharmacokinetics (PK) of CS-121, an in vivo base editing therapy delivered by lipid nanoparticles targeting APOC3, in adult participants (18-65 years) with Severe Hypertriglyceridemia(sHTG).

Conditions

  • Severe Hypertriglyceridemia (sHTG)

Interventions

BIOLOGICAL

CS-121

CS-121 is a in vivo base editing therapy formulated in lipid nanoparticles for targeted editing of the APOC3 gene in hepatocytes.

Sponsors & Collaborators

  • The First Affiliated Hospital of Anhui Medical University

    collaborator OTHER

  • CorrectSequence Therapeutics Co., Ltd

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-15
Primary Completion
2028-03-15
Completion
2028-07-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07657780 on ClinicalTrials.gov