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CS-121 APOC3 Base Editing in FCS

NCT07176923 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2026-02-11

No results posted yet for this study

Summary

This is an open-label, single-arm, dose-escalation Phase I 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 (18-55 years) with familial chylomicronemia syndrome (FCS).

Conditions

  • Familial Chylomicronemia Syndrome (FCS)

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. In this study, participants receive a single intravenous infusion of CS-121 at escalating dose levels. The investigational product is designed to reduce ApoC3 protein expression and serum triglyceride levels in adults with familial chylomicronemia syndrome (FCS).

Sponsors & Collaborators

  • The First Affiliated Hospital of Anhui Medical University

    collaborator OTHER

  • CorrectSequence Therapeutics Co., Ltd

    lead INDUSTRY

Principal Investigators

  • YALIANG LI · CorrectSequence Therapeutics (Shanghai) Co., Ltd

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

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

Timeline & Regulatory

Start
2025-10-15
Primary Completion
2026-12-31
Completion
2026-12-31

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 NCT07176923 on ClinicalTrials.gov