Tundra Space

Tundra Space

Clinical Research Directory

Browse clinical research sites, groups, and studies.

Back to Studies
RECRUITING
NCT07176923
EARLY_PHASE1

CS-121 APOC3 Base Editing in FCS

Sponsor: CorrectSequence Therapeutics Co., Ltd

View on ClinicalTrials.gov

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).

Official title: A Clinical Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3, in Adults With Familial Chylomicronemia Syndrome

Key Details

Gender

All

Age Range

18 Years - 55 Years

Study Type

INTERVENTIONAL

Enrollment

15

Start Date

2025-10-15

Completion Date

2026-12

Last Updated

2026-02-11

Healthy Volunteers

No

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).

Locations (1)

The First Affiliated Hospital of Anhui Medical University

Hefei, Anhui, China