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RECRUITING
NCT07371767
EARLY_PHASE1

CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia

Sponsor: Shanghai Jiao Tong University School of Medicine

View on ClinicalTrials.gov

Summary

This is a Prospective, Single-center, Open-label, Single-arm Clinical Study to Evaluate the Safety and Efficacy of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3, in Children and Adolescents (4-18 years) With Hyperchylomicronemia

Official title: A Prospective, Single-center, Open-label, Single-arm Clinical Study to Evaluate the Safety and Efficacy of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3, in Children and Adolescents With Hyperchylomicronemia

Key Details

Gender

All

Age Range

4 Years - 18 Years

Study Type

INTERVENTIONAL

Enrollment

15

Start Date

2026-01-26

Completion Date

2041-01-31

Last Updated

2026-01-28

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.

Locations (1)

Shanghai Children's Medical Center

Shanghai, Shanghai Municipality, China