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

NCT07371767 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2026-01-28

No results posted yet for this study

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

Conditions

  • Hyperchylomicronemia

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

  • CorrectSequence Therapeutics Co., Ltd

    collaborator INDUSTRY

  • Shanghai Jiao Tong University School of Medicine

    lead OTHER

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

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

Timeline & Regulatory

Start
2026-01-26
Primary Completion
2027-10-31
Completion
2041-01-31

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 NCT07371767 on ClinicalTrials.gov