Actively Recruiting
CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia
Led by Shanghai Jiao Tong University School of Medicine · Updated on 2026-01-28
15
Participants Needed
1
Research Sites
783 weeks
Total Duration
On this page
Sponsors
S
Shanghai Jiao Tong University School of Medicine
Lead Sponsor
C
CorrectSequence Therapeutics Co., Ltd
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female participants aged 4 years 64 age < 18 years
- Severe hypertriglyceridemia with triglyceride levels 65 500 mg/dL
- Confirmed diagnosis of genetically inherited familial chylomicronemia syndrome (FCS) or clinically diagnosed FCS with persistent chylomicronemia
- Inadequate triglyceride control despite prior lipid-lowering therapy (as determined by the investigator for participants under 8 years)
- Participants aged 6 years and above must sign the informed consent form themselves; parent/legal guardian consent required for participants under 18 years
- Female participants of childbearing potential must have a negative serum pregnancy test
You will not qualify if you...
- Participation in other interventional clinical studies or insufficient washout period since last investigational drug
- Use of antisense oligonucleotide (ASO) or siRNA-based lipid-lowering drugs targeting APOC3 within 3 months prior to study drug administration
- History of acute pancreatitis within 1 month before dosing
- Major surgery within 3 months prior to dosing or judged unsuitable for study drug by investigator
- ALT or AST 65 2 times upper limit of normal (ULN)
- Total bilirubin 65 1.5 times ULN
- Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m 2
- Random urine albumin-to-creatinine ratio >30 mg/g or urine protein 65 2+
- HbA1c 65 9%
- Coagulation function abnormalities unsuitable for CS-121 administration
- Positive for hepatitis B surface antigen, hepatitis C antibody and RNA, HIV, or Treponema pallidum infection
- Major organ diseases, mental disorders, Cushing's syndrome, hypothyroidism, lymphoproliferative disorders, or malignant tumors deemed unsuitable by investigator
- Concomitant medications or treatments affecting lipid metabolism, liver/kidney function, coagulation, or study drug efficacy evaluation
- Planning pregnancy, breastfeeding, or fertility plans during study
- History of hypersensitivity to study drug or similar drugs
- Other medical conditions interfering with study compliance or data interpretation
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Shanghai Children's Medical Center
Shanghai, Shanghai Municipality, China
Actively Recruiting
Research Team
G
Guoying Chang, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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