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

Recruiting Early Phase 1 Interventional Study
Hyperchylomicronemia
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
4 – 18
Sex
Any
Trial phase
Early Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
15 (estimated)
Sponsor
Shanghai Jiao Tong University School of Medicine · Other
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About This Trial

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

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Male or female participants aged 4 years ≤ age \< 18 years. * Severe hypertriglyceridemia (sHTG), defined as a triglyceride (TG) level ≥ 500 mg/dL. * Confirmed diagnosis of genetically inherited FCS via genetic testing, or clinically diagnosed FCS plus persistent chylomicronem…
Contacts

Guoying Chang, PhD

86+15000394881

changguoying@126.com

CONTACT