CS-121 APOC3 Base Editing in FCS

Recruiting Early Phase 1 Interventional Study
Familial Chylomicronemia Syndrome (FCS)
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
18 – 55
Sex
Any
Trial phase
Early Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
15 (estimated)
Sponsor
CorrectSequence Therapeutics Co., Ltd · Industry
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About This Trial

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).

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Male or female aged 18 to 55 years (inclusive) at the time of signing informed consent. * On regular standard therapy with good compliance, but fasting triglyceride (TG) levels have not been consistently reduced below 10 mmol/L (880 mg/dL); i.e., before screening, there must b…
Contacts

Yaliang Li

+8618621046122

yaliang.li@correctsequence.com

CONTACT