CS-121 APOC3 Base Editing in FCS
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-55
SponsorCorrectSequence Therapeutics Co., Ltd
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).
Eligibility criteria
Qualifiers
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 be records of at least three separate fasting TG values >10 mmol/L (880 mg/dL), or the participant is intolerant to standard therapy.
North American Familial Chylomicronemia Syndrome (NAFCS) score ≥45
Able to sign informed consent and comply with the requirements and restrictions specified in the informed consent form and the protocol, such as dietary guidance and intake restrictions.
Disqualifiers
Currently participating in another interventional clinical study, or last use of another investigational product with a washout period of less than 5 half-lives or 30 days (whichever is longer).
Use of APOC3-targeted antisense oligonucleotides (ASO) or siRNA lipid-lowering agents within 6 months prior to dosing.
History of acute pancreatitis within 3 months before dosing.
History of acute coronary syndrome (ACS) within 6 months before dosing, such as myocardial infarction or unstable angina, or prior coronary revascularization (e.g., coronary artery bypass grafting, angioplasty, or stent implantation).
Trial design
Treatments tested in this trial
- CS-121
Treatment groups
15 Participants
are divided into 5 treatment groupsSponsors and collaborators
CorrectSequence Therapeutics Co., Ltd
Lead sponsor
The First Affiliated Hospital of Anhui Medical University
Collaborator
Source ClinicalTrials.gov