This is an open-label, single-arm, dose-escalation IIT 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 (18-65 years) with Severe Hypertriglyceridemia(sHTG).
Male or female participants aged 18 years ≤ age < 65 years.
The serum triglyceride levels of the participants failed to be effectively controlled under the standard treatment regimen (or the medication that is available and tolerable as recommended by clinical practice) and were defined as having at least 3 records of different fasting triglyceride levels ≥ 5.65 mmol/L (500 mg/dL) within 2 years..
The screening period should include at least two different days with a TG level of ≥ 5.65 mmol/L (500 mg/dL), with an interval of at least 7 days.
Able to sign informed consent and comply with the requirements and restrictions specified in the informed consent form and the protocol.
Currently participating in other interventional clinical studies, or having an insufficient washout period of less than 5 half-lives or 30 days (whichever is longer) since the last administration of other investigational drugs.
Used antisense oligonucleotide (ASO)-based or small interfering RNA (siRNA)-based lipid-lowering drugs targeting APOC3 within 3 months prior to study drug administration.
Requires long-term use of systemic corticosteroids and steroid drugs and cannot discontinue the medication.
Patients who experienced acute pancreatitis within 4 weeks prior dosing.
CorrectSequence Therapeutics Co., Ltd
Lead sponsor
The First Affiliated Hospital of Anhui Medical University
Collaborator