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CS-121 APOC3 Base Editing in Severe Hypertriglyceridemia

ConditionSevere Hypertriglyceridemia (sHTG)
Trial statusNot yet recruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-65
SponsorCorrectSequence Therapeutics Co., Ltd

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Sequential

Treatments tested in this trial

  • CS-121

    Biological/Vaccine

    CS-121 is a in vivo base editing therapy formulated in lipid nanoparticles for targeted editing of the APOC3 gene in hepatocytes.

Treatment groups

15 Participants
are divided into 5 treatment groups
Group A: Single Low Dose CS-121Experimental treatment 1 intervention
Group B: Single Middle Dose CS-121Experimental treatment 1 intervention
Group C: Single High Dose CS-121Experimental treatment 1 intervention
Group D: Single selected Lower Dose 1 of CS-121Experimental treatment 1 intervention
Group E: Single selected Lower Dose 2 of CS-121Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Treatment-Emergent Adverse Events (TEAEs)

Time frame
From screening to 12 months post last dosing
2

Dose-Limiting Toxicities (DLTs)

Time frame
Within 14 days after CS-121 infusion

Secondary outcomes

1

Change from Baseline in Fasting Serum Triglycerides (TG)

Time frame
Baseline through approximately 12 months post dosing
2

Change from Baseline in Serum ApoC3 Protein Levels

Time frame
From baseline to 12 months post last dosing
3

Concentrations of the active components of CS-121 (sgRNA and mRNA)

Time frame
From baseline to 1 month post last dosing

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

CorrectSequence Therapeutics Co., Ltd

Lead sponsor

The First Affiliated Hospital of Anhui Medical University

Collaborator

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: