Severe Hypertriglyceridemia (sHTG)Early Phase 1NCT07657780
CS-121 APOC3 Base Editing in Severe Hypertriglyceridemia
A Early Phase 1 study testing CS-121 in people with Severe Hypertriglyceridemia (sHTG), run by CorrectSequence Therapeutics Co., Ltd at 1 site in China.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Severe Hypertriglyceridemia (sHTG)
- What is being tested?
- CS-121
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteChina
- Study dates
- 15 Jun 2026to 31 Jul 2028
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- Female participants must meet one of the following: be not of childbearing potential (e.g., documented hysterectomy, bilateral salpingectomy/sterilization, or ≥1 year postmenopausal); or, if of childbearing potential, have a negative pregnancy test at screening and be willing to use strict and effective contraception (e.g., abstinence, pharmacologic, or barrier methods) during the study. Male participants with reproductive potential must agree to use strict and effective contraception (e.g., abstinence, pharmacologic, or barrier methods) throughout the entire post-dose observation period; males without reproductive potential must provide supporting medical history (e.g., post-vasectomy).
Who cannot join
- 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.
- History of acute coronary syndrome (ACS) within 6 months before dosing, such as myocardial infarction or unstable angina, or prior coronary revascularization (such as coronary artery bypass grafting), angioplasty or stent implantation.
- In the investigator's judgment to be unsuitable for the study drug due to receipt of major surgery within 3 months before dosing.
Show all 19Show fewer
- Any of the following laboratory abnormalities at screening:.
- ALT or AST ≥2 × ULN;
- Total bilirubin ≥2 × ULN;
- eGFR <30 mL/min/1.73 m²
- HbA1c ≥9%;
- Absolute neutrophil count < 1.0 × 109/L
- Hemoglobin (female)< 100 g/L, Hemoglobin (male) < 110 g/L
- Platelet count < 100 × 109/L
- Coagulation function abnormalities judged by the investigator as unsuitable for CS-121 administration.
- Positive results for HBsAg, dual positivity for HCV antibody and RNA, positive for HIV, or positive for Treponema pallidum infection.
- Known major organ diseases, mental disorders, Cushing's syndrome, hypothyroidism, history of lymphoproliferative disorders, or malignant tumors in any organ system, which are judged by the investigator as unsuitable for study participation due to potential intolerance to adverse events such as cytokine-Release-Storm.
- Concomitant medications/treatments judged by the investigator to affect lipid metabolism, liver and kidney function, coagulation function, or interfere with the efficacy evaluation of the study drug.
- Patients of childbearing potential who are planning pregnancy, breastfeeding, or have fertility plans.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui
Huan Zhou, +8613665527160, zhouhuanbest@vip.163.com
About this study
Official title: A Clinical Study to the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3 for the Treatment of Severe Hypertriglyceridemia in Adults
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).
Read the detailed description
CS-121 is an investigational, in vivo base editing therapy delivered by lipid nanoparticles (LNPs) targeting the APOC3 gene in the liver. By introducing precise base edits at specific APOC3 loci, CS-121 is intended to mimic naturally occurring protective mutations that reduce ApoC3 expression, thereby restoring triglyceride clearance pathways and lowering pancreatitis risk. Preclinical studies in transgenic mouse and non-human primate models demonstrated dose-dependent APOC3 editing, reductions in serum ApoC3 protein and triglyceride levels, and acceptable safety profiles, supporting advancement into human evaluation.
This open-label, single-arm, dose-escalation early exploratory trial designed to evaluate the safety, tolerability, PK/PD characteristics and preliminary efficacy of CS-121 in patients with sHTG. Based on the properties of gene editing therapy, the primary focus of the study is to identify the optimal biological dose (OBD) rather than the traditional maximum tolerated dose (MTD).
- Conditions
- Severe Hypertriglyceridemia (sHTG)
- Being tested
- CS-121
- Keywords
- Apolipoprotein C3 (APOC3), In Vivo Base Editing, Lipid Nanoparticles (LNP), Gene Editing Therapy
- Medical subject headings
- Hypertriglyceridemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CS-121Biological
CS-121 is a in vivo base editing therapy formulated in lipid nanoparticles for targeted editing of the APOC3 gene in hepatocytes.
| Study group | Role | What they receive |
|---|---|---|
| Single Low Dose CS-121 | Experimental | Participants in this arm will receive a single low dose (0.5mg/kg) of CS-121. Receives: Biological: CS-121 |
| Single Middle Dose CS-121 | Experimental | Participants in this arm will receive a single middle dose (0.7mg/kg) of CS-121. Receives: Biological: CS-121 |
| Single High Dose CS-121 | Experimental | Participants in this arm will receive a single high dose (1.0mg/kg) of CS-121 Receives: Biological: CS-121 |
| Single selected Lower Dose 1 of CS-121 | Experimental | Participants in this arm will receive a single selected lower dose 1 of CS-121. Receives: Biological: CS-121 |
| Single selected Lower Dose 2 of CS-121 | Experimental | Participants in this arm will receive a single selected lower dose 2 of CS-121 Receives: Biological: CS-121 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-Emergent Adverse Events (TEAEs)
Measured: From screening to 12 months post last dosing
Dose-Limiting Toxicities (DLTs)
Measured: Within 14 days after CS-121 infusion
Secondary outcomes (3)
Change from Baseline in Fasting Serum Triglycerides (TG)
Measured: Baseline through approximately 12 months post dosing
Change from Baseline in Serum ApoC3 Protein Levels
Measured: From baseline to 12 months post last dosing
Concentrations of the active components of CS-121 (sgRNA and mRNA)
Measured: From baseline to 1 month post last dosing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2026
- Study start15 Jun 2026 (estimated)
- Primary completion15 Mar 2028 (estimated)
- Study completion31 Jul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Hypertriglyceridemia (sHTG)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CorrectSequence Therapeutics Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The First Affiliated Hospital of Anhui Medical University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07657780
- Sponsor's protocol ID
- CS-121-06
- Status last verified
- Jun 2026
- First submitted
- 15 Jun 2026
- Last updated
- 18 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study to the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3 for the Treatment of Severe Hypertriglyceridemia in Adults
- ClinicalTrials.gov ID
- NCT07657780
- Acronym / sponsor ID
- CS-121-06
- Conditions
- Severe Hypertriglyceridemia (sHTG)
- Keywords
- Apolipoprotein C3 (APOC3), In Vivo Base Editing, Lipid Nanoparticles (LNP), Gene Editing Therapy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- CorrectSequence Therapeutics Co., LtdIndustry (pharma, biotech, devices)
- Collaborators
- The First Affiliated Hospital of Anhui Medical University
- Study start
- 15 Jun 2026 (estimated)
- Primary completion
- 15 Mar 2028 (estimated)
- Study completion
- 31 Jul 2028 (estimated)
- First posted
- 18 Jun 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
CS-121 is an investigational, in vivo base editing therapy delivered by lipid nanoparticles (LNPs) targeting the APOC3 gene in the liver. By introducing precise base edits at specific APOC3 loci, CS-121 is intended to mimic naturally occurring protective mutations that reduce ApoC3 expression, thereby restoring triglyceride clearance pathways and lowering pancreatitis risk. Preclinical studies in transgenic mouse and non-human primate models demonstrated dose-dependent APOC3 editing, reductions in serum ApoC3 protein and triglyceride levels, and acceptable safety profiles, supporting advancement into human evaluation.
This open-label, single-arm, dose-escalation early exploratory trial designed to evaluate the safety, tolerability, PK/PD characteristics and preliminary efficacy of CS-121 in patients with sHTG. Based on the properties of gene editing therapy, the primary focus of the study is to identify the optimal biological dose (OBD) rather than the traditional maximum tolerated dose (MTD).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Single Low Dose CS-121 | Experimental | Biological: CS-121Participants in this arm will receive a single low dose (0.5mg/kg) of CS-121. |
| Single Middle Dose CS-121 | Experimental | Biological: CS-121Participants in this arm will receive a single middle dose (0.7mg/kg) of CS-121. |
| Single High Dose CS-121 | Experimental | Biological: CS-121Participants in this arm will receive a single high dose (1.0mg/kg) of CS-121 |
| Single selected Lower Dose 1 of CS-121 | Experimental | Biological: CS-121Participants in this arm will receive a single selected lower dose 1 of CS-121. |
| Single selected Lower Dose 2 of CS-121 | Experimental | Biological: CS-121Participants in this arm will receive a single selected lower dose 2 of CS-121 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
- Female participants must meet one of the following: be not of childbearing potential (e.g., documented hysterectomy, bilateral salpingectomy/sterilization, or ≥1 year postmenopausal); or, if of childbearing potential, have a negative pregnancy test at screening and be willing to use strict and effective contraception (e.g., abstinence, pharmacologic, or barrier methods) during the study. Male participants with reproductive potential must agree to use strict and effective contraception (e.g., abstinence, pharmacologic, or barrier methods) throughout the entire post-dose observation period; males without reproductive potential must provide supporting medical history (e.g., post-vasectomy).
Key exclusion criteria (19 listed)
- 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.
- History of acute coronary syndrome (ACS) within 6 months before dosing, such as myocardial infarction or unstable angina, or prior coronary revascularization (such as coronary artery bypass grafting), angioplasty or stent implantation.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CS-121CS-121 is a in vivo base editing therapy formulated in lipid nanoparticles for targeted editing of the APOC3 gene in hepatocytes. | Biological | Single High Dose CS-121, Single Low Dose CS-121, Single Middle Dose CS-121, Single selected Lower Dose 1 of CS-121, Single selected Lower Dose 2 of CS-121 |
Primary outcome
Treatment-Emergent Adverse Events (TEAEs)
Time frame: From screening to 12 months post last dosing
Dose-Limiting Toxicities (DLTs)
Time frame: Within 14 days after CS-121 infusion
3 secondary outcomes
Change from Baseline in Fasting Serum Triglycerides (TG)
Time frame: Baseline through approximately 12 months post dosing
Change from Baseline in Serum ApoC3 Protein Levels
Time frame: From baseline to 12 months post last dosing
Concentrations of the active components of CS-121 (sgRNA and mRNA)
Time frame: From baseline to 1 month post last dosing
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Severe Hypertriglyceridemia (sHTG)
Not yet recruitingPhase 2
A Phase II Study of FXR0906 in Patients With Severe Hypertriglyceridemia or Hypertriglyceridemia With ASCVD.
sHTG, HTG With ASCVD (Diagnosed or at Increased Risk)
RecruitingPhase 1
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
Cardiovascular, Metabolic Disease
Not yet recruitingPhase 3
A Phase III Study to Evaluate the Efficacy and Safety of of DR10624 in Subjects With Severe Hypertriglyceridemia
Severe Hypertriglyceridemia (sHTG), Hypertriglyceridemia
Active, not recruitingPhase 3
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
Severe Hypertriglyceridemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing CS-121 in people with Severe Hypertriglyceridemia (sHTG), run by CorrectSequence Therapeutics Co., Ltd at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 18 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xiaokai Li (+8618686610731, xiaokai.li@correctsequence.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment-Emergent Adverse Events (TEAEs) (time frame: From screening to 12 months post last dosing). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jun 2026.