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Gene-Editing shot aims to slash dangerous fat levels in blood

NCT ID NCT07657780

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early study tests a new gene-editing treatment called CS-121 in 15 adults with severe high triglycerides (a type of fat in the blood) that hasn't been controlled by standard treatments. The therapy uses tiny fat particles to deliver a gene editor that targets the APOC3 gene in the liver, aiming to lower fat levels. The main goal is to check safety and side effects, while also seeing if it reduces triglycerides and related proteins.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
CS-121 (a gene-editing therapy delivered by lipid nanoparticles to edit the APOC3 gene)
What this could lead to
If it works, this could point toward a one-time treatment that controls severe high triglycerides by editing a gene, potentially reducing the need for daily medication.
What could go wrong
This is a very early, small trial (15 people) focused on safety, not yet on effectiveness. The therapy is new and may not work as hoped, and there are unknown risks from gene editing.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

About 15 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Jun 2026

An estimate. Start dates often move.

Expected to finish

Jul 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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). Exclusion Criteria: * 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. * 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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • The First Affiliated Hospital of Anhui Medical University

    Hefei, Anhui, China

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