Recruiting

CS-121 Base Editing Therapy for Familial Chylomicronemia Syndrome

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What is being tested

CS-121

Biological
Who is being recruted

Digestive System Diseases+6

+ Hyperlipidemias

+ Metabolic Diseases

From 18 to 55 Years
+16 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Early Phase 1
Interventional
Study Start: October 2025
See protocol details

Summary

Principal SponsorCorrectSequence Therapeutics Co., Ltd
Study ContactYaliang Li
Last updated: February 12, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: October 15, 2025

Actual date on which the first participant was enrolled.

This clinical trial is focused on testing a new treatment for adults with Familial Chylomicronemia Syndrome (FCS), a rare genetic disorder affecting fat metabolism. People with FCS have extremely high levels of triglycerides in their blood, leading to severe health issues like acute pancreatitis and abdominal pain. Current treatments and strict diets often don't work well enough, leaving a significant need for better solutions. This study is exploring CS-121, a new therapy that aims to correct the gene responsible for this condition, potentially improving triglyceride levels and reducing the risk of pancreatitis. In this study, participants will first undergo tests to confirm their eligibility, which includes checking triglyceride levels and genetic testing. Those who qualify will receive a single dose of CS-121 through an infusion into the bloodstream. After receiving the treatment, participants will be closely monitored for immediate safety, and then continue to have follow-up visits for up to 10 months. During these visits, researchers will check the safety of the treatment and its effectiveness in reducing ApoC3 protein and triglyceride levels in the blood. The study seeks to find the best dose of CS-121 and gather initial safety and effectiveness data to support future developments in treating this and similar conditions.

Official TitleA Clinical Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3, in Adults With Familial Chylomicronemia Syndrome
NCT07176923
Principal SponsorCorrectSequence Therapeutics Co., Ltd
Study ContactYaliang Li
Last updated: February 12, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

15 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 55 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Digestive System DiseasesHyperlipidemiasMetabolic DiseasesNutritional and Metabolic DiseasesPancreatic DiseasesPancreatitisHypertriglyceridemiaDyslipidemiasLipid Metabolism Disorders

Criteria

5 inclusion criteria required to participate
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.
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11 exclusion criteria prevent from participating
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).
Known major organ disease, psychiatric disorders, Cushing's syndrome, or malignancy that, in the investigator's judgment, would make the participant unsuitable for the study or unable to tolerate possible cytokine-release-like events.
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.
Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

5 intervention groups are designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
Participants in this arm will receive a single high dose of CS-121.

Group II

Experimental
Participants in this arm will receive a single low dose of CS-121.

Group III

Experimental
Participants in this arm will receive a single lower dose 1 of CS-121.

Group IV

Experimental
Participants in this arm will receive a single lower dose 2 of CS-121.

Group 5

Experimental
Participants in this arm will receive a single middle dose of CS-121.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Recruiting

The First Affiliated Hospital of Anhui Medical University

Hefei, ChinaOpen The First Affiliated Hospital of Anhui Medical University in Google Maps
Recruiting
One Study Center