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New treatment option for Familial Chylomicronemia Syndrome (FCS)
Official title CS-121 APOC3 Base Editing in FCS
ClinicalTrials.gov ID: NCT07176923
What this study is testing
What is CS-121?
CS-121 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for familial chylomicronemia syndrome (fcs).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is an open-label, single-arm, dose-escalation Phase I 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 (18-55 years) with familial chylomicronemia syndrome (FCS).
- Phase 1: an early, usually small safety study
- Time commitment: about 10 months
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 55
You may be able to join if
- 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...
- 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...
- Female participants must meet one of the following: be not of childbearing potential (e.g., documented hysterectomy, bilateral...
You likely can't join if
- Currently participating in another treatment clinical study, or last use of another investigational product with a washout period of less than 5...
- 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.
- History of acute coronary syndrome (ACS) within 6 months before dosing, such as myocardial infarction or unstable angina, or prior coronary...
- In the investigator's judgment, receipt of major surgery within 3 months before dosing that would make the participant unsuitable for this study drug...
- Any of the following laboratory abnormalities at screening: ALT or AST ≥3 × ULN Total bilirubin ≥1.5 × ULN eGFR \ 30 mg/g LDL-C \>130 mg/dL (3.4...
See the full eligibility criteria
- 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...
- 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.
- 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...
- Currently participating in another treatment 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).
- 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.
- History of acute coronary syndrome (ACS) within 6 months before dosing, such as myocardial infarction or unstable angina, or prior coronary revascularization (e.g., coronary artery bypass grafting, angioplasty, or stent...
- In the investigator's judgment, receipt of major surgery within 3 months before dosing that would make the participant unsuitable for this study drug or unable to tolerate possible cytokine-release events.
- Any of the following laboratory abnormalities at screening: ALT or AST ≥3 × ULN Total bilirubin ≥1.5 × ULN eGFR \ 30 mg/g LDL-C \>130 mg/dL (3.4 mmol/L) HbA1c ≥9%
- Coagulation abnormalities deemed by the investigator to make CS-121 administration unsuitable.
- Positive at screening for HBsAg, HCV antibody and RNA, HIV, or Treponema pallidum (syphilis).
- 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...
- Concomitant medications or treatments judged by the investigator to affect lipid metabolism, hepatic or renal function, or coagulation, or to interfere with the evaluation of study drug how well it works, including but...
- Planning pregnancy, currently pregnant, or breastfeeding.
The study team makes the final eligibility decision.
Where it's taking place
- Hefei, Anhui, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 10 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 55 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Hefei, Anhui, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.