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New treatment option for Hyperchylomicronemia
Official title CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia
ClinicalTrials.gov ID: NCT07371767
What this study is testing
What is CS-121?
CS-121 is an investigational medicine, being studied as a potential treatment for hyperchylomicronemia.
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 a Prospective, Single-center, Open-label, Single-arm Clinical Study to Evaluate the Safety and Efficacy of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3, in Children and Adolescents (4-18 years) With Hyperchylomicronemia
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 18
You may be able to join if
- Male or female participants aged 4 years ≤ age \< 18 years.
- Severe hypertriglyceridemia (sHTG), defined as a triglyceride (TG) level ≥ 500 mg/dL.
- Confirmed diagnosis of genetically inherited FCS via genetic testing, or clinically diagnosed FCS plus persistent chylomicronemia.
- Failure to achieve adequate TG control, For participants under 8 years of age, the investigator determine at their discretion whether prior...
- Participants aged 6 years and above must sign the informed consent form themselves; for participants under 18 years of age, their parent/legal...
You likely can't join if
- Currently participating in other treatment clinical studies, or having an insufficient washout period of less than 5 half-lives or 30 days (whichever...
- Used antisense oligonucleotide (ASO)-based or small interfering RNA (siRNA)-based lipid-lowering drugs targeting APOC3 within 3 months prior to study...
- History of acute pancreatitis within 1 month before dosing.
- Patients who underwent major surgery within 3 months prior to study drug administration and are judged by the investigator as unsuitable for...
- ALT or AST ≥2 × ULN
- Total bilirubin ≥1.5 × ULN
See the full eligibility criteria
- Male or female participants aged 4 years ≤ age \< 18 years.
- Severe hypertriglyceridemia (sHTG), defined as a triglyceride (TG) level ≥ 500 mg/dL.
- Confirmed diagnosis of genetically inherited FCS via genetic testing, or clinically diagnosed FCS plus persistent chylomicronemia.
- Failure to achieve adequate TG control, For participants under 8 years of age, the investigator determine at their discretion whether prior lipidlowering therapy has been administered.
- Participants aged 6 years and above must sign the informed consent form themselves; for participants under 18 years of age, their parent/legal guardian must sign the informed consent form. (Participants under 6 years of...
- Female participants of childbearing potential must have a negative result on serum pregnancy testing.
- Currently participating in other treatment 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...
- Used antisense oligonucleotide (ASO)-based or small interfering RNA (siRNA)-based lipid-lowering drugs targeting APOC3 within 3 months prior to study drug administration.
- History of acute pancreatitis within 1 month before dosing.
- Patients who underwent major surgery within 3 months prior to study drug administration and are judged by the investigator as unsuitable for receiving the study drug, due to potential intolerance to adverse events such...
- ALT or AST ≥2 × ULN
- Total bilirubin ≥1.5 × ULN
- eGFR \<30 mL/min/1.73 m²
- Random urine albumin-to-creatinine ratio (UACR) \>30 mg/g, or urine protein is ≥ 2+
- HbA1c ≥9%
- 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...
- Concomitant medications/treatments judged by the investigator to affect lipid metabolism, liver and kidney function, coagulation function, or interfere with the how well it works evaluation of the study drug.
- Patients of childbearing potential who are planning pregnancy, breastfeeding, or have fertility plans.
- History of hypersensitivity to any study drug, its excipients, or drugs of similar chemical classes.
- Other medical conditions or comorbidities that, in the investigator's opinion, may interfere with study compliance or data interpretation.
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, Shanghai Municipality, 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 listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, Shanghai Municipality, 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.