CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia (NCT07371767) | Clinical Trial Compass
RecruitingEarly Phase 1
CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia
China15 participantsStarted 2026-01-26
Plain-language summary
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
Who can participate
Age range4 Years – 18 Years
SexALL
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Inclusion 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 age are exempt from signing the written informed consent form).
* Female participants of childbearing potential must have a negative result on serum pregnancy testing.
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.
* 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 unsu…
What they're measuring
1
Dose-limiting toxicities (DLTs)
Timeframe: Within 14 days post CS-121 dosing
2
The incidence and severity of treatment-emergent adverse events (TEAEs)
Timeframe: from screening to 10 months post last dosing
Trial details
NCT IDNCT07371767
SponsorShanghai Jiao Tong University School of Medicine