Gene-Editing shot aims to tame deadly fat levels in kids
NCT ID NCT07371767
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a new gene-editing treatment called CS-121 in 15 children and teens (ages 4-18) with a rare condition causing extremely high triglycerides. The therapy uses lipid nanoparticles to deliver a base editor that targets the APOC3 gene in the liver. The main goals are to check safety and see if it lowers triglyceride levels.
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 to control severe high triglycerides in children, reducing risk of pancreatitis.
- What could go wrong
- This is a very early, small trial (15 people) testing safety first. Gene editing is new and may have unknown risks or fail to lower triglycerides enough.
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
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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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About 15 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Jan 2026
An estimate. Start dates often move.
- Expected to finish
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Jan 2041
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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4 to 18 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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 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 unsuitable for receiving the study drug, due to potential intolerance to adverse events such as cytokine release storm. * 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 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. * 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.
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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.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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
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Shanghai Children's Medical Center
RECRUITINGShanghai, Shanghai Municipality, China
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