[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"severe-hypertriglyceridemia-shtg\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:severe-hypertriglyceridemia-shtg":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,45],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100641104","early-phase-1-cs-121-apoc3-base-editing-in-severe-hypertriglyceridemia-100641104",false,"NCT07657780","CS-121 APOC3 Base Editing in Severe Hypertriglyceridemia","A Clinical Study to the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3 for the Treatment of Severe Hypertriglyceridemia in Adults","Inclusion Criteria:\n\n* Male or female participants aged 18 years ≤ age \\\u003C 65 years.\n* The serum triglyceride levels of the participants failed to be effectively controlled under the standard treatment regimen (or the medication that is available and tolerable as recommended by clinical practice) and were defined as having at least 3 records of different fasting triglyceride levels ≥ 5.65 mmol\u002FL (500 mg\u002FdL) within 2 years..\n* The screening period should include at least two different days with a TG level of ≥ 5.65 mmol\u002FL (500 mg\u002FdL), with an interval of at least 7 days.\n* Able to sign informed consent and comply with the requirements and restrictions specified in the informed consent form and the protocol.\n* Female participants must meet one of the following: be not of childbearing potential (e.g., documented hysterectomy, bilateral salpingectomy\u002Fsterilization, or ≥1 year postmenopausal); or, if of childbearing potential, have a negative pregnancy test at screening and be willing to use strict and effective contraception (e.g., abstinence, pharmacologic, or barrier methods) during the study. Male participants with reproductive potential must agree to use strict and effective contraception (e.g., abstinence, pharmacologic, or barrier methods) throughout the entire post-dose observation period; males without reproductive potential must provide supporting medical history (e.g., post-vasectomy).\n\nExclusion Criteria:\n\n* 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.\n* Used antisense oligonucleotide (ASO)-based or small interfering RNA (siRNA)-based lipid-lowering drugs targeting APOC3 within 3 months prior to study drug administration.\n* Requires long-term use of systemic corticosteroids and steroid drugs and cannot discontinue the medication.\n* Patients who experienced acute pancreatitis within 4 weeks prior dosing.\n* History of acute coronary syndrome (ACS) within 6 months before dosing, such as myocardial infarction or unstable angina, or prior coronary revascularization (such as coronary artery bypass grafting), angioplasty or stent implantation.\n* In the investigator's judgment to be unsuitable for the study drug due to receipt of major surgery within 3 months before dosing.\n* Any of the following laboratory abnormalities at screening:.\n* ALT or AST ≥2 × ULN;\n* Total bilirubin ≥2 × ULN;\n* eGFR \\\u003C30 mL\u002Fmin\u002F1.73 m²\n* HbA1c ≥9%;\n* Absolute neutrophil count \\\u003C 1.0 × 109\u002FL\n* Hemoglobin (female)\\\u003C 100 g\u002FL, Hemoglobin (male) \\\u003C 110 g\u002FL\n* Platelet count \\\u003C 100 × 109\u002FL\n* Coagulation function abnormalities judged by the investigator as unsuitable for CS-121 administration.\n* Positive results for HBsAg, dual positivity for HCV antibody and RNA, positive for HIV, or positive for Treponema pallidum infection.\n* 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.\n* Concomitant medications\u002Ftreatments judged by the investigator to affect lipid metabolism, liver and kidney function, coagulation function, or interfere with the efficacy evaluation of the study drug.\n* Patients of childbearing potential who are planning pregnancy, breastfeeding, or have fertility plans.","ALL","18 Years","65 Years",{"count":20,"type":21},15,"ESTIMATED","INTERVENTIONAL",[24],"EARLY_PHASE1","This is an open-label, single-arm, dose-escalation IIT 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 (18-65 years) with Severe Hypertriglyceridemia(sHTG).",[27],"Severe Hypertriglyceridemia (sHTG)",[29,30,31,32],"Apolipoprotein C3 (APOC3)","In Vivo Base Editing","Lipid Nanoparticles (LNP)","Gene Editing Therapy","NOT_YET_RECRUITING","2026-06-15",{"date":36,"type":37},"2026-06-18","ACTUAL",{"date":34,"type":21},{"date":40,"type":21},"2028-07-31",{"name":42,"class":43},"CorrectSequence Therapeutics Co., Ltd","INDUSTRY",1,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":52,"enrollmentInfo":53,"targetDuration":4,"studyType":22,"phases":55,"briefSummary":57,"conditions":58,"keywords":68,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":71,"lastUpdatePostDateStruct":72,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":80},"100630707","phase-1-a-safety-and-tolerability-trial-evaluating-ctx310-in-participants-with-refractory-dyslipidemias-100630707","NCT07491172","A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias","A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Participants With Refractory Dyslipidemias","Key Inclusion Criteria:\n\n1. Age of ≥18 and ≤75 years at the time of signing the informed consent.\n2. Able to provide written informed consent.\n3. Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg\u002FdL - and LDL-C ≥70 mg\u002FdL in participants with ASCVD, or LDL-C ≥70 or 100mg\u002FdL in participants with or without ASCVD respectively, or TG ≥500 mg\u002FdL.\n4. Refractory to the maximal intensity or MTD of standard of care lines of lipid-lowering therapies available through routine clinical care, for at least 12 weeks prior to screening\n5. Female participants must be postmenopausal or surgically sterile.\n6. All male participants and their female partners must agree to the use of an acceptable method of effective contraception for the duration of the study.\n\nExclusion Criteria:\n\n1. Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.\n2. Evidence of liver disease, defined as but not limited to:\n\n   LFTS \\>2 × upper limit of normal (ULN), or total bilirubin \\>2 × ULN, or INR \\>1.5 × ULN, or liver stiffness measured by liver elastography\n3. Abnormal or compromised function of kidney, heart, blood or liver.\n4. Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1.\n5. Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran).\n6. Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial.\n7. Any prior malignancy within the past 5 years, or current malignancy (exceptions for resected or removed basal cell carcinoma, squamous cell carcinoma in situ and carcinoma in situ of the cervix or breast).\n8. Women of childbearing potential.\n\nNote: Other protocol defined Inclusion\u002FExclusion criteria may apply.\n\nNote: The inclusion and exclusion criteria listed represent the global protocol. Additional or modified eligibility criteria may apply in certain countries in accordance with local regulatory and ethics committee requirements and the approved country-specific protocol.","75 Years",{"count":54,"type":21},90,[56],"PHASE1","This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.",[59,60,61,62,63,64,65,27,66,67],"Cardiovascular","Metabolic Disease","Dyslipidemias","Lipid Disorder","Hypertriglyceridemia","Heterozygous Familial Hypercholesterolemia (HeFH)","Homozygous Familial Hypercholesterolemia (HoFH)","Mixed Hyperlipemia","Hypercholesterolaemia",[69],"Refractory Dyslipidemias","RECRUITING","2026-05-19",{"date":73,"type":37},"2026-05-22",{"date":75,"type":37},"2024-06-21",{"date":77,"type":21},"2028-06",{"name":79,"class":43},"CRISPR Therapeutics AG",18]