[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07491172":3,"trial-entities:NCT07491172":223,"trial-summary:NCT07491172":231},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":29,"study_type":31,"primary_purpose":32,"phases":33,"enrollment_info":35,"interventions":38,"primary_outcomes":44,"secondary_outcomes":49,"sex":63,"minimum_age":64,"maximum_age":65,"healthy_volunteers":66,"eligibility_criteria":67,"std_ages":71,"locations":74,"central_contacts":214,"overall_officials":220,"references":221,"see_also_links":222},"NCT07491172","CRSP-CVD-400","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","RECRUITING","2028-06","2026-07","2026-07-23","2024-06-21","CRISPR Therapeutics AG","INDUSTRY",true,"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.","This is a phase 1, open-label, multi-center study of CTX310 in participants with refractory dyslipidemias. Subjects will receive a dose of CTX310 via intravenous (IV) infusion.",[19,20,21,22,23,24,25,26,27,28],"Cardiovascular","Metabolic Disease","Dyslipidemias","Lipid Disorder","Hypertriglyceridemia","Heterozygous Familial Hypercholesterolemia (HeFH)","Homozygous Familial Hypercholesterolemia (HoFH)","Severe Hypertriglyceridemia (sHTG)","Mixed Hyperlipemia","Hypercholesterolaemia",[30],"Refractory Dyslipidemias","INTERVENTIONAL","TREATMENT",[34],"PHASE1",{"count":36,"type":37},90,"ESTIMATED",[39],{"type":40,"name":41,"description":42,"armGroupLabels":43},"DRUG","CTX310","CTX310 is a lipid nanoparticle (LNP) formulation of clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) components for in vivo editing of the target gene angiopoietin-like 3 (ANGPTL3).",[41],[45],{"measure":46,"description":47,"timeFrame":48},"To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments","Incidence of dose-limiting toxicities and frequency of adverse events","From CTX310 infusion up to 12 months",[50,54,57,60],{"measure":51,"description":52,"timeFrame":53},"To assess the preliminary efficacy of CTX310 in adult participants with dyslipidemias that are refractory to available treatments","Percentage change from baseline in lipid values (LDL-C, non-HDL-C, Trigs, apoB and HDL-C)","Over 12 months, compared to baseline",{"measure":55,"description":56,"timeFrame":48},"To further characterize the safety of CTX310 in adult participants with dyslipidemias that are refractory to available treatments","Frequency and severity of adverse events (AE), including treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESIs), clinically significant laboratory abnormalities, and clinically significant abnormal vital signs.",{"measure":58,"description":59,"timeFrame":48},"To assess the pharmacokinetics (PK) of CTX310 in adult participants with dyslipidemias that are refractory to available treatments","Levels of CTX310 in blood over time",{"measure":61,"description":62,"timeFrame":53},"To assess the pharmacodynamic (PD) response of CTX310 in adult participants with dyslipidemias that are refractory to available treatments","Percentage change from baseline of ANGPTL3","ALL","18 Years","75 Years",false,{"inclusion":68,"exclusion":69,"raw_text":70},[],[],"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.",[72,73],"ADULT","OLDER_ADULT",[75,84,91,98,105,114,122,129,137,145,153,160,167,174,181,189,196,203,210],{"facility":76,"status":8,"city":77,"state":78,"zip":79,"country":80,"geoPoint":81},"Integrity Clinical Research","Hialeah","Florida","33015","United States",{"lat":82,"lon":83},25.8576,-80.27811,{"facility":85,"status":8,"city":86,"state":78,"zip":87,"country":80,"geoPoint":88},"Jacksonville Center for Clinical Research","Jacksonville","32216",{"lat":89,"lon":90},30.33218,-81.65565,{"facility":92,"status":8,"city":93,"state":78,"zip":94,"country":80,"geoPoint":95},"Flourish Orlando","Orlando","32789",{"lat":96,"lon":97},28.53834,-81.37924,{"facility":99,"status":8,"city":100,"state":78,"zip":101,"country":80,"geoPoint":102},"United Medical Research","Port Orange","32127",{"lat":103,"lon":104},29.13832,-80.99561,{"facility":106,"status":107,"city":108,"state":109,"zip":110,"country":80,"geoPoint":111},"University of Rochester","NOT_YET_RECRUITING","Rochester","New York","14642",{"lat":112,"lon":113},43.15478,-77.61556,{"facility":115,"status":8,"city":116,"state":117,"zip":118,"country":80,"geoPoint":119},"Duke Early Phase Clinical Research","Durham","North Carolina","27710",{"lat":120,"lon":121},35.99403,-78.89862,{"facility":123,"status":8,"city":124,"state":117,"zip":125,"country":80,"geoPoint":126},"Peters Medical Research","High Point","27260",{"lat":127,"lon":128},35.95569,-80.00532,{"facility":130,"status":107,"city":131,"state":132,"zip":133,"country":80,"geoPoint":134},"Cleveland Clinic","Cleveland","Ohio","44195",{"lat":135,"lon":136},41.4995,-81.69541,{"facility":138,"status":107,"city":139,"state":140,"zip":141,"country":80,"geoPoint":142},"Oregon Health & Science University (OHSU)","Portland","Oregon","97239",{"lat":143,"lon":144},45.52345,-122.67621,{"facility":146,"status":8,"city":147,"zip":148,"country":149,"geoPoint":150},"Royal Adelaide","Adelaide","5000","Australia",{"lat":151,"lon":152},-34.92866,138.59863,{"facility":154,"status":107,"city":155,"zip":156,"country":149,"geoPoint":157},"CORE Research","Brisbane","4064",{"lat":158,"lon":159},-27.46794,153.02809,{"facility":161,"status":107,"city":162,"zip":163,"country":149,"geoPoint":164},"Royal Prince Alfred Hospital","Camperdown","2050",{"lat":165,"lon":166},-33.88965,151.17642,{"facility":168,"status":8,"city":169,"zip":170,"country":149,"geoPoint":171},"Austin Health","Heidelberg","3084",{"lat":172,"lon":173},-37.75,145.06667,{"facility":175,"status":8,"city":176,"zip":177,"country":149,"geoPoint":178},"Monash Medical Center","Melbourne","3168",{"lat":179,"lon":180},-37.814,144.96332,{"facility":182,"status":8,"city":183,"zip":184,"country":185,"geoPoint":186},"Aotearoa","Auckland","2025","New Zealand",{"lat":187,"lon":188},-36.84853,174.76349,{"facility":190,"status":8,"city":191,"zip":192,"country":185,"geoPoint":193},"NZCR","Christchurch","8011",{"lat":194,"lon":195},-43.53333,172.63333,{"facility":197,"status":107,"city":198,"country":199,"geoPoint":200},"Royal Papworth Hospital","Cambridge","United Kingdom",{"lat":201,"lon":202},52.2,0.11667,{"facility":204,"status":8,"city":205,"zip":206,"country":199,"geoPoint":207},"Barts","London","E1 1BB",{"lat":208,"lon":209},51.50853,-0.12574,{"facility":211,"status":8,"city":205,"zip":212,"country":199,"geoPoint":213},"Richmond Pharmacology","SE1 1YR",{"lat":208,"lon":209},[215],{"name":216,"role":217,"phone":218,"email":219},"Clinical Trials","CONTACT","877-214-4634","medicalaffairs@crisprtx.com",[],[],[],{"nct_id":4,"conditions":224,"biomarkers":230},[225,226,227,228,229,23],"Dyslipidemia","Familial hypercholesterolemia - heterozygous","Familial hypercholesterolemia - homozygous","Fredrickson Type V Lipidemia","Hypercholesterolemia",[],{"nct_id":4,"found":15,"summary":232,"prompt_version":242},{"design":233,"status":234,"heading":235,"summary":236,"follow_up":237,"word_count":238,"commitments":239,"compensation":240,"drugs_mentioned":241},"This is a Phase 1, single-arm, open-label study, meaning all 90 participants will receive CTX310, and both you and the study team will know which treatment you are getting.","completed","CTX310 for Refractory Dyslipidemias","This study is testing a new treatment called CTX310 for people with dyslipidemias (high cholesterol and triglycerides) that haven't improved with other treatments. CTX310 uses a special technology called CRISPR to target a gene involved in how your body handles fats. The main goal of this study is to see how safe CTX310 is for you, and if you can tolerate it, over a period of up to 12 months after receiving the treatment. You might be able to join if you are between 18 and 75 years old and have specific levels of high triglycerides or cholesterol. This study is currently enrolling participants.","Your safety will be monitored for up to 12 months after receiving the CTX310 infusion.",104,"You will receive CTX310 through an intravenous (IV) infusion. The study will monitor your safety for up to 12 months after this infusion.","Not stated in the trial record.",[41],"v2"]