[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100630707":3},{"organization":4,"outcomesModule":7,"designInfo":28,"detailedDescription":34,"studyPopulation":27,"armGroups":35,"interventions":42,"overallOfficials":27,"centralContacts":47,"locations":53,"responsibleParty":305,"collaborators":27,"id":307,"slug":308,"hasResults":309,"nctId":310,"briefTitle":311,"officialTitle":312,"acronym":27,"eligibilityCriteria":313,"healthyVolunteers":309,"sex":314,"minAge":315,"maxAge":316,"enrollmentInfo":317,"targetDuration":27,"studyType":320,"phases":321,"briefSummary":323,"conditions":324,"keywords":335,"overallStatus":56,"whyStopped":27,"lastUpdateSubmitDate":337,"lastUpdatePostDateStruct":338,"startDateStruct":341,"completionDateStruct":343,"leadSponsor":345,"locationsCount":347},{"fullName":5,"class":6},"CRISPR Therapeutics","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":27},[9],{"measure":10,"description":11,"timeFrame":12},"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",[14,18,21,24],{"measure":15,"description":16,"timeFrame":17},"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":19,"description":20,"timeFrame":12},"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":22,"description":23,"timeFrame":12},"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":25,"description":26,"timeFrame":17},"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",null,{"allocation":29,"interventionModel":30,"interventionModelDescription":27,"primaryPurpose":31,"observationalModel":27,"timePerspective":27,"maskingInfo":32},"NA","SINGLE_GROUP","TREATMENT",{"masking":33,"maskingDescription":27,"whoMasked":27},"NONE","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.",[36],{"label":37,"type":38,"description":39,"interventionNames":40},"CTX310","EXPERIMENTAL","Subjects will receive an intravenous (IV) infusion.",[41],"Drug: CTX310",[43],{"type":44,"name":37,"description":45,"armGroupLabels":46,"otherNames":27},"DRUG","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).",[37],[48],{"name":49,"role":50,"phone":51,"phoneExt":27,"email":52},"Clinical Trials","CONTACT","877-214-4634","medicalaffairs@crisprtx.com",[54,73,86,100,114,129,144,158,173,189,203,218,232,246,262,276,291],{"facility":55,"status":56,"city":57,"state":58,"zip":59,"country":60,"countryCode":61,"cosmosGeoPoint":62,"geoPoint":67,"contacts":68},"Integrity Clinical Research","RECRUITING","Hialeah","Florida","33015","United 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Jordan","336-883-9773","erikaj@petersmedicalresearch.com",{"facility":159,"status":56,"city":160,"state":161,"zip":162,"country":60,"countryCode":61,"cosmosGeoPoint":163,"geoPoint":167,"contacts":168},"Oregon Health & Science University (OHSU)","Portland","Oregon","97239",{"type":63,"coordinates":164},[165,166],-122.67621,45.52345,{"lat":166,"lon":165},[169],{"name":170,"role":50,"phone":171,"phoneExt":27,"email":172},"Hailey Volk","503-494-2004","volkh@ohsu.edu",{"facility":174,"status":56,"city":175,"state":27,"zip":176,"country":177,"countryCode":178,"cosmosGeoPoint":179,"geoPoint":183,"contacts":184},"Royal Adelaide","Adelaide","5000","Australia","AU",{"type":63,"coordinates":180},[181,182],138.59863,-34.92866,{"lat":182,"lon":181},[185],{"name":186,"role":50,"phone":187,"phoneExt":27,"email":188},"Denise Healy","+61 409 877 807","Denise.Healy@sa.gov.au",{"facility":190,"status":56,"city":191,"state":27,"zip":192,"country":177,"countryCode":178,"cosmosGeoPoint":193,"geoPoint":197,"contacts":198},"CORE Research","Brisbane","4064",{"type":63,"coordinates":194},[195,196],153.02809,-27.46794,{"lat":196,"lon":195},[199],{"name":200,"role":50,"phone":201,"phoneExt":27,"email":202},"Daunte Tamaki","07 + 38765688","daunte.tamaki@coreresearchgroup.com",{"facility":204,"status":205,"city":206,"state":27,"zip":207,"country":177,"countryCode":178,"cosmosGeoPoint":208,"geoPoint":212,"contacts":213},"Royal Prince Alfred Hospital","NOT_YET_RECRUITING","Camperdown","2050",{"type":63,"coordinates":209},[210,211],151.17642,-33.88965,{"lat":211,"lon":210},[214],{"name":215,"role":50,"phone":216,"phoneExt":27,"email":217},"Aimei Lee","(02) + 9515 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75111243","debra.oleary@monashhealth.org",{"facility":247,"status":56,"city":248,"state":27,"zip":249,"country":250,"countryCode":251,"cosmosGeoPoint":252,"geoPoint":256,"contacts":257},"Aotearoa","Auckland","2025","New Zealand","NZ",{"type":63,"coordinates":253},[254,255],174.76349,-36.84853,{"lat":255,"lon":254},[258],{"name":259,"role":50,"phone":260,"phoneExt":27,"email":261},"Kate Msiska","+64 (0) 21 850 235","kate.msiska@aotearoatrials.nz",{"facility":263,"status":56,"city":264,"state":27,"zip":265,"country":250,"countryCode":251,"cosmosGeoPoint":266,"geoPoint":270,"contacts":271},"NZCR","Christchurch","8011",{"type":63,"coordinates":267},[268,269],172.63333,-43.53333,{"lat":269,"lon":268},[272],{"name":273,"role":50,"phone":274,"phoneExt":27,"email":275},"Jo Sanders","+64 (0) 3 372 9477","jo.sanders@nzcr.co.nz",{"facility":277,"status":56,"city":278,"state":27,"zip":27,"country":279,"countryCode":280,"cosmosGeoPoint":281,"geoPoint":285,"contacts":286},"Royal Papworth Hospital","Cambridge","United Kingdom","UK",{"type":63,"coordinates":282},[283,284],0.11667,52.2,{"lat":284,"lon":283},[287],{"name":288,"role":50,"phone":289,"phoneExt":27,"email":290},"Jenny Castedo","+441223 638000","jenny.castedo1@nhs.net",{"facility":292,"status":56,"city":293,"state":27,"zip":294,"country":279,"countryCode":280,"cosmosGeoPoint":295,"geoPoint":299,"contacts":300},"Richmond Pharmacology","London","SE1 1YR",{"type":63,"coordinates":296},[297,298],-0.12574,51.50853,{"lat":298,"lon":297},[301],{"name":302,"role":50,"phone":303,"phoneExt":27,"email":304},"Volunteer Recruitment Team","+44 (0)20 7042 5800","volunteer@richmondpharmacology.com",{"type":306,"investigatorFullName":27,"investigatorTitle":27,"investigatorAffiliation":27,"oldNameTitle":27,"oldOrganization":27},"SPONSOR","100630707","phase-1-a-safety-and-tolerability-trial-evaluating-ctx310-in-participants-with-refractory-dyslipidemias-100630707",false,"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.","ALL","18 Years","75 Years",{"count":318,"type":319},90,"ESTIMATED","INTERVENTIONAL",[322],"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.",[325,326,327,328,329,330,331,332,333,334],"Cardiovascular","Metabolic Disease","Dyslipidemias","Lipid Disorder","Hypertriglyceridemia","Heterozygous Familial Hypercholesterolemia (HeFH)","Homozygous Familial Hypercholesterolemia (HoFH)","Severe Hypertriglyceridemia (sHTG)","Mixed Hyperlipemia","Hypercholesterolaemia",[336],"Refractory Dyslipidemias","2026-09-17",{"date":339,"type":340},"2026-09-18","ACTUAL",{"date":342,"type":340},"2024-06-21",{"date":344,"type":319},"2028-06",{"name":346,"class":6},"CRISPR Therapeutics AG",17]