Testing safety of CTX310, a gene-editing treatment, in hard-to-treat high blood fats
Tests CTX310 gene editing in adults whose blood fats resist current treatments.
Plain-language title generated from the registry record
Registry title: A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
Key facts
- Where
- Durham, Hialeah, High Point, Jacksonville, Orlando, Port Orange, Portland, and Rochester, United StatesSee the 8 sites
- Phase
- Phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 75 years old?
Source: NCT07491172, who can take part
Where in United States
- Duke Early Phase Clinical ResearchDurham, United StatesTaking people
- Integrity Clinical ResearchHialeah, United StatesTaking people
- Peters Medical ResearchHigh Point, United StatesTaking people
- Jacksonville Center for Clinical ResearchJacksonville, United StatesTaking people
- Flourish OrlandoOrlando, United StatesTaking people
- United Medical ResearchPort Orange, United StatesTaking people
- Oregon Health & Science University (OHSU)Portland, United StatesTaking people
- University of RochesterRochester, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- CRISPR Therapeutics AG
- Trial code
- NCT07491172
- Record updated
- 18 September 2026
- Original description
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.Read full original descriptionShow less
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
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.
Inclusion: Age of ≥18 and ≤75 years at the time of signing the informed consent; Able to provide written informed consent; Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg/dL - and LDL-C ≥70 mg/dL in participants with ASCVD, or LDL-C ≥70 or 100mg/dL in participants with or without ASCVD respectively, or TG ≥500 mg/dL; 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; Female participants must be postmenopausal or surgically sterile; 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.
Exclusion: Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1; Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial; Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply; Abnormal or compromised function of kidney, heart, blood or liver; 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); Evidence of liver disease, defined as but not limited to:; LFTS >2 × upper limit of normal (ULN), or total bilirubin >2 × ULN, or INR >1.5 × ULN, or liver stiffness measured by liver elastography; Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran); Women of childbearing potential; Note: Other protocol defined Inclusion/Exclusion criteria may apply; Note: 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.