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CTX310 for refractory high triglycerides or LDL cholesterol

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

Phase 1 Interventional CRISPR Therapeutics · NCT07491172

This trial will test whether a single IV dose of CTX310 is safe and tolerated in adults whose high triglycerides or LDL cholesterol have not responded to available treatments.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment90 (estimated)
Ages18 Years to 75 Years
SexAll
SponsorCRISPR Therapeutics Industry-sponsored
Locations18 sites (Jacksonville, Florida and 17 other locations)
Trial IDNCT07491172 on ClinicalTrials.gov

What this trial studies

This is a Phase 1, open-label, single-arm, multicenter ascending-dose trial enrolling adults 18–75 with dyslipidemias that are refractory to maximal tolerated lipid-lowering therapy. Participants will receive one intravenous infusion of CTX310 and be followed for safety and tolerability outcomes. The study will enroll at multiple clinical research sites in Florida and uses dose-escalation cohorts to identify tolerable dosing. Efficacy is not the primary endpoint, but lipid levels will be monitored as exploratory outcomes.

Who should consider this trial

Good fit: Adults aged 18–75 with persistent hypertriglyceridemia or elevated LDL-C despite maximal tolerated standard-of-care lipid-lowering therapy, who meet reproductive safety requirements, are the intended candidates.

Not a fit: Patients with familial chylomicronemia syndrome, significant liver disease (eg, LFTs >2×ULN), or other trial exclusion criteria are unlikely to be eligible or to benefit from participation.

Why it matters

Potential benefit: If successful, CTX310 could provide a new treatment option to lower triglycerides or LDL in patients who have not responded to existing lipid-lowering therapies.

How similar studies have performed: Other novel lipid-lowering biologic and genetic therapies have shown promising lipid reductions in early studies, but CTX310 itself is being tested here in a first-in-patient Phase 1 format.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria:

1. Age of ≥18 and ≤75 years at the time of signing the informed consent.
2. Able to provide written informed consent.
3. 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.
4. 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
5. Female participants must be postmenopausal or surgically sterile.
6. 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 Criteria:

1. Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.
2. 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
3. Abnormal or compromised function of kidney, heart, blood or liver.
4. Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1.
5. Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran).
6. Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial.
7. 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).
8. 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.

Where this trial is running

Jacksonville, Florida and 17 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions CardiovascularMetabolic DiseaseDyslipidemiasLipid DisorderHypertriglyceridemiaHeterozygous Familial HypercholesterolemiaHomozygous Familial HypercholesterolemiaSevere Hypertriglyceridemia
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.