CTX310 for refractory high triglycerides or LDL cholesterol

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Integrity Clinical Research
  Hialeah, Florida, United States Recruiting
• Jacksonville Center for Clinical Research
  Jacksonville, Florida, United States Recruiting
• Flourish Orlando
  Orlando, Florida, United States Recruiting
• United Medical Research
  Port Orange, Florida, United States Recruiting
• University of Rochester
  Rochester, New York, United States Recruiting
• Duke Early Phase Clinical Research
  Durham, North Carolina, United States Recruiting

Study contacts

• Study coordinator: Clinical Trials
• Email: medicalaffairs@crisprtx.com
• Phone: 877-214-4634

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.