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
CRISPR Therapeutics AG
90 participants
Jun 21, 2024
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria6
- 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 Criteria9
- Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.
- 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
- Abnormal or compromised function of kidney, heart, blood or liver.
- Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1.
- Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran).
- Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial.
- 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).
- Women of childbearing potential.
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Interventions
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).
Locations(18)
View Full Details on ClinicalTrials.gov
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NCT07491172