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Clinical Trials in the UK / NCT07491172
Recruiting Phase 1

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

NCT07491172 · tracked via the Priya Life Science UK tracker
Sponsor
CRISPR Therapeutics AG
Phase
Phase 1
Started
2024-06-21
Last updated
2026-09-15

Condition(s) studied

CardiovascularMetabolic DiseaseDyslipidemiasLipid DisorderHypertriglyceridemiaHeterozygous Familial Hypercholesterolemia (HeFH)Homozygous Familial Hypercholesterolemia (HoFH)Severe Hypertriglyceridemia (sHTG)Mixed HyperlipemiaHypercholesterolaemia

Investigational drug(s) / intervention(s)

CTX310

CTX310: 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).

Study 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

Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (17)

FacilityCityRegionStatus
Integrity Clinical Research Hialeah Florida Recruiting
Jacksonville Center for Clinical Research Jacksonville Florida Recruiting
Flourish Orlando Orlando Florida Recruiting
United Medical Research Port Orange Florida Recruiting
University of Rochester Rochester New York Recruiting
Duke Early Phase Clinical Research Durham North Carolina Recruiting
Peters Medical Research High Point North Carolina Recruiting
Oregon Health & Science University (OHSU) Portland Oregon Recruiting
Royal Adelaide Adelaide Australia Recruiting
CORE Research Brisbane Australia Recruiting
Royal Prince Alfred Hospital Camperdown Australia Not Yet Recruiting
Austin Health Heidelberg Australia Recruiting
Monash Medical Center Melbourne Australia Recruiting
Aotearoa Auckland New Zealand Recruiting
NZCR Christchurch New Zealand Recruiting
Royal Papworth Hospital Cambridge United Kingdom Recruiting
Richmond Pharmacology London United Kingdom Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07491172 on ClinicalTrials.gov ↗ ← All trials in the UK