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)
To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments — From CTX310 infusion up to 12 months Incidence of dose-limiting toxicities and frequency of adverse events
Trial sites (17)
Facility
City
Region
Status
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.
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