A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: CTX310.
- Who it may be relevant to
- Registry conditions: Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder. Basic parameters: 18 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, New Zealand, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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.
Detailed description
This is a phase 1, open-label, multi-center study of CTX310 in participants with refractory dyslipidemias. Subjects will receive a dose of CTX310 via intravenous (IV) infusion.
Interventions
- Drug 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).
Primary outcome measures
- To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments [Time frame: From CTX310 infusion up to 12 months]
Secondary outcome measures (4)
- To assess the preliminary efficacy of CTX310 in adult participants with dyslipidemias that are refractory to available treatments [Time frame: Over 12 months, compared to baseline]
- To further characterize the safety of CTX310 in adult participants with dyslipidemias that are refractory to available treatments [Time frame: From CTX310 infusion up to 12 months]
- To assess the pharmacokinetics (PK) of CTX310 in adult participants with dyslipidemias that are refractory to available treatments [Time frame: From CTX310 infusion up to 12 months]
- To assess the pharmacodynamic (PD) response of CTX310 in adult participants with dyslipidemias that are refractory to available treatments [Time frame: Over 12 months, compared to baseline]
Eligibility criteria
Inclusion criteria
- 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 criteria
- 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.
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 9 centers
- Integrity Clinical Research — Hialeah
- Jacksonville Center for Clinical Research — Jacksonville
- Flourish Orlando — Orlando
- United Medical Research — Port Orange
- University of Rochester — Rochester
- Duke Early Phase Clinical Research — Durham
- Peters Medical Research — High Point
- Cleveland Clinic — Cleveland
- … and 1 more center
Australia · 5 centers
- Royal Adelaide — Adelaide
- CORE Research — Brisbane
- Royal Prince Alfred Hospital — Camperdown
- Austin Health — Heidelberg
- Monash Medical Center — Melbourne
United Kingdom · 3 centers
- Royal Papworth Hospital — Cambridge
- Barts — London
- Richmond Pharmacology — London
New Zealand · 2 centers
- Aotearoa — Auckland
- NZCR — Christchurch
Identifiers
NCT: NCT07491172 · CRSP-CVD-400