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

NCT07491172 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: CRSP-CVD-400
• Study Type: interventional
• Target: 90
• Locations: 4 countries
• Sites: 19

Brief 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.

Conditions

Hypercholesterolaemia; Cardiovascular; Metabolic Disease; Dyslipidemias; Lipid Disorder; Hypertriglyceridemia; Heterozygous Familial Hypercholesterolemia (HeFH); Homozygous Familial Hypercholesterolemia (HoFH); Severe Hypertriglyceridemia (sHTG); Mixed Hyperlipemia

Keywords

Refractory Dyslipidemias

Outcome Measures

Primary Outcomes (1)

• To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments

  Incidence of dose-limiting toxicities and frequency of adverse events

  From CTX310 infusion up to 12 months

Secondary Outcomes (4)

• To assess the preliminary efficacy of CTX310 in adult participants with dyslipidemias that are refractory to available treatments

  Over 12 months, compared to baseline

• To further characterize the safety of CTX310 in adult participants with dyslipidemias that are refractory to available treatments

  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

  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

  Over 12 months, compared to baseline

Study Arms (1)

CTX310

EXPERIMENTAL

Subjects will receive an intravenous (IV) infusion.

Drug: CTX310

Interventions

CTX310 DRUG

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).

CTX310

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• CRISPR Therapeutics AG: lead

Study Sites (19)

Integrity Clinical Research

Hialeah, Florida, 33015, United States

RECRUITING

Jacksonville Center for Clinical Research

Jacksonville, Florida, 32216, United States

RECRUITING

Flourish Orlando

Orlando, Florida, 32789, United States

RECRUITING

United Medical Research

Port Orange, Florida, 32127, United States

RECRUITING

University of Rochester

Rochester, New York, 14642, United States

NOT YET RECRUITING

Duke Early Phase Clinical Research

Durham, North Carolina, 27710, United States

RECRUITING

Peters Medical Research

High Point, North Carolina, 27260, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

NOT YET RECRUITING

Oregon Health & Science University (OHSU)

Portland, Oregon, 97239, United States

NOT YET RECRUITING

Royal Adelaide

Adelaide, 5000, Australia

RECRUITING

CORE Research

Brisbane, 4064, Australia

NOT YET RECRUITING

Royal Prince Alfred Hospital

Camperdown, 2050, Australia

NOT YET RECRUITING

Austin Health

Heidelberg, 3084, Australia

RECRUITING

Monash Medical Center

Melbourne, 3168, Australia

RECRUITING

Aotearoa

Auckland, 2025, New Zealand

RECRUITING

NZCR

Christchurch, 8011, New Zealand

RECRUITING

Royal Papworth Hospital

Cambridge, United Kingdom

NOT YET RECRUITING

Barts

London, E1 1BB, United Kingdom

RECRUITING

Richmond Pharmacology

London, SE1 1YR, United Kingdom

RECRUITING

MeSH Terms

Conditions

Metabolic Diseases; Dyslipidemias; Lipid Metabolism Disorders; Hypertriglyceridemia; Homozygous Familial Hypercholesterolemia; Hyperlipoproteinemia Type V; Hypercholesterolemia

Condition Hierarchy (Ancestors)

Nutritional and Metabolic Diseases; Hyperlipidemias; Hyperlipoproteinemia Type II; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias

Central Study Contacts

Clinical Trials

CONTACT

877-214-4634; medicalaffairs@crisprtx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 18, 2026
• First Posted: March 24, 2026
• Study Start: June 21, 2024
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2028
• Last Updated: July 23, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (9); AU (5); NZ (2); GB (3)