NCT07491172

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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
90

participants targeted

Target at P75+ for phase_1

Timeline
24mo left

Started Jun 2024

Longer than P75 for phase_1

Geographic Reach
4 countries

18 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress50%
Jun 2024Jun 2028

Study Start

First participant enrolled

June 21, 2024

Completed
1.7 years until next milestone

First Submitted

Initial submission to the registry

March 18, 2026

Completed
6 days until next milestone

First Posted

Study publicly available on registry

March 24, 2026

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2027

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2028

Last Updated

May 22, 2026

Status Verified

May 1, 2026

Enrollment Period

2.9 years

First QC Date

March 18, 2026

Last Update Submit

May 19, 2026

Conditions

HypercholesterolaemiaCardiovascularMetabolic DiseaseDyslipidemiasLipid DisorderHypertriglyceridemiaHeterozygous 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

CTX310DRUG

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

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (18)

Research Site 10

Jacksonville, Florida, 32216, United States

NOT YET RECRUITING

Research Site 17

Orlando, Florida, 32789, United States

RECRUITING

Research Site 11

Port Orange, Florida, 32127, United States

RECRUITING

Research Site 14

Durham, North Carolina, 27710, United States

NOT YET RECRUITING

Research Site 13

High Point, North Carolina, 27260, United States

RECRUITING

Research Site 9

Cleveland, Ohio, 44195, United States

NOT YET RECRUITING

Research Site 12

Portland, Oregon, 97239, United States

NOT YET RECRUITING

Research Site 1

Adelaide, 5000, Australia

RECRUITING

Research Site 15

Brisbane, 4064, Australia

NOT YET RECRUITING

Research Site 16

Brisbane, Australia

NOT YET RECRUITING

Research Site 7

Camperdown, 2050, Australia

NOT YET RECRUITING

Research Site 3

Heidelberg, 3084, Australia

RECRUITING

Research Site 2

Melbourne, 3168, Australia

RECRUITING

Research Site 5

Auckland, 2025, New Zealand

RECRUITING

Research Site 4

Christchurch, 8011, New Zealand

RECRUITING

Research Site 18

Cambridge, United Kingdom

NOT YET RECRUITING

Research Site 8

London, E1 1BB, United Kingdom

NOT YET RECRUITING

Research Site 6

London, SE1 1YR, United Kingdom

RECRUITING

MeSH Terms

Conditions

Metabolic DiseasesDyslipidemiasLipid Metabolism DisordersHypertriglyceridemiaHomozygous Familial HypercholesterolemiaHyperlipoproteinemia Type VHypercholesterolemia

Condition Hierarchy (Ancestors)

Nutritional and Metabolic DiseasesHyperlipidemiasHyperlipoproteinemia Type IILipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemias

Central Study Contacts

Clinical Trials

CONTACT

877-214-4634medicalaffairs@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

May 22, 2026

Record last verified: 2026-05

Data Sharing

IPD Sharing
Will not share

Locations

US(7)AU(6)NZ(2)GB(3)