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

ClinicalTrials.gov ID

NCT07491172

Lead Sponsor

CRISPR Therapeutics AGINDUSTRY

Overall Status

Recruiting

Study Type

Interventional

Phase

Phase 1

Enrollment

90Estimated

Last Update Posted

Sep 9, 2026Actual

Start Date

Jun 21, 2024Actual

Primary Completion Date

Jun 2027Estimated

Completion Date

Jun 2028Estimated

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

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.

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.

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

• CTX310

Ages Eligible for Study

Adult (18-64)Older Adult (65+)

Sexes Eligible for Study

All

Accepts Healthy Volunteers

No

Eligibility Criteria

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 Purpose

Treatment

Allocation

N/A

Interventional Model

Single Group Assignment

Masking

None (Open Label)

Lead Sponsor

CRISPR Therapeutics AGINDUSTRY

smunoz@flourishresearch.com407-740-8078

Denise.Healy@sa.gov.au+61 409 877 807

adele.manzoney@unimelb.edu.au+613 9496 2160

debra.oleary@monashhealth.org+61 3 75111243

kate.msiska@aotearoatrials.nz+64 (0) 21 850 235

jo.sanders@nzcr.co.nz+64 (0) 3 372 9477

jenny.castedo1@nhs.net+441223 638000

volunteer@richmondpharmacology.com+44 (0)20 7042 5800

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