A Safety and Tolerability Study Evaluating CTX320 in Subjects With Elevated Lipoprotein(a) and a History of Atherosclerotic Cardiovascular Disease or Calcific Aortic Valve Stenosis.

A Phase 1 Open-label, Multicenter, First-in-human, Ascending Single Dose Study Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR–Guide RNA–Cas9 Nuclease (CTX320) for In Vivo Editing of the Apolipoprotein(a) Gene (LPA) in Subjects with Elevated Lipoprotein(a) and a History of Atherosclerotic Cardiovascular Disease or Calcific Aortic Valve Stenosis

Status

Not yet recruiting

Phases

Phase 1

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12623001095651

Enrollment

24

Registered

2023-10-18

Start date

2024-01-01

Completion date

Unknown

Last updated

2023-10-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

This study aims to evaluate the safety and tolerability of a single ascending dose of CTX320 in patients with elevated lipoprotein(a) and a history of atherosclerotic cardiovascular disease or calcific aortic valve stenosis and to determine the recommended Phase 2 dose.

Interventions

This is a single-arm, open-label, multicenter, ascending single-dose Phase 1 study. Subjects will receive a single dose of CTX320 via intravenous (IV) infusion. The duration of the infusion is expected to take 1 hour. Planned ascending doses levels will range from 0.1 mg/kg - 0.8 mg/kg. Participants will receive only one dose.

Sponsors

CRISPR Therapeutics AG

Lead SponsorCommercial sector/Industry

Study design

Allocation

Non-randomised trial

Primary purpose

Treatment

Eligibility

Sex/Gender

All

Age

18 Years to 65 Years

Healthy volunteers

No

Inclusion criteria

1. Subjects diagnosed with a history of stable atherosclerotic cardiovascular disease or presence of mild to moderate calcific aortic valve disease 2. Subjects on available standard of care lines of treatment 3. Elevated serum Lp(a) 4. All subjects and their partners should agree to use an effective method of contraception through at least 12 months after CTX320 infusion

Exclusion criteria

1. Evidence of liver disease 2. History of alcohol or drug abuse 3. History of a significant coagulation disorder 4. Severe aortic stenosis 5. Uncontrolled or untreated thyroid disease 6. Prior treatment with gene therapy/editing product 7. Active HIV, hepatitis B virus or hepatitis C virus infection 8. Any prior or current malignancy or myeloproliferative disorder or a significant immunodeficiency disorder 9. Women who are pregnant or breastfeeding

Outcome results

None listed

Source: ANZCTR · Data processed: Feb 4, 2026