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
CRISPR Therapeutics AG
24 participants
Jan 1, 2024
Interventional
Conditions
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.
Eligibility
Inclusion Criteria4
- Subjects diagnosed with a history of stable atherosclerotic cardiovascular disease or presence of mild to moderate calcific aortic valve disease
- Subjects on available standard of care lines of treatment
- Elevated serum Lp(a)
- All subjects and their partners should agree to use an effective method of contraception through at least 12 months after CTX320 infusion
Exclusion Criteria9
- Evidence of liver disease
- History of alcohol or drug abuse
- History of a significant coagulation disorder
- Severe aortic stenosis
- Uncontrolled or untreated thyroid disease
- Prior treatment with gene therapy/editing product
- Active HIV, hepatitis B virus or hepatitis C virus infection
- Any prior or current malignancy or myeloproliferative disorder or a significant immunodeficiency disorder
- Women who are pregnant or breastfeeding
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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.
Locations(1)
View Full Details on ANZCTR
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ACTRN12623001095651