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 AG0 个研究点目标入组 24 人开始时间: 2023年10月18日最近更新: 2023年10月23日

适应症

试验速览

暂无简介。

•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

•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

发起方