ACTRN12623000809639招募中1 期
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 (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Subjects With Refractory Dyslipidemias
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 24
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Non-randomised trial
- 主要目的
- Treatment
入排标准
- 年龄范围
- 18 Years 至 70 Years（—）
- 性别
- All
入选标准
- •1. Subjects diagnosed with persistent dyslipidemia
- •2. Subjects must be refractory to the maximum tolerated doses of standard of care lines of treatment
- •3. Female subjects must be postmenopausal
- •4. Nonsterile male subjects and their female partners should agree to use an effective method of contraception through at least 12 months after CTX310 infusion
- •5. Adequate renal, liver and cardiac function
- •6. Willing to participate in a long-term follow-up study after completion of this study
排除标准
- •1. No participants with Familial Chylomicronemia Syndrome (FCS)
- •2. Evidence of liver disease
- •3. History of alcohol or drug abuse
- •4. History of a significant coagulation disorder
- •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
研究者
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