NCT07491172招募中1 期
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
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 90
- 试验地点
- 23
- 主要终点
- To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age of ≥18 and ≤75 years at the time of signing the informed consent.
- •Able to provide written informed consent.
- •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.
- •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
- •Female participants must be postmenopausal or surgically sterile.
- •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.
排除标准
- •Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.
- •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
- •Abnormal or compromised function of kidney, heart, blood or liver.
- •Acute coronary syndrome event or stroke within 24 weeks prior to Day
- •Acute pancreatitis within 12 weeks prior to Day
- •Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran).
- •Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial.
- •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).
- •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.
研究组 & 干预措施
CTX310
Experimental
Subjects will receive an intravenous (IV) infusion.
干预措施: CTX310 (Drug)
结局指标
主要结局
To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments
时间窗: From CTX310 infusion up to 12 months
Incidence of dose-limiting toxicities and frequency of adverse events
次要结局
- To assess the preliminary efficacy of CTX310 in adult participants with dyslipidemias that are refractory to available treatments(Over 12 months, compared to baseline)
- To further characterize the safety of CTX310 in adult participants with dyslipidemias that are refractory to available treatments(From CTX310 infusion up to 12 months)
- To assess the pharmacokinetics (PK) of CTX310 in adult participants with dyslipidemias that are refractory to available treatments(From CTX310 infusion up to 12 months)
- To assess the pharmacodynamic (PD) response of CTX310 in adult participants with dyslipidemias that are refractory to available treatments(Over 12 months, compared to baseline)
研究者
研究点 (23)
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