A Multi-Center, Phase 1/2, Open-Label, Single and Multiple Ascending Dose Study of CRMA-1001 to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy in Adults With Chronic Hepatitis B
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 66
- 试验地点
- 2
- 主要终点
- Safety and tolerability of single and multiple doses of CRMA-1001
研究概览
简要总结
This is an open-label study with single- and multiple-ascending dose arms followed by a dose expansion arm. The primary objective of the study is to determine the safety and tolerability of CRMA-1001 in adult participants with Chronic Hepatitis B. In addition, the pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of CRMA-1001 will be evaluated. CRMA-1001 is an epigenetic gene therapy delivered via intravenous (IV) infusion. Up to four dose levels will be tested. Participants will receive a single or multiple doses of CRMA-1001 and will remain on antiviral therapy during the dosing process.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 64 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male/Female, weight 45-150 kg, age 18-64, inclusive
- •Diagnosed with Chronic Hepatitis B
- •On oral antiviral therapy
- •ALT and AST <= 1.5 x ULN
- •Total bilirubin <= ULN
排除标准
- •Significant hepatic fibrosis or cirrhosis
- •Current or prior liver disease other than HBV
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
CRMA-1001 Part A, MAD
Multiple ascending dose arm
干预措施: CRMA-1001 (Genetic)
CRMA-1001 Part B
Dose expansion
干预措施: CRMA-1001 (Genetic)
CRMA-1001 Part A, SAD
Single ascending dose arm
干预措施: CRMA-1001 (Genetic)
结局指标
主要结局
Safety and tolerability of single and multiple doses of CRMA-1001
时间窗: 6 months
Incidence and severity of treatment-emergent adverse events
次要结局
- Long-term safety of single and multiple doses of CRMA-1001(60 Months)
- Pharmacokinetics of CRMA-1001 components (Cmax)(6 Months)
- Pharmacokinetics of CRMA-1001 components (Tmax)(6 Months)
- Pharmacokinetics of CRMA-1001 components (terminal clearance rate)(6 Months)
- Pharmacokinetics of CRMA-1001 components (Vd)(6 Months)
- To evaluate the immunogenicity of CRMA-1001(6 Months)
- To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBsAg)(60 Months)
- To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (anti-HBs)(60 Months)
- To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBV DNA)(60 Months)
- To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBeAg)(60 Months)
- To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (anti-HBe)(60 Months)
- To evaluate the effect of CRMA-1001 on the incidence of functional cure(60 Months)
- To evaluate the rate of antiviral therapy discontinuation after treatment with CRMA-1001(60 Months)
- To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBsAg)(6 Months)
- To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (anti-HBs)(6 Months)
- To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBV DNA)(6 Months)
- To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBeAg)(6 Months)
- To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (anti-HBe)(6 Months)