NCT04676048招募中1 期
Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
ASC Therapeutics2 个研究点 分布在 1 个国家目标入组 12 人开始时间: 2022年8月3日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- Number of adverse events, and serious AEs
研究概览
简要总结
Currently, hemophilia A patients are managed with prophylactic or on-demand replacement therapy with recombinant FVIII or alternative therapeutics. The major challenges of current treatment regimens, such as the short half-life of hemophilia therapeutics with the need for frequent IV injections, encourage the current efforts for gene transfer therapy.
This study will evaluate the safety and preliminary efficacy of ASC618, an AAV vector encoding B-domain deleted codon-optimized human factor VIII under a synthetic liver-directed promoter
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 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male ≥18 years of age
- •Severe or moderately severe hemophilia A (FVIII activity ≤ 2 IU/dL) as evidenced by
- •medical history
- •Received FVIII prophylactic or on-demand replacement therapy for ≥ 150 accumulated
- •days (exposure days)
- •≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months
- •Agree to use double-barrier contraceptive until at least 3 consecutive semen samples are negative after ASC-618 infusion
排除标准
- •Pre-existing immunity to AAV8 vector as defined by AAV8 total antibodies and neutralizing antibodies qualified tests.
- •Current inhibitors, or history of high titer FVIII inhibitors
- •Presence of > Grade 2 liver fibrosis on elastography/Fibroscan or comparable imaging methodology
- •History of chronic renal disease
- •Active infection or any immunosuppressive disorder
- •History of cardiac surgery and need anticoagulant therapy
- •Any cardiovascular / genetic risk factors for thromboembolic disorders
- •Evidence of active Hepatitis B, Hepatitis C, Human Immunodeficiency Virus (HIV)-1/2 or syphilis infection.
- •Receipt of any vector or gene transfer agent
- •Current antiviral therapy for hepatitis B or C
结局指标
主要结局
Number of adverse events, and serious AEs
时间窗: 12 months post-infusion
次要结局
- Changes in FVIII activity levels from baseline(12 months post-infusion)
- Annualized FVIII consumption(12 months post-infusion)
- Annualized bleeding rate (ABR)(12 months post-infusion)
研究者
研究点 (2)
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