2023-505827-29-00
进行中（未招募）
1/2 期
(19429) A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A
Bayer AG, Bayer AG6 个研究点 分布在 4 个国家目标入组 6 人开始时间: 2023年12月18日最近更新:
概览
- 阶段
- 1/2 期
- 状态
- 进行中（未招募）
- 发起方
- Bayer AG, Bayer AG
- 入组人数
- 6
- 试验地点
- 6
- 主要终点
- Incidence of adverse events, treatment-emergent adverse events, serious adverse events and Adverse events/serious adverse events of special interest
概览
简要总结
Investigate the safety and tolerability of single ascending intravenous (IV) doses of BAY 2599023 (DTX201) in adult patients with severe hemophilia A, who have been previously treated with FVIII product
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Overall
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Males ≥ 18 years of age
- •Subjects with severe hemophilia A (baseline FVIII activity FVIII:C <1%) determined by measurement at the time of Screening (unless there is genetic testing compatible with severe deficiency)
- •Previously treated with FVIII concentrate(s) (plasma derived or recombinant) or cryoprecipates for a minimum of 150 exposure days (ED)
- •Are on one of the following therapies: Prophylaxis, and is willing to stop prophylactic treatment at specified time points throughout the study or On-demand: have had > 4 bleeding events in the last 52 weeks
- •Subjects must agree to use double barrier and effective contraception methods. Vasectomized subjects must agree to use condoms. This is applicable from the time of the study drug administration until notified by the investigator. Recommendation to investigators is to continue the contraception until three consecutive blood and semen samples BLOD of shed virus have been obtained. Acceptable methods of contraception include, but are not limited to, (i) condoms with a spermicidal agent (ii) diaphragm or cervical cap with spermicide; if an intra-uterine device or hormone-based contraception is used by the patient's partner, an additional barrier method must be used.
- •Male subjects must agree not to donate cells, semen, blood, tissue or organs from the time of study drug administration
排除标准
- •Current evidence of inhibitor to FVIII with a titer ≥ 0.6 BU/mL
- •History of a malignancy for which the subject has received treatment in the past 2 years except for prostate cancer being monitored without medical intervention, or surgically removed non-melanoma skin cancer
- •Known or suspected autoimmune diseases
- •Known prior history of hypersensitivity or anaphylaxis associated with any FVIII or immunoglobulin administration.
- •Known or suspected hypersensitivity or allergic reaction to trial product(s) or related FVIII products or any component of BAY2599023 (DTX201)
- •Live vaccines within the last 30 days prior to the study drug administration; live vaccines may be re-introduced after viral shedding has been cleared
- •Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study
- •Any individual who requires any pre-medication to tolerate FVIII treatment (e.g., antihistamines)
- •Prior use of emicizumab within 3 months before dosing
- •History of inhibitor to FVIII with a titer ≥ 0.6 BU, or clinical history suggestive of inhibitor requiring modification of treatment
结局指标
主要结局
Incidence of adverse events, treatment-emergent adverse events, serious adverse events and Adverse events/serious adverse events of special interest
Incidence of adverse events, treatment-emergent adverse events, serious adverse events and Adverse events/serious adverse events of special interest
次要结局
- Expression pattern of FVIII activity.
- Proportion of patients in the respective dose step, that reached an expression of FVIII above 5% at 6 months and 12 months following the IV administration of BAY2599023
研究者
Therapeutic Area Head
Scientific
Bayer AG
研究点 (6)
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