NCT03588299进行中（未招募）1 期
A Phase 1/2 Open-label Safety and Dose-finding Study of BAY2599023 (DTX201), an Adeno-associated Virus (AAV) hu37-mediated Gene Transfer of B-domain Deleted Human Factor VIII, in Adults With Severe Hemophilia A
Bayer26 个研究点 分布在 6 个国家目标入组 11 人开始时间: 2018年11月7日最近更新:
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 11
- 试验地点
- 26
- 主要终点
- Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interest
研究概览
简要总结
In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the disease. By replacing the defective gene with a healthy copy the human body may produce clotting factor on its own. Hemophilia A is a bleeding disorder in which the human body does not have enough clotting factor VIII, a protein that controls bleeding. Researcher want to find the optimal dose of BAY 2599023 (DTX201) so that the body may produce enough clotting factor on its own.
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
- •Males age 18 years or older.
- •Confirmed diagnosis of hemophilia A as evidenced by their medical history with plasma FVIII activity levels < 1% of normal or at screening.
- •Have >150 exposure days (EDs) to FVIII concentrates (recombinant or plasma-derived).
- •If on prophylaxis, are required to be willing to stop prophylactic treatment at specified time points throughout the study or If on-demand: should have had > 4 bleeding events in the last 52 weeks
- •- Agree to use reliable barrier contraception.
排除标准
- •History of allergic reaction to any FVIII product.
- •Clinically relevant findings in the physical examination considered critical by the treating physician, including obesity with BMI > 35 kg/m*2
- •Current evidence of measurable inhibitor against factor VIII, prior history of inhibitors to FVIII protein or clinical history suggestive of inhibitor.
- •Evidence of active hepatitis B or C.
- •Currently on antiviral therapy for hepatitis B or C.
- •Significant underlying liver disease.
- •Serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm*3; HIV+ and stable participants with CD4 count >200/mm*3 and undetectable viral load are eligible to enroll.
- •Detectable antibodies reactive with AAVhu37capsid.
- •Participant with another bleeding disorder that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B).
- •Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational product within the last 12 weeks.
- •Known or suspected hypersensitivity or allergic reaction to trial product(s) or related FVIII products or any component of BAY2599023 (DTX201), or a contraindication to prednisolone
研究组 & 干预措施
BAY2599023 / (DTX201)
Experimental
Adult patients with severe hemophilia A, who have been previously treated with FVIII products
干预措施: BAY2599023 (DTX201) (Drug)
结局指标
主要结局
Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interest
时间窗: Up to 5 years.
次要结局
- Expression pattern of FVIII activity.(Up to 5 years)
- 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
研究点 (26)
Loading locations...
相似试验
已完成
2 期
Safety and Tolerability of 4975 in the Treatment of Moderate to Severe Knee Pain Due to Osteoarthritis (OA)Osteoarthritis of the KneeNCT00667654Centrexion Therapeutics54
终止
1 期
A Study of Tolinapant in Combination With Oral Decitabine/Cedazuridine and Oral Decitabine/Cedazuridine Alone in Participants With Relapsed/Refractory Peripheral T-cell Lymphoma (R/R PTCL)Relapsed/Refractory Peripheral T-cell LymphomaNCT05403450Taiho Oncology, Inc.33
终止
1 期
A Phase 1/2 Study to Evaluate OTX-2002 in Patients With Hepatocellular Carcinoma and Other Solid Tumor Types Known for Association With the MYC OncogeneSolid TumorHepatocellular Carcinoma Non-resectableHepatocellular Carcinoma RecurrentHepatocellular CancerLiver, Cancer Of, Non-ResectableHepatocellular CarcinomaLiver CancerNCT05497453Omega Therapeutics24
招募中
1 期
A Study of BGB-11417 in Participants With Myeloid MalignanciesAcute Myeloid LeukemiaMyelodysplastic SyndromesMyelodysplastic/Myeloproliferative NeoplasmNCT04771130BeOne Medicines260
终止
1 期
A Study Of Avelumab In Combination With Other Cancer Immunotherapies In Advanced Malignancies (JAVELIN Medley)Advanced CancerNCT02554812Pfizer409