EUCTR2015-001486-67-BG进行中（未招募）1 期
A Phase I/II Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 15
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •1. Male =18 years of age.
- •2. Moderate/severe or severe hemophilia B (baseline FIX activity =2% of normal or documented history of FIX activity =2%).
- •3. At least 3 bleeding episodes per year that require on-demand treatment with FIX OR are treated with a prophylactic regimen of FIX.
- •4. At least 100 days exposure history to FIX.
- •5. No documented history of inhibitors (neutralizing antibodies) to exogenous FIX.
- •6. No known allergic reaction to exogenous FIX or any component of DTX101.
- •7. Willing to stop prophylactic treatment with recombinant FIX at specified time points during the study.
- •8. Willing and able to provide written informed consent.
- •9. Willing and able to comply with study procedures and requirements.
- •10. Willing to use effective contraception at the time of administration of DTX101 and for 3 months following administration of DTX101. Appropriate contraceptive methods include a condom with spermicide. Abstinence, defined as sexual inactivity, is an acceptable form of birth control; however, appropriate contraception must be used if the subject becomes sexually active.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 13
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •1. History of liver disease as evidenced by any of the following: portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy, or a liver biopsy with evidence of stage 3 fibrosis.
- •2. Significant hepatic inflammation or cirrhosis as evidenced by any of the following: aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2.0 × upper limit of normal (ULN), total bilirubin >1.5 × ULN, alkaline phosphatase (ALP) >2.5 × ULN, platelet count <75,000 cells/µL, and prothrombin time (PT) or international normalized ratio (INR) >1.5 × ULN.
- •3. Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection, documented by current use of antiviral therapy for HBV or HCV or by hepatitis B surface antigen (HBsAg) or HCV RNA positivity. NOTE: Two negative viral assays by polymerase chain reaction (PCR), collected at least 6 months apart, will be required to be considered negative for HCV. Subjects can be rescreened once if they have one negative sample and must wait to have the second sample collected within the following 6 months.
- •4. History of human immunodeficiency virus (HIV) infection AND any of the following: CD4+ cell count <350 cells/mm3, change in antiretroviral therapy regimen within 6 months prior to Day 0, or plasma viral load >200 copies/mL, on 2 separate occasions, as measured by PCR.
- •5. Anti-AAVrh10 neutralizing antibody titer >1:5.
- •6. Participation (current or previous) in another gene therapy study.
- •7. Participation in another investigational medicine study within 3 months before screening.
- •8. History of a malignancy for which the subject has received treatment in the past 2 years except for prostate cancer treated with watchful waiting or surgically removed non-melanoma skin cancer.
- •9. Has any other significant medical condition that the investigator feels would be a risk to the subject or would impede the study.
研究者
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