NCT03489291已完成2 期
Phase IIb, Open-label, Single-dose, Single-arm, Multi-center Trial to Confirm the Factor IX Activity Level of the Serotype 5 Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene (AAV5-hFIXco-Padua, AMT-061) Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 3
- 试验地点
- 8
- 主要终点
- Factor IX Activity Levels
研究概览
简要总结
This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase.
The IMP AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The IMP is identified as AAV5-hFIXco-Padua (AMT- 061). The pharmaceutical form of AMT-061 is a solution for intravenous infusion.
The administered dose of AMT-061 will be 2 x 10^13 gc/kg.
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
- •Age ≥18 years
- •Subjects with congenital hemophilia B classified as severe or moderately severe
- •>20 previous exposure days of treatment with FIX protein
排除标准
- •History of FIX inhibitors
- •Positive FIX inhibitor test at screening
- •Select screening laboratory values > 2 times upper normal limit:
- •Positive human immunodeficiency virus (HIV) at screening, not controlled with anti-viral therapy
- •Active infection with Hepatitis B or C virus at screening
- •History of Hepatitis B or C exposure, currently controlled by antiviral therapy
结局指标
主要结局
Factor IX Activity Levels
时间窗: 6 weeks post-dose
To confirm that a single dose of 2x10\^13 gc/kg AMT-061 (CSL222) resulted in factor IX activity levels of ≥5% at 6 weeks after dosing measured by the one-stage (activated partial thromboplastin \[aPTT\]-based) assay.
次要结局
- Annualized Bleeding Rate (ABR)(5 years post-dose)
- Number of Participants Remaining Free of Continuous Prophylaxis(1 year post-dose)
- Annualized Exogenous Factor IX Usage Post-Continuous Prophylaxis(Up to 5 years post-dose)
- Annualized Exogenous Factor IX Usage(52 weeks post-dose)
- Factor IX Activity Levels(52 weeks post-dose)
- Number of Participants With Treatment Emergent (TE): Adverse Events (AE), Mild, Moderate, and Severe AEs, AEs Related and Unrelated to the Study Treatment, and Serious AEs(Up to 5 years post-dose)
- Number of Participants With Clinically Meaningful Findings in Hematology and Serum Chemistry Parameters(Up to 5 years post-dose)
- Number of Participants Receiving Corticosteroids for AST and ALT Elevations(Up to 5 years post-dose)
- Number of Participants With AAV5 Capsid-specific T Cell Response(Up to Week 52)
- Time to First Negative Results for Vector Deoxyribonucleic Acid (DNA) From Semen and Blood(Up to 5 years post-dose)
- Number of Participants Positive With AAV5 and Factor IX Neutralising Antibodies in Serum(Baseline and at 5 years post-dose)
- Number of Participants With Newly Occurring or Worsening Potentially Clinically Significant Changes in Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) Levels(From baseline and up to 5 years post-dose)
- Number of Participants With Inflammatory Marker Levels Outside Normal Ranges(Baseline, Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 14, 16, 18, 20, 22, 24, 26, 31, 36, 40, 44, 48 and 52)
- Number of Participants With Abnormal Values in Alpha-fetoprotein (AFP) Levels(Up to 5 years post-dose)
- Number of Participants With Abnormal Results in Abdominal Ultrasound(At Months 36, 42, 48, 54, and 60 post-dose)
研究者
研究点 (8)
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