NCT05164471终止1 期
A Dose Confirmation Study of FLT180a (Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene) in Adult Subjects With Hemophilia B
Spur Therapeutics7 个研究点 分布在 2 个国家目标入组 6 人开始时间: 2021年12月6日最近更新:
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 6
- 试验地点
- 7
- 主要终点
- Number of Participants With Adverse Events
研究概览
简要总结
Study of FLT180a gene therapy in adults with Hemophilia B. Up to 9 patients will be enrolled to receive a single dose of FLT180a and be followed for 52 weeks. Results will confirm the dose for a future Phase 3 study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Hemophilia B with known severe or moderately severe FIX deficiency (≤2% normal circulating FIX activity) for which the subject is on continuous, stable and adequate FIX prophylaxis
- •Have acceptable laboratory values of a) Hemoglobin ≥11g/dL; b) Platelets ≥100,000 cells/µL; c) AST, ALT and alkaline phosphatase (ALP) ≤ upper limit of normal (ULN); d) Serum albumin > lower limit of normal (LLN); e) Total bilirubin ≤1.5 x ULN (except if caused by Gilbert's disease); f) Serum creatinine ≤2.0mg/dL.
- •Level of neutralizing anti-AAV-S3 antibodies below the limit of the pre-established clinical cutoff using an in vitro transduction inhibition assay within the 4 weeks prior to FLT180a administration
- •Has demonstrated ability to accurately, independently and in a timely manner enter bleed diary data during the lead-in study, as judged by the investigator
- •At least 150 exposure days to FIX concentrates
- •At least 6 months of satisfactory controlled prospective baseline data for bleeding events and FIX consumption data from the FLT-01 lead-in study (ECLIPSE)
排除标准
- •Any history of alcohol or drug dependence
- •Presence of neutralizing anti human FIX antibodies (inhibitor; determined by the Nijmegen modified Bethesda inhibitor assay) at the time of enrolment or a previous history of FIX inhibitor
- •Subjects at high risk of thromboembolic events
- •Evidence of advanced liver fibrosis
- •Prior treatment with a gene transfer medicinal product
- •Subjects with active hepatitis B or C
- •Serological evidence of HIV-1, not controlled with anti-viral therapy and as evidenced by cluster of differentiation 4 (CD4)+ counts ≤200 μL
- •Cytomegalovirus (CMV) immunoglobulin G positive subjects who are CMV polymerase chain reaction (PCR) positive at screening
- •Known coagulation disorder other than hemophilia B
- •High sensitivity (hs) troponin-T ≥14 pg/mL during screening
- •History of uncontrolled cardiac failure, unstable angina, or myocardial infarction or other acute cardiac conditions requiring clinical management in the past 6 months
- •Planned surgical procedure within the next 12 months requiring prophylactic FIX treatment
- •Known active severe infection (including documented coronavirus (COVID)-19 infection), or any other significant concurrent, uncontrolled medical condition
研究组 & 干预措施
FLT180a
Experimental
A single dose of FLT180a will be administered. Dose will be determined by enrollment cohort. The first 3 patients will receive 7.7 x 10e11 vg/kg. The dose in subsequent cohorts will be determined by the DMC based on review of data from the prior cohort(s).
干预措施: verbrinacogene setparvovec (Genetic)
结局指标
主要结局
Number of Participants With Adverse Events
时间窗: Dosing through last study visit (average of 48 weeks)
次要结局
- Number of Participants With FIX Activity Levels >=5% and >= 10%(Last study visit (average of 48 weeks))
- Number of Participants With FIX Inhibitor at Any Time Point(Dosing through last study visit (average of 48 weeks))
- Number Patients With Bleeding Episode(Dosing through last study visit (average of 48 weeks))
- Mean Time to Cleared Viral Shedding(Dosing through last study visit (average of 48 weeks))
- Number of Participants With Abnormal Serum Alpha-fetoprotein Levels(Dosing through last study visit (either Week 26 or W52))
研究者
研究点 (7)
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