A Phase I/II, Open Label, Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Genethon
- 入组人数
- 17
- 试验地点
- 8
- 主要终点
- Proportion of patients having received the selected dose of GNT0003 with serum bilirubin ≤ 300µmol/L within 48 meeks after GNT0003 administration and without phototherapy from week 16
研究概览
简要总结
This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy. Patients will received a single administration of GNT0003 and will be followed for safety and efficacy of approximately 60 months (5 years):
- a follow-up of approximately 12 months (48 weeks)
- a long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene therapy medicinal products, released on 22 Oct.2009 by the Committee for medicinal products for human use.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 9 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with severe Crigler-Najjar syndrome resulting from a molecular confirmation of mutations in the UGT1A1 gene and requiring phototherapy
- •Male or female at least 9 years at the date of signature of informed consent
- •Patient able to give informed assent and/or consent in writing
排除标准
- •Patients who underwent liver transplantation
- •Patients with chronic hepatitis B or C
- •Patients infected with Human immunodeficiency virus (HIV)
- •Patients with significant underlying liver disease
- •Patients with significant encephalopathy
- •Participation in any other investigational trial during this trial
- •Patients unable or unwilling to comply with the protocol requirements
结局指标
主要结局
Proportion of patients having received the selected dose of GNT0003 with serum bilirubin ≤ 300µmol/L within 48 meeks after GNT0003 administration and without phototherapy from week 16
时间窗: 48 weeks
Decrease of total Serum bilirubin level after interruption of daily phototherapy (Efficacy); change in serum total biliirubin from baseline to week 48
Incidence of Treatment Emergent Adverse Events or Treatement Serious Adverse Events
时间窗: 48 weeks
Incidence of AE/SAE evaluated by changes in laboratory parameters, vital signs, physical examination, reported from baseline to each visit study. Clinically relevant abnormal findings on Laboratory values, Vital Signs, Physical findings will be reported as Adverse Events. Incidence and Severity of Adverse Events for each body system will be presented for each dose level and summarized overall.
次要结局
- Change in Health-related quality of Life for Adults from Baseline to Week 48 after GNT0003 administration(48 weeks)
- Change in Health-related quality of Life for Children from Baseline to Week 48 after GNT0003 administration(48 weeks)