CareCN: A phase I/II, open-label, study to evaluate the safety and efficacy of an intravenous injection of GNT0003 (Adeno-associated Viral Vector expressing the UGT1A1 transgene) in patients with severe Crigler-Najjar syndrome requiring phototherapy
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- Genethon
- 入组人数
- 17
- 试验地点
- 4
- 主要终点
- Dose escalation part: Incidence of treatment-emergent adverse events (TEAEs) or treatment-emergent serious adverse events (TESAEs) up to Week 17
研究概览
简要总结
Dose escalation part: To assess the safety and tolerability of an intravenous single-dose administration of GNT0003 in patients with severe Crigler-Najjar syndrome requiring phototherapy Confirmatory part: To assess the efficacy of the intravenous single-dose administration of GNT0003 selected dose in patients with severe Crigler-Najjar syndrome requiring phototherapy
入排标准
- 年龄范围
- 0 years 至 65+ years（0-17 Years, 18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Patients with severe Crigler-Najjar syndrome requiring daily phototherapy (≥ 6h/day)
- •Molecular confirmation of mutations in the UGT1A1 gene by DNA sequencing
排除标准
- •Fibrosis score ≥ 3 (METAVIR) or 10 kPa
- •Patients who underwent liver transplantation
结局指标
主要结局
Dose escalation part: Incidence of treatment-emergent adverse events (TEAEs) or treatment-emergent serious adverse events (TESAEs) up to Week 17
Dose escalation part: Incidence of treatment-emergent adverse events (TEAEs) or treatment-emergent serious adverse events (TESAEs) up to Week 17
Dose escalation part: Change in laboratory parameters, vital signs and in physical examination from baseline to Week 17
Dose escalation part: Change in laboratory parameters, vital signs and in physical examination from baseline to Week 17
Confirmatory part: The proportion of patients having received the selected dose of GNT0003 with serum total bilirubin ≤ 300 µmol/L at Week 48 after IMP infusion and without phototherapy from Week 16
Confirmatory part: The proportion of patients having received the selected dose of GNT0003 with serum total bilirubin ≤ 300 µmol/L at Week 48 after IMP infusion and without phototherapy from Week 16
次要结局
- Dose escalation part: Time to GNT0003 vector clearance from blood, urine, saliva and feces
- Dose escalation part: Number of patients with serum total bilirubin ≤ 300 µmol/L within 7 days after interruption of daily phototherapy
- Confirmatory part: Incidence of treatment-emergent adverse events (TEAEs) or treatment-emergent serious adverse events (TESAEs) up to Week 48
- Confirmatory part: Change in laboratory parameters, vital signs and in the physical examination from baseline up to Week 48
- Confirmatory part: Change in serum total bilirubin from baseline up to Week 48
- Confirmatory part: Change in bilirubin/albumin ratio from baseline up to Week 48
- Confirmatory part: Change in Health-related quality of life as measured by QOL questionnaires, SF-36 (adult) and PedsQL (pediatric), and by the quality of sleep questionnaire from baseline up to Week 48.
- Confirmatory part: Time to GNT0003 vector clearance from blood, urine, saliva and feces
研究者
Clinical Development Department
Scientific
Genethon