Open-label, Phase 3b Study to Evaluate Effectiveness, Safety and Pharmacokinetic Parameters of Metreleptin in Patients Under 6 Years of Age With Generalised Lipodystrophy and Associated Diabetes Mellitus and/or Hypertriglyceridaemia
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 1
- 试验地点
- 20
- 主要终点
- Percent change from baseline in fasting serum TG levels at Month 12 for subjects with fasting TG levels ≥2.3 mmol/L (200 mg/dL) at baseline
研究概览
简要总结
This is an open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or hypertriglyceridaemia
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
- — 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of Generalised Lipodystrophy
- •Metreleptin treatment naive
排除标准
- •Weight <9 kg at Screening (Visit 1)
研究组 & 干预措施
Metreleptin
Metreleptin [Recombinant-methionyl human Leptin; rmetHuLeptin] for daily injection is a sterile, white, solid lyophilised cake
干预措施: Metreleptin (Drug)
结局指标
主要结局
Percent change from baseline in fasting serum TG levels at Month 12 for subjects with fasting TG levels ≥2.3 mmol/L (200 mg/dL) at baseline
时间窗: 12 months
To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia
Absolute change from baseline in glycated haemoglobin (HbA1c) at Month 12 for subjects with HbA1c ≥6.5% at baseline
时间窗: 12 months
To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia
次要结局
- Proportion of subjects of those with Baseline HbA1c ≥6.5% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <6.5% or HbA1c <5.7% at Month 12(12 months)
- Proportion of subjects of those with Baseline HbA1c ≥5.7% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <5.7% at Month 12(12 months)
- Proportion of subjects of those with fasting serum TG ≥ 1.7 mmol/L (150 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12(12 months)
- Proportion of subjects of those with fasting serum TG ≥ 2.3 mmol/L (200 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12(12 months)
- Change from baseline in liver volume and liver span as assessed by ultrasound at each post-baseline visit through Month 12(12 months)
- Incidence of, treatment emergent adverse events (TEAEs), serious adverse events (SAEs), treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation(12 months)