A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients With Partial Lipodystrophy
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- 入组人数
- 12
- 试验地点
- 12
- 主要终点
- Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%.
研究概览
简要总结
This is an Open Label, Phase IV, Post Authorisation Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in people with Partial Lipodystrophy
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of familial or acquired partial lipodystrophy
排除标准
- •Treatment with any Investigational Medicinal Product (IMP) within 6 months or 5 times the terminal half-life of the corresponding IMP, whichever is longer, before the screening visit.
- •Other protocol defined inclusion/exclusion criteria apply
研究组 & 干预措施
Metreleptin
Metreleptin [Recombinant-methionyl human Leptin; r-metHuLeptin] for daily injection is a sterile, white, solid lyophilised cake
干预措施: Metreleptin (Drug)
结局指标
主要结局
Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%.
时间窗: 12 months
To evaluate the efficacy (HbA1c) of metreleptin treatment in patients with PL
Number of patients with decrease of at least 30% in triglycerides (TG) at Month 12 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L)
时间窗: 12 months
To evaluate the efficacy (TG) of metreleptin treatment in patients with PL
次要结局
- Number of patients with decrease of at least 0.5% in HbA1c at Month 24 compared to Baseline or HbA1c <6.5 % at Month 24, in patients with baseline HbA1c ≥6.5%.(24 months)
- Number of patients with decrease of at least 30% in TG levels at Month 24 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L).(24 months)
- Change from baseline in liver volume at Month 12 and Month 24(12 months and 24 months)
- Incidence of, Treatment emergent adverse events (TEAEs), Deaths and other serious adverse events (SAEs), Treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation(24 months)