The ENERGY 3 Study: A Randomized, Controlled, Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Children With Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 27
- 试验地点
- 15
- 主要终点
- Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) concentration through Week 52
研究概览
简要总结
The primary purpose of Study INZ701-106 (The ENERGY 3 Study) is to assess the efficacy and safety of INZ-701 in children with ENPP1 Deficiency.
详细描述
INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy (ERT) in development for the treatment of ENPP1 Deficiency, an ultra-rare genetic disorder with an incidence of 1 in 64,000 pregnancies.
Study INZ701-106 (The ENERGY 3 Study) is a multi-center, randomized in a 2:1 ratio, controlled, open-label Phase 3 study to evaluate the efficacy and safety of INZ-701 in children with ENPP1 Deficiency.
The study will consist of a Screening Period of up to 52 days (including a washout period of up to 7 days for prohibited medications post-Randomization) and a Randomized Treatment Period (INZ-701 or control) of 52 weeks, followed by an Open-label Extension Period during which all study participants may receive INZ-701, and an End of Study (EOS) Safety visit 30 days after the last dose of INZ-701.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
INZ-701
Subjects randomized to the INZ-701 arm will be administered a 2.4 mg/kg once weekly dose by subcutaneous (SC) injection for the duration of the 52-week Randomized Treatment Period and the Open-label Extension Period.
干预措施: INZ-701 (Drug)
Control Arm (Conventional Therapy)
Subjects randomized to the control arm will continue taking their conventional therapy as clinically indicated by their treating physician for the duration of the 52-week Randomized Treatment Period.
干预措施: Control Arm (Conventional Therapy) (Drug)
结局指标
主要结局
Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) concentration through Week 52
时间窗: 52 weeks (Baseline through Week 52)
For each subject, plasma PPi will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
次要结局
- Maximum Plasma Concentration (Cmax) of INZ-701(52 weeks (Randomized Treatment Period))
- Change from Baseline in growth Z-score (height/body length and weight) through Week 52(Baseline, Day 29, Week 8, Week 13, Week 26, Week 39, Week 52)
- Change from Baseline in ENPP1 activity (µM/min) through week 52(52 weeks (Randomized Treatment Period))
- Change from Baseline in skeletal abnormalities as measured by the Radiographic Global Impression of Change (RGI-C) global score through Week 52(Baseline, Week 26, Week 52)
- Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701(52 weeks (Randomized Treatment Period))
- Change from Baseline in rickets as measured by Rickets Severity Score (RSS) total score through Week 52(Baseline, Week 26, Week 52)