The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants 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
- 12
- 试验地点
- 15
- 主要终点
- To determine if INZ-701 increases inorganic pyrophosphate (PPi) levels
研究概览
简要总结
The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
详细描述
ENPP1 Deficiency is an ultra-rare genetic disorder in which inactivating mutations in the ENPP1 gene lead to a deficiency in the ENPP1 enzyme.
ENERGY 2 (Study INZ701-105) is a multicenter, single-arm, open-label Phase 3 study to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
The study will consist of a Screening Period of up to 60 days, a Treatment Period of 52 weeks, a 52-week Extension Period, and an End of Treatment (EOT) Visit 30 days after the last dose of INZ-701.
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
- 0 Years 至 1 Year（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must meet all of the following:
- •Inclusion Criteria:
- •Infant aged ≤ 1 year at the time of enrollment.
- •Confirmed diagnosis of ENPP1 deficiency, based on genetic testing.
- •Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement).
- •Medically stable to participate in a 52-week treatment study.
- •Written informed consent provided by a parent or legal guardian.
排除标准
- •Participants will not be eligible if any of the following apply:
- •Receiving end-of-life or hospice care.
- •Prior treatment with INZ-701, unless received through an approved expanded access program.
- •Concurrent participation in another interventional clinical trial.
- •Planned major surgery during the study period that would interfere with study participation.
研究组 & 干预措施
INZ-701
Participants receive INZ-701 (rhENPP1-Fc) administered by subcutaneous injection once weekly at the protocol-specified dose. The visit final volume to administer (mL) is determined using protocol-defined parameters.
干预措施: INZ-701 (Drug)
结局指标
主要结局
To determine if INZ-701 increases inorganic pyrophosphate (PPi) levels
时间窗: 52 weeks (Baseline through Week 52)
For each subject, their change from baseline in Plasma Inorganic Pyrophosphate (PPi) concentration will be assessed.
To determine if INZ-701 increases overall survival
时间窗: 52 weeks (Baseline through Week 52)
For each subject, their change in overall survival based on time from date of birth to event of all-cause mortality will be assessed.
次要结局
- To determine if INZ-701 prevents decline in cardiac ejection fraction(52 weeks (Baseline through Week 52))
- To determine if INZ-701 prevents heart failure(52 weeks (Baseline through Week 52))
- To determine if INZ-701 attenuates progression of arterial calcification(52 weeks (Baseline through Week 52))