Overview
The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
Description
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.
Eligibility
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.
Exclusion Criteria
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.
