The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
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.
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.
Inozyme Pharma
Lead sponsor
BioMarin Pharmaceutical
Collaborator