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.
Single group
INZ-701
DrugRecombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.
To determine if INZ-701 increases inorganic pyrophosphate (PPi) levels
For each subject, their change from baseline in Plasma Inorganic Pyrophosphate (PPi) concentration will be assessed.
To determine if INZ-701 increases overall survival
For each subject, their change in overall survival based on time from date of birth to event of all-cause mortality will be assessed.
To determine if INZ-701 prevents decline in cardiac ejection fraction
For each subject, their change from baseline in left ventricular ejection fraction will be assessed via echocardiography.
To determine if INZ-701 prevents heart failure
For each subject, their incidence of heart failure will be assessed.
To determine if INZ-701 attenuates progression of arterial calcification
For each subject, their change from baseline in vascular calcification in the coronary arteries and aorta will be examined via CT scan.
Click on the lead sponsor to view all of their trials.
Inozyme Pharma
Lead sponsor
BioMarin Pharmaceutical
Collaborator
