The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants With ENPP1 or ABCC6 Deficiency

What this Trial Is About

This research aims to evaluate the safety and tolerability of INZ-701, an enzyme replacement therapy, in infants diagnosed with ENPP1 Deficiency or ABCC6 Deficiency—rare genetic disorders that can cause serious health problems like generalized arterial calcification of infancy (GACI). The trial is a Phase 1b, open-label study focusing on infants up to 1 year old who have these conditions and related clinical symptoms such as heart failure or respiratory distress. Participants will undergo a screening period of up to 60 days before receiving INZ-701 treatment over 52 weeks. The treatment involves different dosing levels of INZ-701 given by intravenous infusion, starting with low doses for the first participants and adjusted based on safety reviews. After the treatment period, there is an extension phase allowing continued treatment until the drug becomes commercially available or other studies are accessible. A follow-up visit occurs 30 days after the last dose, with ongoing monitoring for survival outcomes quarterly. Throughout the study, infants will be closely monitored through physical exams, laboratory tests, and heart function assessments including left ventricular ejection fraction. Researchers will track safety by recording adverse events and immune responses to the drug, as well as measuring drug levels in the blood and its effects on enzyme activity and plasma pyrophosphate levels. The total study duration includes screening, treatment, extension, and long-term follow-up to ensure comprehensive safety and effectiveness data collection.

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency