The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency

What this Trial Is About

Researchers are evaluating the efficacy and safety of INZ-701 in infants diagnosed with ENPP1 Deficiency, an ultra-rare genetic disorder caused by mutations in the ENPP1 gene leading to enzyme deficiency. This open-label, single-arm Phase 3 study focuses on understanding how this investigational drug may affect infants with related conditions including generalized arterial calcification of infancy (GACI) and autosomal recessive hypophosphatemic rickets. Participants in this study will receive INZ-701, a recombinant fusion protein, administered by weekly subcutaneous injections. The study includes a Screening Period lasting up to 60 days, followed by a 52-week Treatment Period, then a 52-week Extension Period. After the last dose, an End of Treatment visit will occur 30 days later to assess outcomes. During the trial, infants will be closely monitored through clinical evaluations to measure changes such as inorganic pyrophosphate levels, overall survival, cardiac function including ejection fraction, prevention of heart failure, and progression of arterial calcification. The study involves frequent visits and assessments over two years, with the aim to gather detailed safety and efficacy data on INZ-701 in this vulnerable population.

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency