drugset
drugset / Trial / NCT05734196

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

NCT05734196 ↗

Phase 1 Recruiting 16 enrolled Inozyme Pharma BioMarin Pharmaceutical · collab
NaSingle-groupOpen-labelTreatment

Summary

The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.

Timeline

Start
2023-06-25
Primary completion
2027-11-11
Completion
2027-11-11

Drugs

EvaluationDrugModalityDoseRoute
Subject INZ-701 Protein / enzyme biologic 0.2 mg/kg —
Subject INZ-701 Protein / enzyme biologic 0.6 mg/kg —
Subject INZ-701 Protein / enzyme biologic 1.8 mg/kg —

Indications

arterial calcification of infancy autosomal recessive hypophosphatemic rickets pseudoxanthoma elasticum (inherited or acquired)