drugset
drugset / Trial / NCT04686175

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency

NCT04686175 ↗

Phase 1/2 Completed 9 enrolled Inozyme Pharma
NaSingle-groupOpen-labelTreatment

Summary

The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy, for the treatment of ENPP1 Deficiency. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ENPP1 Deficiency.

Timeline

Start
2021-11-21
Primary completion
2024-10-29
Completion
2024-12-13

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