drugset / Trial / NCT05030831

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE

NCT05030831

Phase 1/2 Completed 10 enrolled Inozyme Pharma IQVIA Biotech · collab
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) recombinant fusion protein, for the treatment of ABCC6 Deficiency. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ABCC6 Deficiency.

Timeline

Start
2022-04-11
Primary completion
2024-07-09
Completion
2024-07-09

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