Clinical Trials Logo

Clinical Trial Summary

The purpose of this study is to assess safety and tolerability of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant fusion protein, for the treatment of ABCC6 Deficiency (including Generalized Arterial Calcification of Infancy Type 2 [GACI-2] and Pseudoxanthoma elasticum [PXE]).


Clinical Trial Description

INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant fusion protein in development for the treatment of ABCC6 Deficiency (including Generalized Arterial Calcification of Infancy Type 2 [GACI-2] and Pseudoxanthoma elasticum [PXE]). This study is a Phase 1/2, multi-center, open-label, first-in-human (FIH), first-in-patient (FIP), and multiple ascending dose (MAD) study followed by a long-term open-label extension period conducted in adults with ABCC6 Deficiency manifesting as pseudoxanthoma elasticum (PXE). It is designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses of INZ-701. The goal of the study is to identify a safe, tolerable dose that restores inorganic pyrophosphate (PPi) to therapeutically effective levels in ABCC6 Deficiency to be used in further clinical development. Exploratory endpoints for the Extension Period of the study will include evaluations of cardiac, vascular, ophthalmologic, skeletal, and physical function as well as patient reported outcomes. Subject participation consists of a Screening Period, a 32-day Dose Evaluation Period, and a 48-week Extension Period following completion of the Dose Evaluation Period. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05030831
Study type Interventional
Source Inozyme Pharma
Contact Inozyme Clinical Trial Information
Phone +1 857 330 4340
Email [email protected]
Status Not yet recruiting
Phase Phase 1/Phase 2
Start date September 2021
Completion date December 2023

See also
  Status Clinical Trial Phase
Completed NCT02537054 - Intravitreal Aflibercept for Therapy of Patients With Pseudoxanthoma Elasticum (PXE) Phase 2
Not yet recruiting NCT04868578 - PyROphosPHate Supplementation to Fight ECtopIc Calcification in PseudoXanthoma Elasticum Phase 2
Completed NCT03070860 - What's Happen Under the Calcification Process in Pseudoxanthoma Elasticum N/A
Completed NCT01446393 - Functional and Structural Characterization of Arteriopathy in Pseudoxanthoma Elasticum (PXE) N/A
Completed NCT00470977 - Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy Phase 1/Phase 2
Recruiting NCT03758534 - Natural History of GACI With or Without ARHR2 or PXE
Recruiting NCT01446380 - Phenotypic Expressions in a French Pseudoxanthoma-Elasticum Cohort N/A
Recruiting NCT03813550 - Intestinal Microbiota and Vitamin K Levels in PXE Patients (IMPROVE Study) N/A
Not yet recruiting NCT03364504 - Biological Collection of Kidney Cells N/A
Active, not recruiting NCT02108392 - Characterization of Pseudoxanthoma Elasticum
Recruiting NCT01731080 - Arterial Wall Calcium Load in Pseudoxanthoma Elasticum N/A
Completed NCT00555113 - Evolution of Visual Impairment During Pseudoxanthoma Elasticum N/A
Completed NCT00341419 - Genetic Analysis of Patients With Pseudoxanthoma Elasticum
Withdrawn NCT04441671 - Oral Pyrophosphate Absorption in PXE Disease Phase 2
Recruiting NCT05025722 - Pseudoxanthoma Elasticum (PXE) Natural History Biomarkers in PXE Individuals and Their Biological Non-PXE Siblings
Completed NCT01525875 - Magnesium Supplements In The Treatment Of Pseudoxanthoma Elasticum (PXE) Phase 2