NCT05030831

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

A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults With ABCC6 Deficiency Manifesting as Pseudoxanthoma Elasticum (PXE)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Inozyme Pharma · Industry
• Sex: All
• Age: 18 Years – 69 Years
• Healthy volunteers: Not accepted

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.

Detailed description

INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant fusion protein in development for the treatment of ABCC6 Deficiency, a rare genetic disorder. Study INZ701-201 is a Phase 1/2, multicenter, open-label, multiple ascending dose (MAD), dose-finding study followed by a long-term open-label Extension Period conducted in adults with ABCC6 Deficiency manifesting as Pseudoxanthoma elasticum (PXE). This study 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 dose and dose schedule (number of doses per week) for further clinical development in ABCC6 Deficiency. No placebo will be used in this study. Exploratory endpoints for the Extension Period of the study include evaluations of arterial and organ calcification, ophthalmologic, cardiac and renal parameters, and physical function as well as patient reported outcomes. Subject participation consists of a Screening Period of up to 60 days, a 32-day Dose Evaluation Period, and an Extension Period during which subjects may continue to receive INZ-701 (with options for self-, caregiver-, or healthcare provider administration) until INZ-701 is approved and available in the country where the subject resides or until an alternative study for subjects to continue receiving study drug is available. During the Extension Period, follow-up visits will be conducted every 4 weeks until Week 48 of the Extension Period and then every 12 weeks (remote or in-clinic) until the subject leaves the study. Subjects will complete a follow up visit 30 days after their last dose of INZ-701.

Conditions

• ATP-Binding Cassette Subfamily C Member 6 Deficiency
• Pseudoxanthoma Elasticum
• Generalized Arterial Calcification of Infancy

Interventions

Type	Name	Description
DRUG	INZ-701	INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody (rhENPP1-Fc).

Timeline

• Start date: 2022-04-11
• Primary completion: 2024-07-09
• Completion: 2024-07-09
• First posted: 2021-09-01
• Last updated: 2025-02-05

Locations

2 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study