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

NCT05030831 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-02-05

No results posted yet for this study

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.

Conditions

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

Interventions

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).

Sponsors & Collaborators

  • IQVIA Biotech

    collaborator INDUSTRY

  • Inozyme Pharma

    lead INDUSTRY

Principal Investigators

  • Kurt Gunter, MD · Inozyme Pharma, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
69 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-04-11
Primary Completion
2024-07-09
Completion
2024-07-09
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05030831 on ClinicalTrials.gov