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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)

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Trial Profile

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 of Trial: Phase I/II

Latest Information Update: 10 Feb 2025

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At a glance

  • Drugs INZ-701 (Primary)
  • Indications Calcinosis; Pseudoxanthoma elasticum
  • Focus Adverse reactions; Biomarker; First in man; Pharmacodynamics; Pharmacokinetics; Therapeutic Use
  • Sponsors Inozyme Pharma

    • Most Recent Events

    • 05 Feb 2025 Status changed from active, no longer recruiting to completed.
    • 06 Aug 2024 According to an Inozyme Pharma media release, positive topline data of this study, announced in April 2024, were presented at the European Calcified Tissue Society Congress (ECTS) 2024 in May in Marseille, France.
    • 28 May 2024 Planned End Date changed from 5 Dec 2023 to 31 Dec 2024.

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