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Study identifier: NCT04686175 Synced from ClinicalTrials.gov · July 30, 2026
● Completed

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency

Condition: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency · Autosomal Recessive Hypophosphatemic Rickets · Generalized Arterial Calcification of Infancy  ·  Sponsor: Inozyme Pharma

PhasePhase 1/Phase 2
Planned participants9
Who can joinAll sexes, 18 Years to 64 Years
Healthy volunteersNo

About this study

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) enzyme replacement therapy, for the treatment of ENPP1 Deficiency. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ENPP1 Deficiency.

This description comes directly from the study's public registry record.

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Contact details are listed per location below or on the registry record.

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Locations (7)

Mayo ClinicRochester, Minnesota, United States
Clinilabs Drug Development CorporationEatontown, New Jersey, United States
University of SaskatchewanSaskatoon, Saskatchewan, Canada
Necker University Hospital-Sick ChildrenParis, France
Parexel International GmbHBerlin, Germany
University of Hamburg (Universitatklinikum Hamburg-Eppendorf)Hamburg, Germany
Richmond Pharmacology (RPL)London, London Bridge, United Kingdom

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Source record: clinicaltrials.gov/study/NCT04686175