Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency
Completed · Phase 1/Phase 2
Conditions studied: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy
In brief
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.
Key facts
- Study ID
- NCT04686175
- Run by
- Inozyme Pharma
- People needed
- 9
- Starts
- 2021-11-21
- Expected to finish
- 2024-12-13
- Last updated by the study team
- 2025-02-05
Who can join
Age: 18 and older, up to 64. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Mayo Clinic — Rochester, Minnesota, United States
- Clinilabs Drug Development Corporation — Eatontown, New Jersey, United States
- University of Saskatchewan — Saskatoon, Saskatchewan, Canada
- Necker University Hospital-Sick Children — Paris, France
- Parexel International GmbH — Berlin, Germany
- University of Hamburg (Universitatklinikum Hamburg-Eppendorf) — Hamburg, Germany
- Richmond Pharmacology (RPL) — London, London Bridge, United Kingdom
Full record on ClinicalTrials.gov
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