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 Pseudoxantho

Study Description

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.

Study Details

Registry ID:

NCT05030831

Conditions:

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

Phase:

Phase 1,Phase 2

Target Enrollment:

10 participants

Start Date:

April 11, 2022

Healthy Volunteers:

No

Study Locations (3 sites)

• Richmond Pharmacology Ltd (RPL)
  LondonUnited Kingdom, United Kingdom
• Clinilabs
  Eatontown, New JerseyUnited States, United States
• Icahn School of Medicine at Mount Sinai
  New York, New YorkUnited States, United States

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