Phase 1/2Completed
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE
A Phase 1/2 Open-Label Multiple Ascending Dose Study of INZ-701 With a Long-Term Extension in Adults With ABCC6 Deficiency Manifesting as Pseudoxanthoma Elasticum (PXE)
- Sponsor
- Inozyme Pharma (Industry)
- Enrolment
- 10 enrolled
- Conditions
- ATP-Binding Cassette Subfamily C Member 6 Deficiency; Pseudoxanthoma Elasticum; Generalized Arterial Calcification of Infancy
- Interventions
- INZ-701
- Ages
- adults, older adults
- Registry
- NCT05030831
Full record on ClinicalTrials.gov
From the registry
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.
Primary outcomes
- Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) During the Dose Evaluation Period
- Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) During the Extension Period
History
Results first posted
Last registry update
Primary completion
Started
Registered