← Back to all trials
Completed
NCT05030831
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE
Conditions: ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, Generalized Arterial Calcification of Infancy
Sex: All
Ages: 18 Years – 69 Years
Healthy volunteers: No
Phase: PHASE1, PHASE2
Enrollment: 10
Sponsor: Inozyme Pharma
Location: Clinilabs Eatontown New Jersey
Summary
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.
Eligibility Criteria
Inclusion Criteria
Individuals eligible to participate must meet all of the following inclusion criteria:
1. Must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP)
2. Clinical diagnosis of PXE supported by prior genetic identification of biallelic ABCC6 mutations (ie, homozygous or compound heterozygous)
3. Male or female, 18 to \
Source: ClinicalTrials.gov (NCT05030831). StuddyBuddy aggregates publicly available trial information.
