Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency and ATP-binding cassette sub-family C member 6 (ABCC6) Deficiency, Generalized Arterial Calcification of Infancy (GACI), Pseudoxanthoma elasticum (PXE)
Conditions
Brief summary
Incidence, frequency, and severity of adverse events (AEs), treatment-emergent adverse event (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs), Changes in clinical laboratory tests including chemistry, hematology, and urinalysis resulting in an AE, Concomitant medications, Titers of anti-drug antibodies (ADA)
Detailed description
INZ-701 plasma PK parameters measured by immunoassay and enzymatic activity, Plasma PPi concentrations based on a validated assay
Interventions
DRUGINZ-701
Sponsors
Inozyme Pharma Inc.
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence, frequency, and severity of adverse events (AEs), treatment-emergent adverse event (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs), Changes in clinical laboratory tests including chemistry, hematology, and urinalysis resulting in an AE, Concomitant medications, Titers of anti-drug antibodies (ADA) | — |
Secondary
| Measure | Time frame |
|---|---|
| INZ-701 plasma PK parameters measured by immunoassay and enzymatic activity, Plasma PPi concentrations based on a validated assay | — |
Countries
France, Germany
Outcome results
None listed