The purpose of this study (Study INZ701-304 \[ADAPT\]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
Provide written or electronic informed consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP)
Provide assent in accordance with local regulations, if <18 years of age
Male or female, greater than 1 year of age
Must have completed the protocol-required safety and PK/PD and/or efficacy period(s) of a previous INZ-701 clinical study in ENPP1 or ABCC6 Deficiency, as confirmed by the Sponsor
In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency or ABCC6 Deficiency, that will preclude study participation and/or may confound interpretation of study results
Known intolerance to INZ-701 or any of its excipients
Concurrent participation in another interventional clinical study and/or has received an investigational drug other than INZ-701 within 5 half-lives or within 4 weeks prior to the first dose of INZ-701 in this study, whichever is longer, or use of an investigational device
Pregnant, trying to become pregnant, or breastfeeding
Single group
INZ-701
DrugINZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody (rhENPP1-Fc).
Number of Treatment Emergent Adverse Events (TEAEs)
Treatment-emergent AEs are defined as any AE occurring from the first dose of INZ-701 through 30 days after the last dose of INZ-701.
Incidence of Anti-Drug Antibodies (ADA)
For each subject, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
Time to maximum serum concentration (Tmax)
For each subject, the time to reach maximum concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the participant's baseline value over time.
Maximum Plasma Concentration (Cmax) of INZ-701
For each subject, the maximum concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the participant's baseline value over time.collected and assessed.
Mean Change from Baseline in Plasma PPi Concentration
For each subject, their plasma PPi concentrations based on a validated assay will be collected and assessed throughout the study, comparing the participant's baseline value over time.
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