ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
Inozyme Pharma
200 participants
Jun 19, 2024
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria7
- 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
- Female participants of childbearing potential who are sexually active must be using or agree to use 1 highly effective form of contraception (per CTFG 2020) from at least 1 month before the first dose of INZ-701 through 30 days after last dose of INZ-701 (greater than 5 half-lives of INZ-701); participants must agree to not donate ova from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701
- Male participants who are sexually active must agree to use condoms from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701; participants must agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701
- In the opinion of the Investigator, able to complete all aspects of the study
Exclusion Criteria5
- 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
- Male participants trying to father a child
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Interventions
INZ-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).
Locations(5)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06462547