The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
Inozyme Pharma
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.
Description
The investigational product INZ-701 is being developed as a therapeutic protein for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency and adenosine triphosphate (ATP)-binding cassette subfamily C member 6 (ABCC6) Deficiency. INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin (Ig) G1 antibody. The ADAPT Study (INZ701-304) is an open-label study to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701…
Eligibility
- Age range
- 1+ years
- Sex
- All
- Healthy volunteers
- No
Individuals eligible to participate must meet all of the following inclusion criteria: 1. 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) 2. Provide assent in accordance with local regulations, if \<18 years of age 3. Male or female, greater than 1 year of age 4. Must have completed the protocol-required safety and PK/PD and/or efficacy period(s) of a previous IN…
Interventions
- DrugINZ-701
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)
- Mayo ClinicRochester, Minnesota
- Clinilabs Drug Development CorporationEatontown, New Jersey
- Necker-Enfants Malades HospitalParis
- Universitätsklinikum Hamburg-Eppendorf (UKE)Hamburg
- VCTCOxford