RecruitingPhase 3NCT07473973
ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants With Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
Sponsor
Inozyme Pharma
Enrollment
12 participants
Start Date
Mar 26, 2025
Study Type
INTERVENTIONAL
Conditions
Summary
The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
Eligibility
Min Age: 0 YearsMax Age: 1 Year
Inclusion Criteria6
- Participants must meet all of the following:
- Infant aged ≤ 1 year at the time of enrollment.
- Confirmed diagnosis of ENPP1 deficiency, based on genetic testing.
- Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement).
- Medically stable to participate in a 52-week treatment study.
- Written informed consent provided by a parent or legal guardian.
Exclusion Criteria5
- Participants will not be eligible if any of the following apply:
- Receiving end-of-life or hospice care.
- Prior treatment with INZ-701, unless received through an approved expanded access program.
- Concurrent participation in another interventional clinical trial.
- Planned major surgery during the study period that would interfere with study participation.
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Interventions
DRUGINZ-701
Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.
Locations(8)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07473973