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Terminated
NCT07473973
ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
Conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy 1
Sex: All
Ages: 0 Years – 1 Year
Healthy volunteers: No
Phase: PHASE3
Enrollment: 2
Sponsor: Inozyme Pharma
Location: Hospital Universitario Pedro Ernesto/Rio de Janeiro Rio de Janeiro
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 Criteria
Participants must meet all of the following:
Inclusion Criteria:
1. Infant aged ≤ 1 year at the time of enrollment.
2. Confirmed diagnosis of ENPP1 deficiency, based on genetic testing.
3. Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement).
4. Medically stable to participate in a 52-week treatment study.
5. Written informed consent provided by a parent or legal guardian.
Exclusion Criteria
Participants will not be eligible if any of the following apply:
1. Receiving end-of-life or hospice care.
2. Prior treatment with INZ-701, unless received through an approved expanded access program.
3. Concurrent participation in another interventional clinical trial.
4. Planned major surgery during the study period that would interfere with study participation.
Source: ClinicalTrials.gov (NCT07473973). StuddyBuddy aggregates publicly available trial information.
