NCT07473973
ENPP1 Deficiency, Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy 1
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
All
0 Years to 1 Year
No
INZ-701
Phase 3
Interventional
12
2025-03-26
2026-03-18
Rio de Janeiro, Brazil
Trial Status Recruiting
Paris, France
Trial Status Recruiting
Debrecen, Hungary
Trial Status Recruiting
Florence, Italy
Trial Status Recruiting
Riyadh, Saudi Arabia
Trial Status Recruiting
Barcelona, Esplugues de Llobregat, Spain
Trial Status Recruiting
Istanbul, Turkey (Türkiye)
Trial Status Recruiting
Manchester, United Kingdom
Trial Status Recruiting
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 e
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.
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