INZ-701: Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.
Study 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
Sex
ALL
Min age
0 Years
Max age
1 Year
Healthy volunteers
No
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.
Primary outcome measure(s)
To determine if INZ-701 increases inorganic pyrophosphate (PPi) levels — 52 weeks (Baseline through Week 52) For each subject, their change from baseline in Plasma Inorganic Pyrophosphate (PPi) concentration will be assessed.
To determine if INZ-701 increases overall survival — 52 weeks (Baseline through Week 52) For each subject, their change in overall survival based on time from date of birth to event of all-cause mortality will be assessed.
Trial sites (8)
Facility
City
Region
Status
Hospital Universitario Pedro Ernesto/Rio de Janeiro
Rio de Janeiro
Brazil
Hopital Necker - Enfants Malades
Paris
France
Gyermekgyogyaszat, DE
Debrecen
Hungary
Azienda Ospedaliera Universitaria Meyer
Florence
Italy
King Faisal Specialist Hospital and Research Center
Riyadh
Saudi Arabia
Hospital Sant Joan de Deu Edificio Consultas Externas. Unitat de Recerca
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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