RecruitingPhase 3

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency

Brazil, France, Hungary12 participantsStarted 2025-03-26

Plain-language 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.

Age range0 Years – 1 Year

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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.

✕. 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.

To determine if INZ-701 increases inorganic pyrophosphate (PPi) levels

Timeframe: 52 weeks (Baseline through Week 52)

To determine if INZ-701 increases overall survival

Timeframe: 52 weeks (Baseline through Week 52)

NCT IDNCT07473973

SponsorInozyme Pharma

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-09-11

Trial Specialist