ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
Recruiting
Phase 3
Interventional Study
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
Autosomal Recessive Hypophosphatemic Rickets
Generalized Arterial Calcification of Infancy 1
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 0 – 1
- Sex
- Any
- Trial phase
- Phase 3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 12 (estimated)
- Sponsor
- Inozyme Pharma · Industry
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About This Trial
The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
Trial Locations
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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 calcif…
Contacts