NCT06046820

The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency

The ENERGY 3 Study: A Randomized, Controlled, Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Children With Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 27 (actual)
• Sponsor: Inozyme Pharma · Industry
• Sex: All
• Age: 1 Year – 12 Years
• Healthy volunteers: Not accepted

Summary

The primary purpose of Study INZ701-106 (The ENERGY 3 Study) is to assess the efficacy and safety of INZ-701 in children with ENPP1 Deficiency.

Detailed description

INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy (ERT) in development for the treatment of ENPP1 Deficiency, an ultra-rare genetic disorder with an incidence of 1 in 64,000 pregnancies. Study INZ701-106 (The ENERGY 3 Study) is a multi-center, randomized in a 2:1 ratio, controlled, open-label Phase 3 study to evaluate the efficacy and safety of INZ-701 in children with ENPP1 Deficiency. The study will consist of a Screening Period of up to 52 days (including a washout period of up to 7 days for prohibited medications post-Randomization) and a Randomized Treatment Period (INZ-701 or control) of 52 weeks, followed by an Open-label Extension Period during which all study participants may receive INZ-701, and an End of Study (EOS) Safety visit 30 days after the last dose of INZ-701.

Conditions

• Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency
• Autosomal Recessive Hypophosphatemic Rickets
• Generalized Arterial Calcification of Infancy

Interventions

Type	Name	Description
DRUG	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.
DRUG	Control Arm (Conventional Therapy)	Conventional therapy is defined as oral phosphate supplements and calcitriol or other active forms of vitamin D3 (or analogs). No other agents for treatment of ENPP1 Deficiency are allowed in the control arm.

Timeline

• Start date: 2023-11-05
• Primary completion: 2026-01-01
• Completion: 2026-02-01
• First posted: 2023-09-21
• Last updated: 2025-05-01

Locations

15 sites across 9 countries: United States, Australia, Canada, France, Saudi Arabia, Spain, Turkey (Türkiye), United Arab Emirates, United Kingdom

Regulatory

• FDA-regulated drug study