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

Generalized Arterial Calcification of Infancy Clinical Trial

Official title: 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 Recruiting

Clinical Trial 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

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

• NCT number: NCT06046820
• Study type: Interventional
• Source: Inozyme Pharma
• Contact: Inozyme Clinical Trial Information
• Phone: +1 857 330 4340
• Email: clinicaltrials@inozyme.com
• Status: Recruiting
• Phase: Phase 3
• Start date: November 5, 2023
• Completion date: June 2025