NCT05734196

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency

The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 16 (estimated)
• Sponsor: Inozyme Pharma · Industry
• Sex: All
• Age: 1 Year
• Healthy volunteers: Not accepted

Summary

The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.

Detailed description

INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy in development for the treatment of the ultra-rare genetic disorder, ENPP1 Deficiency or with ABCC6 Deficiency. Study INZ701-104 (the ENERGY study) is a Phase 1b, open-label study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in infant study participants with ENPP1 Deficiency or ABCC6 Deficiency. The study will consist of up to a 60-day Screening Period, a 52-week Treatment Period during which study participants will receive INZ-701, an Extension Period during which participants may continue to receive INZ-701 until it is commercially available in the country where the participant resides, or until an alternative study of INZ-701 is available, and an End of Treatment (EOT) visit 30 days after the last dose of INZ-701. Upon treatment discontinuation, participants will continue to be followed for their ongoing disposition for survival outcome at least quarterly through the end of the study.

Conditions

• Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
• Autosomal Recessive Hypophosphatemic Rickets
• Generalized Arterial Calcification of Infancy
• ATP-Binding Cassette Subfamily C Member 6 Deficiency
• Pseudoxanthoma Elasticum

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.

Timeline

• Start date: 2023-06-25
• Primary completion: 2027-11-11
• Completion: 2027-11-11
• First posted: 2023-02-17
• Last updated: 2026-03-24

Locations

7 sites across 3 countries: United States, Spain, United Kingdom

Regulatory

• FDA-regulated drug study