RecruitingPhase 1NCT05734196

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

Sponsor

Inozyme Pharma

Enrollment

16 participants

Start Date

Jun 25, 2023

Study Type

INTERVENTIONAL

Conditions

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

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.

Eligibility

Max Age: 1 Year

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called INZ-701 for people with atp-binding cassette subfamily c member 6 deficiency, autosomal recessive hypophosphatemic rickets, and other related conditions. The study is currently recruiting participants at 7 locations. People eligible for this study include up to age 1 Year.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGINZ-701

Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.