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

Inclusion Criteria5

Infant aged ≤ 1 year at the time of enrollment
Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency
Study participants must have clinical manifestations of generalized arterial calcification of infancy (GACI) or GACI-2, which must include at least one of the following: ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly.
Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
Written informed consent provided by a parent or legal guardian

Exclusion Criteria5

In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality that precludes study participation or may confound interpretation of study result
Receiving end of life or hospice care
Known malignancy
Concurrent participation in another non-Inozyme interventional study
Treatment with any non-Inozyme product or investigational device during study participation

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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.