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

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

Trial statusRecruiting

Trial phasePhase 1

Trial typeInterventional

Biological sexAll

AgeUp to 1

SponsorInozyme Pharma

About this trial

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 criteria

Qualifiers

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

Disqualifiers

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