The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infant Subjects With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency

INZ701-104

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.

2023-05-25

2025-03-01

2025-04-01

Recruiting

Early phase I

8

Inclusion Criteria: 1. Caregiver(s) must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP). 2. Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory or local equivalent. 3. Study participant must be male or female from birth (newborn) to \<1 year of age at Baseline (Day 1). 4. Study participant must weigh ≥ 0.5 kg at the time of the first dose of INZ-701 5. In the opinion of the Investigator, the subject must be able to complete all aspects of the study 6. Study participant's caregiver(s) must agree to provide access to their child's relevant medical records Exclusion Criteria: 1. In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality (outside of those considered associated with the diagnosis of ENPP1 Deficiency) that precludes study participation or may confound interpretation of study results, including known uncontrolled thyroid disease or unrelated connective tissue, bone, mineral, or muscle disease 2. Care has been withdrawn or subject is receiving end of life care or hospice only 3. Known malignancy 4. Known intolerance to INZ-701 or any of its excipients 5. Concurrent participation in another non-Inozyme interventional study and/or receipt of any other investigational new drug within 5 half-lives of the last dose of the other investigational product or from 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device, through completion of participation in the study

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2024-04-22