The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
Recruiting now · Phase 1
Conditions studied: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
In brief
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.
Key facts
- Study ID
- NCT05734196
- Run by
- Inozyme Pharma
- People needed
- 16
- Starts
- 2023-06-25
- Expected to finish
- 2027-11-11
- Last updated by the study team
- 2026-03-24
Who can join
Age: any, up to 1. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
- Nationwide Children's Hospital — Columbus, Ohio, United States (enrolling)
- Rady Children's Hospital — San Diego, California, United States (enrolling)
- Royal Manchester Children's Hospital — Manchester, United Kingdom
- The University of Utah — Salt Lake City, Utah, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- Hospital Sant Joan de Déu — Barcelona, Spain
Full record on ClinicalTrials.gov
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