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Active Not Recruiting
NCT05734196
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
Conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
Sex: All
Ages: N/A – 1 Year
Healthy volunteers: No
Phase: PHASE1
Enrollment: 16
Sponsor: Inozyme Pharma
Location: Rady Children's Hospital San Diego California
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 Criteria
Inclusion Criteria:
1. Infant aged ≤ 1 year at the time of enrollment
2. Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency
3. 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.
4. Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
5. Written informed consent provided by a parent or legal guardian
Exclusion Criteria:
1. 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
2. Receiving end of life or hospice care
3. Known malignancy
4. Concurrent participation in another non-Inozyme interventional study
5. Treatment with any non-Inozyme product or investigational device during study participation
Source: ClinicalTrials.gov (NCT05734196). StuddyBuddy aggregates publicly available trial information.