The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
About the study
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.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
Inclusion Criteria:
- 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 CRITERIA
Exclusion Criteria:
- 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
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study Details
Contition
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency,Autosomal Recessive Hypophosphatemic Rickets,Generalized Arterial Calcification of Infancy,ATP-Binding Cassette Subfamily C Member 6 Deficiency,Pseudoxanthoma Elasticum
Age (in years)
< 1
Phase
PHASE1
Participants Needed
16
Est. Completion Date
Nov 11, 2027
Treatment Type
INTERVENTIONAL
Sponsor
Inozyme Pharma
ClinicalTrials.gov NCT Identifier
NCT05734196
Study Number
INZ701-104
Understanding Clinical Trials
Get answers to your questions about clinical trials.What is clinical research?What does taking part in clinical trials involve?What should I ask the trial doctor?