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

clipboard-pencil

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.
user-3

Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION 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

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

pin location

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.
Phone iconCall +1 800-983-4587Email iconEmail Study Center

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?