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The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
Sponsor: Inozyme Pharma
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.
Official title: The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency
Key Details
Gender
All
Age Range
Any - 1 Year
Study Type
INTERVENTIONAL
Enrollment
16
Start Date
2023-06-25
Completion Date
2027-11-11
Last Updated
2026-03-24
Healthy Volunteers
No
Conditions
Interventions
INZ-701
Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.
Locations (7)
Rady Children's Hospital
San Diego, California, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
The University of Utah
Salt Lake City, Utah, United States
Hospital Sant Joan de Déu
Barcelona, Spain
Royal Manchester Children's Hospital
Manchester, United Kingdom