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
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.
Description
INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy in development for the treatment of the ultra-rare genetic disorder, ENPP1 Deficiency or with ABCC6 Deficiency. Study INZ701-104 (the ENERGY study) is a Phase 1b, open-label study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in infant study participants with ENPP1 Deficiency or ABCC6 Deficiency. The study will consist of up to a 60-day Screening Period, a 52-week Treatment Period during which study participants will receive INZ-701, an Extensi…
Eligibility
- Age range
- Up to 1 years
- Sex
- All
- Healthy volunteers
- No
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 pr…
Interventions
- DrugINZ-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 HospitalSan Diego, California
- Boston Children's HospitalBoston, Massachusetts
- Nationwide Children's HospitalColumbus, Ohio
- The Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania
- The University of UtahSalt Lake City, Utah
- Hospital Sant Joan de DéuBarcelona