Summary

Eligibility
for people ages up to 1 year (full criteria)
Location
at UCSD
Dates
study started
completion around

Description

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

Details

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 Extension Period during which participants may continue to receive INZ-701 until it is commercially available in the country where the participant resides, or until an alternative study of INZ-701 is available, and an End of Treatment (EOT) visit 30 days after the last dose of INZ-701. Upon treatment discontinuation, participants will continue to be followed for their ongoing disposition for survival outcome at least quarterly through the end of the study.

Keywords

Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, hypopyrophosphatemia, ENPP1, GACI, Autosomal Recessive Hypophosphatemic Rickets Type 2, ARHR2, ABCC6, PXE, Rickets, Hypophosphatemic Rickets, Familial Hypophosphatemic Rickets, Calcinosis, INZ-701

Eligibility

Locations

  • Rady Children's Hospital accepting new patients
    San Diego California 92123 United States
  • The University of Utah accepting new patients
    Salt Lake City Utah 84108 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Inozyme Pharma
ID
NCT05734196
Phase
Phase 1 research study
Study Type
Interventional
Participants
Expecting 16 study participants
Last Updated