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

a study on Rickets Generalized Arterial Calcification of Infancy Pseudoxanthoma Elasticum

Summary

Eligibility
for people ages up to 1 year (full criteria)
Location
at San Diego, California and other locations
Dates
study started
study ends 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, Hypophosphatemic Rickets, Autosomal Recessive, 1, Arterial calcification of infancy, INZ-701

Eligibility

You can join if…

Open to people ages up to 1 year

  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

You CAN'T join if...

  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

Locations

  • Rady Children's Hospital accepting new patients
    San Diego California 92123 United States
  • The University of Utah withdrawn
    Salt Lake City Utah 84108 United States
  • Nationwide Children's Hospital accepting new patients
    Columbus Ohio 43205 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
Contact us about this trial