Summary

Eligibility
for people ages up to 1 year (full criteria)
Location
at San Diego, California and other locations
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.

Official Title

The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infant Subjects With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) 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.

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.

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 study participants may continue to receive INZ-701, and an End of Treatment (EOT) visit 30 days after the last dose of INZ-701. Upon treatment discontinuation, study participants may continue to be followed for their ongoing disposition for survival outcome at least quarterly, if feasible through the end of the study. If the study site is not able to complete the survival outcome follow-up for up to 26 weeks post-treatment discontinuation, then the study participant can be considered "lost to follow up.

Keywords

Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, hypopyrophosphatemia, ENPP1, GACI, Autosomal Recessive Hypophosphatemic Rickets Type 2, ARHR2, Rickets, Hypophosphatemic Rickets, Familial Hypophosphatemic Rickets, Calcinosis, Vascular Calcification, INZ-701

Eligibility

You can join if…

Open to people ages up to 1 year

  1. Caregiver(s) must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP).
  2. Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory or local equivalent.
  3. Study participant must be male or female from birth (newborn) to <1 year of age at Baseline (Day 1).
  4. Study participant must weigh ≥ 0.5 kg at the time of the first dose of INZ-701
  5. In the opinion of the Investigator, the subject must be able to complete all aspects of the study
  6. Study participant's caregiver(s) must agree to provide access to their child's relevant medical records

You CAN'T join if...

  1. In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality (outside of those considered associated with the diagnosis of ENPP1 Deficiency) that precludes study participation or may confound interpretation of study results, including known uncontrolled thyroid disease or unrelated connective tissue, bone, mineral, or muscle disease
  2. Care has been withdrawn or subject is receiving end of life care or hospice only
  3. Known malignancy
  4. Known intolerance to INZ-701 or any of its excipients
  5. Concurrent participation in another non-Inozyme interventional study and/or receipt of any other investigational new drug within 5 half-lives of the last dose of the other investigational product or from 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device, through completion of participation in the study

Locations

  • Rady Children's Hospital accepting new patients
    San Diego California 92123 United States
  • Nationwide Children's Hospital accepting new patients
    Columbus Ohio 43205 United States
  • The Children's Hospital of Philadelphia accepting new patients
    Philadelphia Pennsylvania 19104 United States
  • Boston Children's Hospital accepting new patients
    Boston Massachusetts 02115 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 8 study participants
Last Updated