RECRUITING

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

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Conditions

Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic RicketsGeneralized Arterial Calcification of InfancyATP-Binding Cassette Subfamily C Member 6 DeficiencyPseudoxanthoma ElasticumhypopyrophosphatemiaENPP1GACIAutosomal Recessive Hypophosphatemic Rickets Type 2ARHR2ABCC6PXE

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2023-06-25
Study Completion:2026-04-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05734196

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 1 Year
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. 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 Council for Harmonisation (ICH) Good Clinical Practice (GCP)
  2. 2. Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory
  3. 3. Study participants must have clinical manifestations of GACI or GACI-2, which may include, but are not limited to, pathologic ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly
  4. 4. Study participant must be male or female from birth to \<1 year of age at Baseline (Day 1)
  5. 5. Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
  6. 6. In the opinion of the Investigator, the study participant must be able to complete all aspects of the study
  7. 7. Study participant's caregiver(s) must agree to provide access to their child's relevant medical records
  1. 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 or ABCC6 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. 2. Care has been withdrawn or study participant is receiving end of life care or hospice only
  3. 3. Known malignancy
  4. 4. Known intolerance to INZ-701 or any of its excipients
  5. 5. Concurrent participation in another non-Inozyme interventional study
  6. 6. Receipt of any non-Inozyme investigational new drug within 5 half-lives of the last dose of the other investigational product or within 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

Contacts and Locations

Study Contact

Inozyme Clinical Trial Information
CONTACT
+1 857 330 4340
clinicaltrials@inozyme.com

Principal Investigator

Alex Lai, MD
STUDY_DIRECTOR
Inozyme Pharma

Study Locations (Sites)

Rady Children's Hospital
San Diego, California, 92123
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
The University of Utah
Salt Lake City, Utah, 84108
United States

Collaborators and Investigators

Sponsor: Inozyme Pharma

  • Alex Lai, MD, STUDY_DIRECTOR, Inozyme Pharma

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-06-25
Study Completion Date2026-04-01

Study Record Updates

Study Start Date2023-06-25
Study Completion Date2026-04-01

Terms related to this study

Keywords Provided by Researchers

  • ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency
  • hypopyrophosphatemia
  • ENPP1
  • Generalized Arterial Calcification of Infancy
  • GACI
  • Autosomal Recessive Hypophosphatemic Rickets Type 2
  • ARHR2
  • ATP-Binding Cassette Subfamily C Member 6 Deficiency
  • ABCC6
  • Pseudoxanthoma elasticum
  • PXE

Additional Relevant MeSH Terms

  • Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
  • Autosomal Recessive Hypophosphatemic Rickets
  • Generalized Arterial Calcification of Infancy
  • ATP-Binding Cassette Subfamily C Member 6 Deficiency
  • Pseudoxanthoma Elasticum