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.
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy
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 subjects with ENPP1 Deficiency. The study will consist of up to a 60-day Screening Period, a 52-week Treatment Period during which subjects will receive INZ-701, an Extension Period during which subjects 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, they will continue to be followed for their ongoing disposition for survival outcome at least quarterly, if feasible, through the end of the study.
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).
Subject must have a post-natal confirmed 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 equivalent.
Subject must be male or female ages ≥ 1 month to <1 year of age at Baseline
Subject must weigh ≥ 0.5 kg at the time of the first dose of INZ-701
In the opinion of the Investigator, the subject must be able to complete all aspects of the study
Subject's caregiver(s) must agree to provide access to their child's relevant medical records
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
Care has been withdrawn or subject is receiving end of life care or hospice only
Known intolerance to INZ-701 or any of its excipients
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
September 28, 2023
Primary Contact Information
Andrea Hale, RN, MHP
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For more information on this trial, visit clinicaltrials.gov.
For more information and to contact the study team: