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Recruiting NCT05734196

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

Phase I Interventional Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Autosomal Recessive Hypophosphatemic Rickets Generalized Arterial Calcification of Infancy ATP-Binding Cassette Subfamily C Member 6 Deficiency

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: INZ-701.
Who it may be relevant to
Registry conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency. Basic parameters: up to 1 year · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Spain, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

Overview

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.

Detailed description

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.

Interventions

  • Drug INZ-701
    Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.

Primary outcome measures

  • Number of Treatment Emergent Adverse Events (TEAEs) [Time frame: 52 weeks (Treatment Period)]
  • Incidence of Anti-Drug Antibodies (ADA) [Time frame: 52 weeks (Treatment Period)]
  • Left Ventricular Ejection Fraction [Time frame: 52 weeks (Treatment Period)]
Secondary outcome measures (4)
  • Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels [Time frame: 52 weeks (Treatment Period)]
  • Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701 [Time frame: 52 weeks (Treatment Period)]
  • Maximum Plasma Concentration (Cmax) of INZ-701 [Time frame: 52 weeks (Treatment Period)]
  • ENPP1 Activity [Time frame: 52 weeks (Treatment Period)]

Eligibility criteria

Inclusion criteria

  • Infant aged ≤ 1 year at the time of enrollment
  • Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency
  • 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.
  • Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
  • Written informed consent provided by a parent or legal guardian

Exclusion criteria

  • 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
  • Receiving end of life or hospice care
  • Known malignancy
  • Concurrent participation in another non-Inozyme interventional study
  • Treatment with any non-Inozyme product or investigational device during study participation

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 5 centers
  • Rady Children's Hospital — San Diego
  • Boston Children's Hospital — Boston
  • Nationwide Children's Hospital — Columbus
  • The Children's Hospital of Philadelphia — Philadelphia
  • The University of Utah — Salt Lake City
Spain · 1 center
  • Hospital Sant Joan de Déu — Barcelona
United Kingdom · 1 center
  • Royal Manchester Children's Hospital — Manchester

Identifiers

NCT: NCT05734196 · INZ701-104

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗