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Recruiting NCT07473973

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency

Phase III Interventional Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Autosomal Recessive Hypophosphatemic Rickets Generalized Arterial Calcification of Infancy 1

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 1. Basic parameters: 0 years — 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
Brazil, France, Hungary, Italy, Saudi Arabia +3
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 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants With Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

Overview

The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.

Detailed description

ENPP1 Deficiency is an ultra-rare genetic disorder in which inactivating mutations in the ENPP1 gene lead to a deficiency in the ENPP1 enzyme.

ENERGY 2 (Study INZ701-105) is a multicenter, single-arm, open-label Phase 3 study to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.

The study will consist of a Screening Period of up to 60 days, a Treatment Period of 52 weeks, a 52-week Extension Period, and an End of Treatment (EOT) Visit 30 days after the last dose of INZ-701.

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

  • To determine if INZ-701 increases inorganic pyrophosphate (PPi) levels [Time frame: 52 weeks (Baseline through Week 52)]
  • To determine if INZ-701 increases overall survival [Time frame: 52 weeks (Baseline through Week 52)]
Secondary outcome measures (3)
  • To determine if INZ-701 prevents decline in cardiac ejection fraction [Time frame: 52 weeks (Baseline through Week 52)]
  • To determine if INZ-701 prevents heart failure [Time frame: 52 weeks (Baseline through Week 52)]
  • To determine if INZ-701 attenuates progression of arterial calcification [Time frame: 52 weeks (Baseline through Week 52)]

Eligibility criteria

Participants must meet all of the following:

Inclusion criteria

  • Infant aged ≤ 1 year at the time of enrollment.
  • Confirmed diagnosis of ENPP1 deficiency, based on genetic testing.
  • Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement).
  • Medically stable to participate in a 52-week treatment study.
  • Written informed consent provided by a parent or legal guardian.

Exclusion criteria

Participants will not be eligible if any of the following apply:

  • Receiving end-of-life or hospice care.
  • Prior treatment with INZ-701, unless received through an approved expanded access program.
  • Concurrent participation in another interventional clinical trial.
  • Planned major surgery during the study period that would interfere with 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

Brazil · 1 center
  • Hospital Universitario Pedro Ernesto/Rio de Janeiro — Rio de Janeiro
France · 1 center
  • Hopital Necker - Enfants Malades — Paris
Hungary · 1 center
  • Gyermekgyogyaszat, DE — Debrecen
Italy · 1 center
  • Azienda Ospedaliera Universitaria Meyer — Florence
Saudi Arabia · 1 center
  • King Faisal Specialist Hospital and Research Center — Riyadh
Spain · 1 center
  • Hospital Sant Joan de Deu Edificio Consultas Externas. Unitat de Recerca — Barcelona
Turkey (Türkiye) · 1 center
  • Umraniye Traiing and Research Hospital — Istanbul
United Kingdom · 1 center
  • Royal Manchester Children's Hospital — Manchester

Identifiers

NCT: NCT07473973 · INZ701-105

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗