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NCT Number: NCT07473973

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

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

Recruiting

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Key information

Conditions

Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency Arterial calcification of infancy Autosomal Recessive Hypophosphatemic Rickets Generalized Arterial Calcification of Infancy 1 Hypophosphatemic Rickets, Autosomal Recessive, 1

Age range

0 year–1 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Hospital Universitario Pedro Ernesto/Rio de Janeiro, Rio de Janeiro, Brazil

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

INZ-701

Drug

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

Other names: (rhENPP1-Fc).

Primary outcomes

  1. To determine if INZ-701 increases inorganic pyrophosphate (PPi) levels

    Time frame: 52 weeks (Baseline through Week 52)

    For each subject, their change from baseline in Plasma Inorganic Pyrophosphate (PPi) concentration will be assessed.

  2. To determine if INZ-701 increases overall survival

    Time frame: 52 weeks (Baseline through Week 52)

    For each subject, their change in overall survival based on time from date of birth to event of all-cause mortality will be assessed.

Secondary outcomes

  1. To determine if INZ-701 prevents decline in cardiac ejection fraction

    Time frame: 52 weeks (Baseline through Week 52)

    For each subject, their change from baseline in left ventricular ejection fraction will be assessed via echocardiography.

  2. To determine if INZ-701 prevents heart failure

    Time frame: 52 weeks (Baseline through Week 52)

    For each subject, their incidence of heart failure will be assessed.

  3. To determine if INZ-701 attenuates progression of arterial calcification

    Time frame: 52 weeks (Baseline through Week 52)

    For each subject, their change from baseline in vascular calcification in the coronary arteries and aorta will be examined via CT scan.

Study contacts

Contact information is provided by the study sponsor or research team.

Medical Director, MD

CONTACT

[email protected]

+1 800-983-4587

Trial Specialist

CONTACT

[email protected]

+1 800-983-4587

Sponsors and collaborators

Lead sponsor

Inozyme Pharma

Industry

Collaborators

  • BioMarin Pharmaceutical

Registry information

Official study 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

Important dates

Study start
2025
Primary completion
2028
Study completion
2028
First posted
Mar 16, 2026
Registry last updated
Mar 18, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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