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

NCT07473973 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: INZ701-105
• Study Type: interventional
• Target: 12
• Locations: 8 countries
• Sites: 8

Brief Summary

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

Conditions

Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency; Autosomal Recessive Hypophosphatemic Rickets; Generalized Arterial Calcification of Infancy 1

Keywords

Generalized Arterial Calcification of Infancy; GACI; Autosomal Recessive Hypophosphatemic Rickets Type 2; ARHR2

Outcome Measures

Primary Outcomes (2)

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

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

  52 weeks (Baseline through Week 52)

• To determine if INZ-701 increases overall survival

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

  52 weeks (Baseline through Week 52)

Secondary Outcomes (3)

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

  52 weeks (Baseline through Week 52)

• To determine if INZ-701 prevents heart failure

  52 weeks (Baseline through Week 52)

• To determine if INZ-701 attenuates progression of arterial calcification

  52 weeks (Baseline through Week 52)

Study Arms (1)

INZ-701

EXPERIMENTAL

Participants receive INZ-701 (rhENPP1-Fc) administered by subcutaneous injection once weekly at the protocol-specified dose. The visit final volume to administer (mL) is determined using protocol-defined parameters.

Drug: INZ-701

Interventions

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.

Also known as: (rhENPP1-Fc).

INZ-701

Eligibility Criteria

• Age: 0 Years - 1 Year
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Inozyme Pharma: lead
• BioMarin Pharmaceutical: collaborator

Study Sites (8)

Hospital Universitario Pedro Ernesto/Rio de Janeiro

Rio de Janeiro, 20551-030, Brazil

RECRUITING

Hopital Necker - Enfants Malades

Paris, 75015, France

RECRUITING

Gyermekgyogyaszat, DE

Debrecen, Hungary

RECRUITING

Azienda Ospedaliera Universitaria Meyer

Florence, 50139, Italy

RECRUITING

King Faisal Specialist Hospital and Research Center

Riyadh, 12713, Saudi Arabia

RECRUITING

Hospital Sant Joan de Deu Edificio Consultas Externas. Unitat de Recerca

Barcelona, Esplugues de Llobregat, 08950, Spain

RECRUITING

Umraniye Traiing and Research Hospital

Istanbul, Turkey (Türkiye)

RECRUITING

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

RECRUITING

MeSH Terms

Conditions

Hypophosphatemic Rickets, Autosomal Recessive, 1; Arterial calcification of infancy

Study Officials

• Medical Director, MD

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Central Study Contacts

Trial Specialist

CONTACT

+1 800-983-4587; medinfo@bmrn.com

Medical Director, MD

CONTACT

+1 800-983-4587; medinfo@bmrn.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: March 11, 2026
• First Posted: March 16, 2026
• Study Start: March 26, 2025
• Primary Completion (Estimated): September 11, 2028
• Study Completion (Estimated): November 7, 2028
• Last Updated: March 18, 2026

Record last verified: 2026-03

Locations

ES (1); SA (1); BR (1); IT (1); HU (1); TU (1); GB (1); FR (1)