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

NCT05734196 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2026-03-24

No results posted yet for this study

Summary

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.

Conditions

  • Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
  • Autosomal Recessive Hypophosphatemic Rickets
  • Generalized Arterial Calcification of Infancy
  • ATP-Binding Cassette Subfamily C Member 6 Deficiency
  • Pseudoxanthoma Elasticum

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.

Sponsors & Collaborators

Principal Investigators

  • Medical Director, MD, MD · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
1 Year
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-06-25
Primary Completion
2027-11-11
Completion
2027-11-11
FDA Drug
Yes

Countries

  • United States
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05734196 on ClinicalTrials.gov