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

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Trial Status Recruiting

Trial Identifier

NCT05734196

Condition

ENPP1 Deficiency, ABCC6 Deficiency, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, Pseudoxanthoma Elasticum

Notes

Following BioMarin’s acquisition of Inozyme in 2025, INZ-701 is now known as BMN 401.

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

Up to 1 Year

Healthy Volunteers

No

Drug/Treatment:

INZ-701

Phase:

Phase 1

Study Type:

Interventional

Number of Participants:

16

Study Started:

2023-06-25

Study Updated:

2026-03-24

Trial Locations

Inclusion Criteria

1. Infant aged ≤ 1 year at the time of enrollment
2. Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency
3. 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.
4. Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
5. Written informed consent provided by a parent or legal guardian

Exclusion Criteria

1. 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
2. Receiving end of life or hospice care
3. Known malignancy
4. Concurrent participation in another non-Inozyme interventional study
5. Treatment with any non-Inozyme product or investigational device during study participation

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