The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants with Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-507384-20-00

Acronym

INZ701-104

Enrollment

Registered

2024-03-11

Start date

2024-10-21

Completion date

2025-04-10

Last updated

2025-04-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency

Brief summary

Adverse events (incidence, frequency, and severity of AEs, TEAEs, AESIs, and SAEs), Vital signs and weight, Laboratory tests including chemistry, hematology, and if feasible, urine tests, Immunogenicity, Concomitant medications, Left ventricular ejection fraction via echocardiogram, Electrocardiogram

Detailed description

Measurement of INZ-701 serum concentration-time profiles and PK parameters, Change from Baseline over time in PPi levels through Week 52, Measurement of ENPP1 activity, Please refer to the protocol for Exploratory endpoints

Interventions

DRUGINZ-701

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Adverse events (incidence, frequency, and severity of AEs, TEAEs, AESIs, and SAEs), Vital signs and weight, Laboratory tests including chemistry, hematology, and if feasible, urine tests, Immunogenicity, Concomitant medications, Left ventricular ejection fraction via echocardiogram, Electrocardiogram	—

Secondary

Measure	Time frame
Measurement of INZ-701 serum concentration-time profiles and PK parameters, Change from Baseline over time in PPi levels through Week 52, Measurement of ENPP1 activity, Please refer to the protocol for Exploratory endpoints	—

Countries

Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026