The ENERGY 3 Study: A Randomized, Controlled, Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Children with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-507382-26-00

Acronym

INZ701-106

Enrollment

Registered

2024-03-06

Start date

2024-05-24

Completion date

Unknown

Last updated

2025-07-30

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

Brief summary

Change from Baseline over time in plasma PPi concentration through Week 52, Change from Baseline in RGI-C global score through Week 52

Detailed description

Change from Baseline in RSS total score through Week 52, Change from Baseline in growth Z-score (height/body length and weight) through Week 52, Measurement of INZ-701 serum concentration and specific activity, Please refer to the protocol for Tertiary and Safety endpoints

Interventions

DRUGINZ-701

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Change from Baseline over time in plasma PPi concentration through Week 52, Change from Baseline in RGI-C global score through Week 52	—

Secondary

Measure	Time frame
Change from Baseline in RSS total score through Week 52, Change from Baseline in growth Z-score (height/body length and weight) through Week 52, Measurement of INZ-701 serum concentration and specific activity, Please refer to the protocol for Tertiary and Safety endpoints	—

Countries

France, Spain

Outcome results

None listed

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