RecruitingPhase 3

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

Brazil, France, Hungary12 participantsStarted 2025-03-26

Plain-language 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.

Who can participate

Age range

0 Years – 1 Year

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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.

Exclusion criteria

. 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: 52 weeks (Baseline through Week 52)

To determine if INZ-701 increases overall survival

Timeframe: 52 weeks (Baseline through Week 52)

Trial details

NCT IDNCT07473973

SponsorInozyme Pharma

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-09-11

Contact for this trial

Trial Specialist

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

View on ClinicalTrials.gov More Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency trials