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New hope for babies: drug targets rare genetic disorder that hardens arteries

NCT ID NCT07473973

Disease control Sponsor: Inozyme Pharma Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 11, 2026 · Updated 2 times

Summary

This study tests a new medicine called INZ-701 in babies up to 1 year old who have a rare genetic condition (ENPP1 deficiency) that causes severe hardening of the arteries and bone problems. The goal is to see if the drug can raise a key substance in the blood, improve survival, and protect the heart. About 12 infants will receive the treatment for 52 weeks to check safety and effectiveness.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2025

Expected to finish

Oct 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

0 years to 1 year

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Participants must meet all of the following: Inclusion Criteria: 1. Infant aged ≤ 1 year at the time of enrollment. 2. Confirmed diagnosis of ENPP1 deficiency, based on genetic testing. 3. Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement). 4. Medically stable to participate in a 52-week treatment study. 5. Written informed consent provided by a parent or legal guardian. Exclusion Criteria Participants will not be eligible if any of the following apply: 1. Receiving end-of-life or hospice care. 2. Prior treatment with INZ-701, unless received through an approved expanded access program. 3. Concurrent participation in another interventional clinical trial. 4. Planned major surgery during the study period that would interfere with study participation.

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Conditions

The condition(s) this trial relates to.

arterial calcification of infancy arterial calcification, generalized, of infancy, 1 autosomal recessive hypophosphatemic rickets hypophosphatemic rickets, autosomal recessive, 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Autosomal recessive hypophosphatemic rickets Ectonucleotide pyrophosphatase/PHOSPHODIESTERASE1 deficiency Generalized arterial calcification of infancy 1

Contacts and locations

Locations

  • Azienda Ospedaliera Universitaria Meyer

    Florence, 50139, Italy

  • Gyermekgyogyaszat, DE

    Debrecen, Hungary

  • Hopital Necker - Enfants Malades

    Paris, 75015, France

  • Hospital Sant Joan de Deu Edificio Consultas Externas. Unitat de Recerca

    Barcelona, Esplugues de Llobregat, 08950, Spain

  • Hospital Universitario Pedro Ernesto/Rio de Janeiro

    Rio de Janeiro, 20551-030, Brazil

  • King Faisal Specialist Hospital and Research Center

    Riyadh, 12713, Saudi Arabia

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL, United Kingdom

  • Umraniye Traiing and Research Hospital

    Istanbul, Turkey (Türkiye)

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