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New hope for babies with rare calcification disease: first drug trial launches

NCT ID NCT05734196

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 drug called INZ-701 in up to 16 infants (up to 1 year old) with rare genetic conditions (ENPP1 or ABCC6 deficiency) that cause dangerous calcium buildup in arteries and other problems. The main goal is to check the drug's safety and how the body handles it, while also looking at heart function and blood markers. This is an early-stage (Phase 1) trial, so it focuses on safety first, with potential for future treatments.

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 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 16 people

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

Started

Jun 2023

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

Up 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.

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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Conditions

The condition(s) this trial relates to.

arterial calcification of infancy autosomal recessive hypophosphatemic rickets autosomal recessive inherited pseudoxanthoma elasticum hypophosphatemic rickets, autosomal recessive, 1 pseudoxanthoma elasticum (inherited or acquired)

As listed by the trial registrant

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

Atp-binding cassette subfamily C member 6 deficiency Autosomal recessive hypophosphatemic rickets Ectonucleotide pyrophosphatase/PHOSPHODIESTERASE1 deficiency Generalized arterial calcification of infancy Pseudoxanthoma elasticum

Contacts and locations

Locations

  • Hospital Sant Joan de Déu

    Barcelona, Spain

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Rady Children's Hospital

    San Diego, California, 92123, United States

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL, United Kingdom

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.