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

NCT ID NCT05734196

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••
Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Boston Children's Hospital
WITHDRAWN

Boston, Massachusetts, 02115, United States
Hospital Sant Joan de Déu
TERMINATED

Barcelona, Spain
Nationwide Children's Hospital
RECRUITING

Columbus, Ohio, 43205, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••

Contact Phone: •••-•••-•••• Email: •••••@•••••
Rady Children's Hospital
RECRUITING

San Diego, California, 92123, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••

Contact Phone: •••-•••-•••• Email: •••••@•••••
Royal Manchester Children's Hospital
TERMINATED

Manchester, M13 9WL, United Kingdom
The Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••

Contact Phone: •••-•••-•••• Email: •••••@•••••
The University of Utah
WITHDRAWN

Salt Lake City, Utah, 84108, United States