New hope for babies with rare calcification disease: experimental drug enters human testing

NCT ID NCT05734196

First seen Mar 29, 2026 · Last updated May 14, 2026 · Updated 7 times

Summary

This study tests an experimental drug called INZ-701 in 16 infants (up to 1 year old) with rare genetic conditions (ENPP1 or ABCC6 deficiency) that cause dangerous calcium buildup in blood vessels and other tissues. The main goal is to check the drug's safety and how the body processes it. Researchers will monitor side effects, heart function, and blood markers to see if the drug can help control the disease.

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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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Contacts and locations

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    WITHDRAWN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    WITHDRAWN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

Conditions

Explore the condition pages connected to this study.