Cure My Disease

Don't miss your cure!

 Log in / Sign up

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

NCT ID NCT07473973

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 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.

Disclaimer Read more

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE1 DEFICIENCY are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

Study contacts

  • Contact

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

  • Contact

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

Locations

  • Azienda Ospedaliera Universitaria Meyer

    RECRUITING

    Florence, 50139, Italy

  • Gyermekgyogyaszat, DE

    RECRUITING

    Debrecen, Hungary

  • Hopital Necker - Enfants Malades

    RECRUITING

    Paris, 75015, France

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

    RECRUITING

    Barcelona, Esplugues de Llobregat, 08950, Spain

  • Hospital Universitario Pedro Ernesto/Rio de Janeiro

    RECRUITING

    Rio de Janeiro, 20551-030, Brazil

  • King Faisal Specialist Hospital and Research Center

    RECRUITING

    Riyadh, 12713, Saudi Arabia

  • Royal Manchester Children's Hospital

    RECRUITING

    Manchester, M13 9WL, United Kingdom

  • Umraniye Traiing and Research Hospital

    RECRUITING

    Istanbul, Turkey (Türkiye)