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Hope for babies with Ultra-Rare fatal disease: experimental drug enters final testing phase

NCT ID NCT07473973

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

Summary

This study is testing an investigational drug called INZ-701 in infants with a severe, ultra-rare genetic disorder called ENPP1 deficiency. The main goals are to see if the drug improves survival and prevents dangerous calcium buildup in the heart and blood vessels. About 12 infants under one year old will receive the drug for one year, with researchers closely monitoring their health.

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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)

Conditions

Explore the condition pages connected to this study.

AUTOSOMAL RECESSIVE HYPOPHOSPHATEMIC RICKETS ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE1 DEFICIENCY GENERALIZED ARTERIAL CALCIFICATION OF INFANCY 1