New hope for babies: drug targets rare genetic disorder that hardens arteries
NCT ID NCT07473973
First seen Jun 27, 2026 · Last updated Aug 11, 2026 · Updated 2 times
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Azienda Ospedaliera Universitaria Meyer
Florence, 50139, Italy
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Gyermekgyogyaszat, DE
Debrecen, Hungary
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Hopital Necker - Enfants Malades
Paris, 75015, France
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Hospital Sant Joan de Deu Edificio Consultas Externas. Unitat de Recerca
Barcelona, Esplugues de Llobregat, 08950, Spain
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Hospital Universitario Pedro Ernesto/Rio de Janeiro
Rio de Janeiro, 20551-030, Brazil
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King Faisal Specialist Hospital and Research Center
Riyadh, 12713, Saudi Arabia
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Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
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Umraniye Traiing and Research Hospital
Istanbul, Turkey (Türkiye)
More trials for these conditions
Other studies related to the condition(s) this trial covers.