New drug offers hope for babies with rare, deadly artery disease
NCT ID NCT07473973
First seen Mar 16, 2026 · Last updated May 22, 2026 · Updated 7 times
Summary
This study tests a new medicine called INZ-701 in babies up to 1 year old who have a rare genetic condition called ENPP1 deficiency. The condition causes severe hardening of the arteries and rickets. The goal is to see if the medicine can raise a key substance in the blood, improve survival, and protect the heart. About 12 infants will receive the drug for 52 weeks.
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the original study
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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.
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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Azienda Ospedaliera Universitaria Meyer
RECRUITINGFlorence, 50139, Italy
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Gyermekgyogyaszat, DE
RECRUITINGDebrecen, Hungary
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Hopital Necker - Enfants Malades
RECRUITINGParis, 75015, France
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Hospital Sant Joan de Deu Edificio Consultas Externas. Unitat de Recerca
RECRUITINGBarcelona, Esplugues de Llobregat, 08950, Spain
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Hospital Universitario Pedro Ernesto/Rio de Janeiro
RECRUITINGRio de Janeiro, 20551-030, Brazil
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King Faisal Specialist Hospital and Research Center
RECRUITINGRiyadh, 12713, Saudi Arabia
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Royal Manchester Children's Hospital
RECRUITINGManchester, M13 9WL, United Kingdom
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Umraniye Traiing and Research Hospital
RECRUITINGIstanbul, Turkey (Türkiye)
Conditions
Explore the condition pages connected to this study.