New hope for babies with rare bone and heart disease: experimental drug enters final testing
NCT ID NCT07473973
First seen Mar 16, 2026 · Last updated May 07, 2026 · Updated 6 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 disease can cause serious heart and bone problems. The goal is to see if the medicine can raise certain blood levels, improve survival, and protect the heart. About 12 babies will take part in this 52-week study.
Disclaimer
Read more
Show less
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.
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.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Azienda Ospedaliera Universitaria Meyer
RECRUITINGFlorence, 50139, Italy
-
Gyermekgyogyaszat, DE
RECRUITINGDebrecen, Hungary
-
Hopital Necker - Enfants Malades
RECRUITINGParis, 75015, France
-
Hospital Sant Joan de Deu Edificio Consultas Externas. Unitat de Recerca
RECRUITINGBarcelona, Esplugues de Llobregat, 08950, Spain
-
Hospital Universitario Pedro Ernesto/Rio de Janeiro
RECRUITINGRio de Janeiro, 20551-030, Brazil
-
King Faisal Specialist Hospital and Research Center
RECRUITINGRiyadh, 12713, Saudi Arabia
-
Royal Manchester Children's Hospital
RECRUITINGManchester, M13 9WL, United Kingdom
-
Umraniye Traiing and Research Hospital
RECRUITINGIstanbul, Turkey (Türkiye)
Conditions
Explore the condition pages connected to this study.