Hope for kids with rare blood disorder: new pill under study
NCT ID NCT06934967
First seen Dec 08, 2025 · Last updated May 15, 2026 · Updated 23 times
Summary
This study tests a medicine called iptacopan in 12 children aged 2 to 18 who have a rare blood disease called PNH. The goal is to see how the drug works in their bodies and if it is safe. PNH causes red blood cells to break apart, and this treatment aims to control that damage.
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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: •••-•••-••••
Locations
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Cancer Institute of New Jersey
RECRUITINGNew Brunswick, New Jersey, 08901, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Childrens Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104 4399, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Novartis Investigative Site
RECRUITINGBrasília, Federal District, 70684-831, Brazil
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Novartis Investigative Site
RECRUITINGNatal, Rio Grande do Norte, 59012 300, Brazil
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Novartis Investigative Site
RECRUITINGPorto Alegre, Rio Grande do Sul, 90035-003, Brazil
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Novartis Investigative Site
RECRUITINGSanto André, São Paulo, 09090-401, Brazil
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Novartis Investigative Site
RECRUITINGSão Paulo, São Paulo, 01323001, Brazil
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Novartis Investigative Site
RECRUITINGSão Paulo, São Paulo, 04038-002, Brazil
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Novartis Investigative Site
RECRUITINGCali, Valle del Cauca Department, 760012, Colombia
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Novartis Investigative Site
RECRUITINGBerlin, 13353, Germany
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Novartis Investigative Site
RECRUITINGGenova, GE, 16147, Italy
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Novartis Investigative Site
RECRUITINGUtrecht, 3584 CX, Netherlands
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St Jude Childrens Research Hospital
RECRUITINGMemphis, Tennessee, 38105, United States
Contact Email: •••••@•••••
Contact
Conditions
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