Hope for kids with rare blood disease: new drug trial launches
NCT ID NCT06934967
First seen Dec 08, 2025 · Last updated May 19, 2026 · Updated 24 times
Summary
This study tests a drug called iptacopan in 12 children (ages 2 to 18) with a rare blood disease called PNH. The main goals are to see how the drug moves through the body and if it is safe. Participants will take the drug and be monitored for side effects and changes in their blood counts.
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 PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
-
Contact
Phone: •••-•••-••••
Locations
-
Cancer Institute of New Jersey
RECRUITINGNew Brunswick, New Jersey, 08901, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Childrens Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104 4399, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Novartis Investigative Site
RECRUITINGBrasília, Federal District, 70684-831, Brazil
-
Novartis Investigative Site
RECRUITINGNatal, Rio Grande do Norte, 59012 300, Brazil
-
Novartis Investigative Site
RECRUITINGPorto Alegre, Rio Grande do Sul, 90035-003, Brazil
-
Novartis Investigative Site
RECRUITINGSanto André, São Paulo, 09090-401, Brazil
-
Novartis Investigative Site
RECRUITINGSão Paulo, São Paulo, 01323001, Brazil
-
Novartis Investigative Site
RECRUITINGSão Paulo, São Paulo, 04038-002, Brazil
-
Novartis Investigative Site
RECRUITINGCali, Valle del Cauca Department, 760012, Colombia
-
Novartis Investigative Site
RECRUITINGBerlin, 13353, Germany
-
Novartis Investigative Site
RECRUITINGGenova, GE, 16147, Italy
-
Novartis Investigative Site
RECRUITINGUtrecht, 3584 CX, Netherlands
-
St Jude Childrens Research Hospital
RECRUITINGMemphis, Tennessee, 38105, United States
Contact Email: •••••@•••••
Contact
Conditions
Explore the condition pages connected to this study.