New oral drug shows promise for rare blood disorder PNH
NCT ID NCT05630001
First seen Jan 05, 2026 · Last updated May 13, 2026 · Updated 23 times
Summary
This study tested an oral medication called iptacopan in 52 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Participants switched from their usual intravenous treatments (eculizumab or ravulizumab) to iptacopan pills taken twice daily. The main goal was to see if iptacopan could maintain stable hemoglobin levels without needing blood transfusions. Results showed that iptacopan was effective in keeping hemoglobin levels stable, offering a potential oral alternative to lifelong infusions.
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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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Locations
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City Of Hope National Med Center
Duarte, California, 91010, United States
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Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
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Huntsman Cancer Institute Univ of Utah
Salt Lake City, Utah, 84112 0550, United States
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Lakes Research
Miami Lakes, Florida, 33014, United States
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Mass Gen Hosp Cancer Center
Boston, Massachusetts, 02114, United States
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Montefiore Medical Center
The Bronx, New York, 10461, United States
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Novartis Investigative Site
Nantes, 44093, France
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Novartis Investigative Site
Nice, 06202, France
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Novartis Investigative Site
Paris, 75475, France
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Novartis Investigative Site
Dresden, Saxony, 01307, Germany
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Novartis Investigative Site
Aachen, 52074, Germany
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Novartis Investigative Site
Essen, 45147, Germany
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Novartis Investigative Site
Ulm, 89081, Germany
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Novartis Investigative Site
Florence, FI, 50134, Italy
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Novartis Investigative Site
Bassano del Grappa, VI, 36061, Italy
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Novartis Investigative Site
Seoul, Seoul, 06351, South Korea
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Novartis Investigative Site
Barcelona, 08036, Spain
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Novartis Investigative Site
Istanbul, Fatih, 34093, Turkey (Türkiye)
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Novartis Investigative Site
Leeds, West Yorkshire, LS9 7TF, United Kingdom
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Novartis Investigative Site
London, SE5 9RS, United Kingdom
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Prisma Health Upstate
Greenville, South Carolina, 29615, United States
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USC Norris Cancer Center
Los Angeles, California, 90033, United States
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University Of Minnesota
Minneapolis, Minnesota, 55455, United States
Conditions
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