Oral pill shows promise for rare blood disease, could replace injections
NCT ID NCT05630001
Summary
This study tested whether adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder, could safely switch from their standard injection treatment to a new oral medication called iptacopan. Fifty-two patients who were already doing well on their current treatment took iptacopan twice daily for 24 weeks. Researchers measured whether the pill could maintain their hemoglobin levels and reduce the need for blood transfusions.
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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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