Experimental drug for rare blood diseases shows early promise but trial halted
NCT ID NCT05086744
First seen Nov 01, 2025 · Last updated May 03, 2026 · Updated 21 times
Summary
This study tested an experimental drug called iptacopan in people with two rare blood disorders: immune thrombocytopenia (ITP) and cold agglutinin disease (CAD). The goal was to see if the drug could improve platelet counts in ITP or hemoglobin levels in CAD. The trial was terminated early, and only 19 people participated.
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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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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Novartis Investigative Site
Essen, 45147, Germany
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Novartis Investigative Site
Milan, MI, 20122, Italy
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Novartis Investigative Site
Seoul, 06351, South Korea
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Novartis Investigative Site
Barcelona, Catalonia, 08035, Spain
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Novartis Investigative Site
Madrid, 28009, Spain
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Novartis Investigative Site
Murcia, 30008, Spain
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Novartis Investigative Site
London, W12 0HS, United Kingdom
Conditions
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