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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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

Locations

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Novartis Investigative Site

    Essen, 45147, Germany

  • Novartis Investigative Site

    Milan, MI, 20122, Italy

  • Novartis Investigative Site

    Seoul, 06351, South Korea

  • Novartis Investigative Site

    Barcelona, Catalonia, 08035, Spain

  • Novartis Investigative Site

    Madrid, 28009, Spain

  • Novartis Investigative Site

    Murcia, 30008, Spain

  • Novartis Investigative Site

    London, W12 0HS, United Kingdom

Conditions

Explore the condition pages connected to this study.