Experimental drug iptacopan tested for rare blood disorders

NCT ID NCT05086744

Terminated Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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, so results are limited.

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Conditions

The condition(s) this trial relates to.

autoimmune hemolytic anemia autoimmune thrombocytopenic purpura cold agglutinin disease Purpura, Thrombocytopenic, Idiopathic thrombocytopenia due to immune destruction

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

COLD AGGLUTININ DISEASE (CAD) IMMUNE THROMBOCYTOPENIA (ITP)

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

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