Can a targeted drug outperform standard care for rare blood cancers?

NCT ID NCT02577926

First seen Aug 13, 2026 · Last updated Aug 14, 2026 · Updated 1 time

Summary

This trial compares the drug ruxolitinib against the best available standard treatments for people with high-risk polycythemia vera (PV) or essential thrombocythemia (ET), two rare blood cancers that cause overproduction of blood cells and can lead to clots, bleeding, and severe symptoms. Participants receive either ruxolitinib or a standard therapy chosen by their doctor, such as hydroxyurea or interferon. The main goal is to see if ruxolitinib leads to a higher rate of complete clinicohematologic response, meaning better control of blood counts and symptoms, while also monitoring side effects.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
Ruxolitinib, a JAK1/2 inhibitor, compared to best available therapy (e.g., hydroxyurea, anagrelide, interferon).
What this could lead to
If successful, ruxolitinib could become a more effective treatment option for high-risk PV and ET, potentially improving symptom control and reducing complications.
What could go wrong
This is a phase 2 trial, so results are preliminary. Ruxolitinib may not prove superior to existing therapies, and it carries risks like infection and blood count changes.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Charite Universitätsmedizin Berlin; Medizinische Klinik m.S. Hämatologie, Onkologie und Tumorimmunologie

    Berlin, 13353, Germany

  • III. Medizinischen Klinik des Klinikums rechts der Isar der TU München

    Müchen, Bavaria, 81675, Germany

  • Johanniter-Krankenhaus Rheinhausen GmbH Hämatologie / Internistische Onkologie / Tagesklinik

    Duisburg, North Rhine-Westphalia, 47228, Germany

  • Klinikum Chemnitz gGmbH Klinik für Innere Medizin III

    Chemnitz, Saxony, 09113, Germany

  • Klinikum Nürnberg Nord Medizinische Klinik 5

    Nuremberg, Bavaria, 90419, Germany

  • Marienhospital

    Düsseldorf, North Rhine-Westphalia, 40479, Germany

  • Mühlenkreiskliniken Johannes Wesling Klinikum Minden Klinik für Hämatologie, Onkologie und Palliativmedizin

    Minden, North Rhine-Westphalia, 32429, Germany

  • Rems-Murr Klinikum Winnenden

    Winnenden, Baden-Wurttemberg, 71364, Germany

  • Studienzentrum Aschaffenburg

    Aschaffenburg, Bavaria, 63739, Germany

  • UNIVERSITÄTSKLINIKUM Schleswig-Holstein - Klinik für Hämatologie und Onkologie, Campus Lübeck

    Lübeck, Germany

  • Uniklinik RWTH Aachen

    Aachen, North Rhine-Westphalia, 52074, Germany

  • Universitätsklinik Jena - Klinik für Innere Medizin II

    Jena, 07705, Germany

  • Universitätsklinikum Bonn Medizinische Klinik und Poliklinik III

    Bonn, North Rhine-Westphalia, 53105, Germany

  • Universitätsklinikum Dresden Medizinische Klinik und Poliklinik I

    Dresden, Saxony, 01307, Germany

  • Universitätsklinikum Düsseldorf Klinik für Hämatologie, Onkologie und Klinische Immunologie

    Düsseldorf, North Rhine-Westphalia, 40225, Germany

  • Universitätsklinikum Essen Klinik für Hämatologie

    Essen, North Rhine-Westphalia, 45122, Germany

  • Universitätsklinikum Freiburg - Klinik für Innere Medizin I

    Freiburg im Breisgau, 79106, Germany

  • Universitätsklinikum Halle (Saale)

    Halle, Saxony-Anhalt, 06120, Germany

  • Universitätsklinikum Hamburg Eppendorf Klinik und Poliklinik für Onkologie, Hämatologie und KMT mit Sektion Pneumologie

    Hamburg, Free and Hanseatic City of Hamburg, 20246, Germany

  • Universitätsklinikum Magdeburg

    Magdeburg, Sachesen-Anhalt, 39120, Germany

  • Universitätsklinikum Ulm Klinik für Innere Medizin III

    Ulm, Baden-Wurttemberg, 89081, Germany

  • Universitätsmedizin Mainz III. Medizinische Klinik und Poliklinik

    Mainz, Hesse, 55131, Germany

  • Universitätsmedizin Mannheim III. Medizinische Klinik Hämatologie und Internistische Onkologie

    Mannheim, Baden-Wurttemberg, 68167, Germany

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