New hope for myelofibrosis patients who stop responding to standard therapy

NCT ID NCT01523171

First seen Jul 16, 2026 · Last updated Jul 17, 2026 · Updated 1 time

Summary

This phase 2 trial tests an experimental drug called SAR302503 in people with myelofibrosis who have already been treated with ruxolitinib. The main goal is to see if the drug can shrink the spleen by at least 35% after six months of daily oral capsules. The study also tracks whether symptoms like night sweats and belly discomfort improve. Participants have intermediate- or high-risk myelofibrosis, including forms that started after polycythemia vera or essential thrombocythemia.

What this could mean

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

Active substance
an experimental drug called SAR302503
What this could lead to
If successful, this could offer a new treatment option for people with myelofibrosis whose disease no longer responds to ruxolitinib.
What could go wrong
This is a mid-stage trial with a small number of participants and no comparison group, so results may not confirm benefit. The drug may also have side effects.

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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

  • Investigational Site Number 040001

    Vienna, 1090, Austria

  • Investigational Site Number 040002

    Salzburg, 5020, Austria

  • Investigational Site Number 056002

    Antwerp, 2060, Belgium

  • Investigational Site Number 056003

    Leuven, 3000, Belgium

  • Investigational Site Number 124001

    Toronto, M5G 2M9, Canada

  • Investigational Site Number 250001

    Marseille, 13273, France

  • Investigational Site Number 250002

    Paris, 75475, France

  • Investigational Site Number 250003

    Nîmes, 30029, France

  • Investigational Site Number 250004

    Toulouse, 31000, France

  • Investigational Site Number 250006

    Paris, 75571, France

  • Investigational Site Number 276001

    Mannheim, 68167, Germany

  • Investigational Site Number 276003

    Frankfurt am Main, 60590, Germany

  • Investigational Site Number 276005

    Ulm, 89081, Germany

  • Investigational Site Number 276006

    Magdeburg, 39120, Germany

  • Investigational Site Number 276007

    Leipzig, 04103, Germany

  • Investigational Site Number 380001

    Milan, 20122, Italy

  • Investigational Site Number 380002

    Roma, 00161, Italy

  • Investigational Site Number 380003

    Varese, 21100, Italy

  • Investigational Site Number 380004

    Florence, 50134, Italy

  • Investigational Site Number 528001

    Nijmegen, 6525 GA, Netherlands

  • Investigational Site Number 528002

    Amsterdam, 1081 HV, Netherlands

  • Investigational Site Number 528003

    Maastricht, 6229 HX, Netherlands

  • Investigational Site Number 724001

    Barcelona, 08036, Spain

  • Investigational Site Number 724002

    Salamanca, 37007, Spain

  • Investigational Site Number 724003

    Majadahonda, 28222, Spain

  • Investigational Site Number 826001

    London, SE1 9RT, United Kingdom

  • Investigational Site Number 840001

    Kansas City, Kansas, 66160-7321, United States

  • Investigational Site Number 840002

    Houston, Texas, 77030, United States

  • Investigational Site Number 840003

    San Francisco, California, 94143, United States

  • Investigational Site Number 840004

    San Francisco, California, 94143, United States

  • Investigational Site Number 840005

    Atlanta, Georgia, 30322, United States

  • Investigational Site Number 840007

    Phoenix, Arizona, 85054, United States

  • Investigational Site Number 840009

    New York, New York, 10021, United States

  • Investigational Site Number 840010

    Ann Arbor, Michigan, 48109-0759, United States

  • Investigational Site Number 840013

    Baltimore, Maryland, 21229, United States

  • Investigational Site Number 840014

    Chicago, Illinois, 60637, United States

  • Investigational Site Number 840015

    Salt Lake City, Utah, 84112-5550, United States

  • Investigational Site Number 840017

    Baltimore, Maryland, 21201, United States

  • Investigational Site Number 840018

    New York, New York, 10032, United States

  • Investigational Site Number 840019

    Middletown, Ohio, 45042, United States

  • Investigational Site Number 840022

    Cleveland, Ohio, 44195, United States

  • Investigational Site Number 840024

    Charleston, South Carolina, 29406, United States

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