Can a daily pill shrink the spleen in myelofibrosis?

NCT ID NCT01437787

First seen Aug 11, 2026 · Last updated Aug 12, 2026 · Updated 1 time

Summary

This phase 3 trial is testing an experimental oral drug, SAR302503, in people with a rare bone marrow disorder called myelofibrosis who have an enlarged spleen. Participants are randomly assigned to receive one of two doses of the drug or a placebo. The main goal is to see if the drug can reduce spleen size by at least 35% after six months of treatment, which could help ease related symptoms like pain and discomfort.

What this could mean

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

Active substance
SAR302503, an experimental oral drug taken daily as a capsule
What this could lead to
If it works, this could offer a new treatment option to shrink the enlarged spleen and ease symptoms in people with myelofibrosis, potentially improving quality of life.
What could go wrong
This is an early-stage test in a specific group of patients, and the drug may not prove more effective than placebo. Side effects are possible, and results may not apply to all patients with myelofibrosis.

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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 036001

    Box Hill, 3128, Australia

  • Investigational Site Number 036002

    Wodonga, 3690, Australia

  • Investigational Site Number 036003

    Randwick, 2031, Australia

  • Investigational Site Number 036004

    Tweed Heads, 2485, Australia

  • Investigational Site Number 036005

    Herston, 4029, Australia

  • Investigational Site Number 040001

    Vienna, 1090, Austria

  • Investigational Site Number 056001

    Leuven, 3000, Belgium

  • Investigational Site Number 056003

    Antwerp, 2060, Belgium

  • Investigational Site Number 076001

    Rio de Janeiro, 20230-130, Brazil

  • Investigational Site Number 076002

    Jaú, 17210-120, Brazil

  • Investigational Site Number 076004

    Porto Alegre, 90110-270, Brazil

  • Investigational Site Number 124001

    Montreal, H1T 2M4, Canada

  • Investigational Site Number 124002

    Saint John, E2L 4L2, Canada

  • Investigational Site Number 124003

    Montreal, H2W 1S6, Canada

  • Investigational Site Number 158001

    Taipei, 112, Taiwan

  • Investigational Site Number 158002

    Changhua, 500, Taiwan

  • Investigational Site Number 158003

    Kaohsiung City, 833, Taiwan

  • Investigational Site Number 250001

    Villejuif, 94805, France

  • Investigational Site Number 250002

    Pierre-Bénite, 69495, France

  • Investigational Site Number 250003

    Toulouse, 31000, France

  • Investigational Site Number 250004

    Nîmes, 30029, France

  • Investigational Site Number 250005

    Nantes, 44093, France

  • Investigational Site Number 250006

    Marseille, 13273, France

  • Investigational Site Number 250007

    Poitiers, 86000, France

  • Investigational Site Number 276001

    Mannheim, 68167, Germany

  • Investigational Site Number 276006

    Aachen, 52074, Germany

  • Investigational Site Number 276007

    Bonn, 53127, Germany

  • Investigational Site Number 276008

    Dresden, 01307, Germany

  • Investigational Site Number 348001

    Debrecen, 4032, Hungary

  • Investigational Site Number 348002

    Budapest, 1097, Hungary

  • Investigational Site Number 348003

    Miskolc, 3529, Hungary

  • Investigational Site Number 348006

    Kecskemét, 6000, Hungary

  • Investigational Site Number 348007

    Győr, 9023, Hungary

  • Investigational Site Number 372001

    Galway, Ireland

  • Investigational Site Number 372002

    Dublin, DUBLIN 8, Ireland

  • Investigational Site Number 376002

    Tel Litwinsky, 52621, Israel

  • Investigational Site Number 376003

    Haifa, 31048, Israel

  • Investigational Site Number 380001

    Pavia, 27100, Italy

  • Investigational Site Number 380002

    Bergamo, 24127, Italy

  • Investigational Site Number 380003

    Varese, 21100, Italy

  • Investigational Site Number 380004

    Florence, 50134, Italy

  • Investigational Site Number 380006

    Pavia, 27100, Italy

  • Investigational Site Number 380007

    Bologna, 40138, Italy

  • Investigational Site Number 410001

    Seoul, 135-710, South Korea

  • Investigational Site Number 410002

    Bundang-Gu, 463-707, South Korea

  • Investigational Site Number 410003

    Seoul, 120-752, South Korea

  • Investigational Site Number 410004

    Seoul, 110-744, South Korea

  • Investigational Site Number 410005

    Seoul, 137-701, South Korea

  • Investigational Site Number 410006

    Seoul, 138-878, South Korea

  • Investigational Site Number 410007

    Seoul, South Korea

  • Investigational Site Number 440001

    Kaunas, LT-50009, Lithuania

  • Investigational Site Number 440002

    Klaipėda, LT-92288, Lithuania

  • Investigational Site Number 484001

    Querétaro, 76000, Mexico

  • Investigational Site Number 616002

    Gdansk, 80-952, Poland

  • Investigational Site Number 616003

    Wroclaw, 50-367, Poland

  • Investigational Site Number 616005

    Brzozów, 36-200, Poland

  • Investigational Site Number 616006

    Lodz, 93-510, Poland

  • Investigational Site Number 616010

    Warsaw, 02-106, Poland

  • Investigational Site Number 620001

    Lisbon, 1649-035, Portugal

  • Investigational Site Number 620003

    Porto, 4200-072, Portugal

  • Investigational Site Number 620004

    Lisbon, 1169-050, Portugal

  • Investigational Site Number 620005

    Coimbra, 3000-075, Portugal

  • Investigational Site Number 642001

    Timișoara, Romania

  • Investigational Site Number 642002

    Bucharest, 030171, Romania

  • Investigational Site Number 642003

    Brasov, Romania

  • Investigational Site Number 642004

    Bucharest, 022328, Romania

  • Investigational Site Number 642006

    Bucharest, Romania

  • Investigational Site Number 643001

    Moscow, 125284, Russia

  • Investigational Site Number 643004

    Saint Petersburg, 197341, Russia

  • Investigational Site Number 643005

    Saint Petersburg, 191024, Russia

  • Investigational Site Number 643007

    Volgograd, 400138, Russia

  • Investigational Site Number 643008

    Petrozavodsk, 185019, Russia

  • Investigational Site Number 643009

    Moscow, 125167, Russia

  • Investigational Site Number 643010

    Nizhny Novgorod, 603126, Russia

  • Investigational Site Number 702001

    Singapore, 169608, Singapore

  • Investigational Site Number 702002

    Singapore, 119228, Singapore

  • Investigational Site Number 710002

    Parktown, 2193, South Africa

  • Investigational Site Number 710003

    Johannesburg, 2013, South Africa

  • Investigational Site Number 724001

    Barcelona, 08036, Spain

  • Investigational Site Number 724002

    Madrid, 28046, Spain

  • Investigational Site Number 752001

    Stockholm, 14186, Sweden

  • Investigational Site Number 752002

    Uddevalla, 451 80, Sweden

  • Investigational Site Number 826001

    London, SE1 9RT, United Kingdom

  • Investigational Site Number 826002

    Glasgow, G12 0YN, United Kingdom

  • Investigational Site Number 826003

    Birmingham, B9 5SS, United Kingdom

  • Investigational Site Number 826004

    Leeds, LS9 7TF, United Kingdom

  • Investigational Site Number 826005

    London, W12 0HS, United Kingdom

  • Investigational Site Number 826006

    Belfast, BT9 7AB, United Kingdom

  • Investigational Site Number 826007

    Manchester, M20 4BX, United Kingdom

  • Investigational Site Number 826008

    Newcastle upon Tyne, NE7 7DN, United Kingdom

  • Investigational Site Number 826009

    Oxford, OX3 7LJ, United Kingdom

  • Investigational Site Number 826010

    Southampton, SO16 6YD, United Kingdom

  • Investigational Site Number 840001

    La Jolla, California, 92093, United States

  • Investigational Site Number 840002

    Canton, Ohio, 44718, United States

  • Investigational Site Number 840004

    Houston, Texas, 77030, United States

  • Investigational Site Number 840006

    Los Angeles, California, 90033, United States

  • Investigational Site Number 840008

    Rochester, Minnesota, 55905, United States

  • Investigational Site Number 840009

    Newark, New Jersey, 07112, United States

  • Investigational Site Number 840012

    La Jolla, California, 92093, United States

  • Investigational Site Number 840013

    Baton Rouge, Louisiana, 70808, United States

  • Investigational Site Number 840014

    Scottsdale, Arizona, 85259-5499, United States

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