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
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Investigational Site Number 036001
Box Hill, 3128, Australia
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Investigational Site Number 036002
Wodonga, 3690, Australia
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Investigational Site Number 036003
Randwick, 2031, Australia
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Investigational Site Number 036004
Tweed Heads, 2485, Australia
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Investigational Site Number 036005
Herston, 4029, Australia
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Investigational Site Number 040001
Vienna, 1090, Austria
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Investigational Site Number 056001
Leuven, 3000, Belgium
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Investigational Site Number 056003
Antwerp, 2060, Belgium
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Investigational Site Number 076001
Rio de Janeiro, 20230-130, Brazil
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Investigational Site Number 076002
Jaú, 17210-120, Brazil
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Investigational Site Number 076004
Porto Alegre, 90110-270, Brazil
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Investigational Site Number 124001
Montreal, H1T 2M4, Canada
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Investigational Site Number 124002
Saint John, E2L 4L2, Canada
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Investigational Site Number 124003
Montreal, H2W 1S6, Canada
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Investigational Site Number 158001
Taipei, 112, Taiwan
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Investigational Site Number 158002
Changhua, 500, Taiwan
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Investigational Site Number 158003
Kaohsiung City, 833, Taiwan
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Investigational Site Number 250001
Villejuif, 94805, France
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Investigational Site Number 250002
Pierre-Bénite, 69495, France
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Investigational Site Number 250003
Toulouse, 31000, France
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Investigational Site Number 250004
Nîmes, 30029, France
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Investigational Site Number 250005
Nantes, 44093, France
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Investigational Site Number 250006
Marseille, 13273, France
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Investigational Site Number 250007
Poitiers, 86000, France
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Investigational Site Number 276001
Mannheim, 68167, Germany
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Investigational Site Number 276006
Aachen, 52074, Germany
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Investigational Site Number 276007
Bonn, 53127, Germany
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Investigational Site Number 276008
Dresden, 01307, Germany
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Investigational Site Number 348001
Debrecen, 4032, Hungary
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Investigational Site Number 348002
Budapest, 1097, Hungary
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Investigational Site Number 348003
Miskolc, 3529, Hungary
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Investigational Site Number 348006
Kecskemét, 6000, Hungary
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Investigational Site Number 348007
Győr, 9023, Hungary
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Investigational Site Number 372001
Galway, Ireland
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Investigational Site Number 372002
Dublin, DUBLIN 8, Ireland
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Investigational Site Number 376002
Tel Litwinsky, 52621, Israel
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Investigational Site Number 376003
Haifa, 31048, Israel
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Investigational Site Number 380001
Pavia, 27100, Italy
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Investigational Site Number 380002
Bergamo, 24127, Italy
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Investigational Site Number 380003
Varese, 21100, Italy
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Investigational Site Number 380004
Florence, 50134, Italy
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Investigational Site Number 380006
Pavia, 27100, Italy
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Investigational Site Number 380007
Bologna, 40138, Italy
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Investigational Site Number 410001
Seoul, 135-710, South Korea
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Investigational Site Number 410002
Bundang-Gu, 463-707, South Korea
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Investigational Site Number 410003
Seoul, 120-752, South Korea
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Investigational Site Number 410004
Seoul, 110-744, South Korea
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Investigational Site Number 410005
Seoul, 137-701, South Korea
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Investigational Site Number 410006
Seoul, 138-878, South Korea
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Investigational Site Number 410007
Seoul, South Korea
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Investigational Site Number 440001
Kaunas, LT-50009, Lithuania
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Investigational Site Number 440002
Klaipėda, LT-92288, Lithuania
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Investigational Site Number 484001
Querétaro, 76000, Mexico
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Investigational Site Number 616002
Gdansk, 80-952, Poland
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Investigational Site Number 616003
Wroclaw, 50-367, Poland
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Investigational Site Number 616005
Brzozów, 36-200, Poland
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Investigational Site Number 616006
Lodz, 93-510, Poland
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Investigational Site Number 616010
Warsaw, 02-106, Poland
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Investigational Site Number 620001
Lisbon, 1649-035, Portugal
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Investigational Site Number 620003
Porto, 4200-072, Portugal
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Investigational Site Number 620004
Lisbon, 1169-050, Portugal
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Investigational Site Number 620005
Coimbra, 3000-075, Portugal
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Investigational Site Number 642001
Timișoara, Romania
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Investigational Site Number 642002
Bucharest, 030171, Romania
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Investigational Site Number 642003
Brasov, Romania
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Investigational Site Number 642004
Bucharest, 022328, Romania
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Investigational Site Number 642006
Bucharest, Romania
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Investigational Site Number 643001
Moscow, 125284, Russia
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Investigational Site Number 643004
Saint Petersburg, 197341, Russia
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Investigational Site Number 643005
Saint Petersburg, 191024, Russia
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Investigational Site Number 643007
Volgograd, 400138, Russia
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Investigational Site Number 643008
Petrozavodsk, 185019, Russia
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Investigational Site Number 643009
Moscow, 125167, Russia
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Investigational Site Number 643010
Nizhny Novgorod, 603126, Russia
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Investigational Site Number 702001
Singapore, 169608, Singapore
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Investigational Site Number 702002
Singapore, 119228, Singapore
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Investigational Site Number 710002
Parktown, 2193, South Africa
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Investigational Site Number 710003
Johannesburg, 2013, South Africa
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Investigational Site Number 724001
Barcelona, 08036, Spain
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Investigational Site Number 724002
Madrid, 28046, Spain
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Investigational Site Number 752001
Stockholm, 14186, Sweden
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Investigational Site Number 752002
Uddevalla, 451 80, Sweden
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Investigational Site Number 826001
London, SE1 9RT, United Kingdom
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Investigational Site Number 826002
Glasgow, G12 0YN, United Kingdom
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Investigational Site Number 826003
Birmingham, B9 5SS, United Kingdom
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Investigational Site Number 826004
Leeds, LS9 7TF, United Kingdom
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Investigational Site Number 826005
London, W12 0HS, United Kingdom
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Investigational Site Number 826006
Belfast, BT9 7AB, United Kingdom
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Investigational Site Number 826007
Manchester, M20 4BX, United Kingdom
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Investigational Site Number 826008
Newcastle upon Tyne, NE7 7DN, United Kingdom
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Investigational Site Number 826009
Oxford, OX3 7LJ, United Kingdom
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Investigational Site Number 826010
Southampton, SO16 6YD, United Kingdom
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Investigational Site Number 840001
La Jolla, California, 92093, United States
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Investigational Site Number 840002
Canton, Ohio, 44718, United States
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Investigational Site Number 840004
Houston, Texas, 77030, United States
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Investigational Site Number 840006
Los Angeles, California, 90033, United States
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Investigational Site Number 840008
Rochester, Minnesota, 55905, United States
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Investigational Site Number 840009
Newark, New Jersey, 07112, United States
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Investigational Site Number 840012
La Jolla, California, 92093, United States
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Investigational Site Number 840013
Baton Rouge, Louisiana, 70808, United States
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Investigational Site Number 840014
Scottsdale, Arizona, 85259-5499, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a Dual-Action pill tame bone marrow tumors?
- Can a new pill shrink the spleen in myelofibrosis?
- Could a platelet-boosting drug shorten recovery after stem cell transplants?
- Can a modified interferon rein in early myelofibrosis?
- Can a drug free myelofibrosis patients from frequent blood transfusions?
- New hope for myelofibrosis patients who stop responding to standard therapy