New pill shows promise for rare blood cancer in early trial
NCT ID NCT03011372
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 trial tested a daily pill called pemigatinib in 47 people with a rare type of blood cancer caused by a specific genetic change (FGFR1 rearrangement). The drug aims to shrink or eliminate cancer cells by blocking a faulty protein. Participants took the drug in cycles, and researchers measured how many achieved a complete response (no signs of cancer). The study is completed, but results are not yet widely available.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Pemigatinib (a targeted cancer pill)
- What this could lead to
- If successful, this could provide a new treatment option for people with rare blood cancers driven by FGFR1 rearrangements, especially those who have relapsed or cannot have a stem cell transplant.
- What could go wrong
- This is a small, early-phase study (47 participants) without a comparison group. Results may not apply to all patients, and side effects or lack of lasting benefit are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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Locations
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Azienda Ospedaliero-Universitaria Careggi (Aouc)
Florence, 50134, Italy
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Centre Leon Berard
Lyon, 69373, France
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Chu de Nice - Hospital L Archet
Nice, 06202, France
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City of Hope National Medical Center
Duarte, California, 91010, United States
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Emory University - Winship Cancer Institute
Atlanta, Georgia, 30322, United States
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Franciscan St. Francis Health
Indianapolis, Indiana, 46237, United States
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Guys and St Thomas Nhs Foundation Trust
London, SE1 9RT, United Kingdom
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Hospital Clinico Universitario de Valencia
Valencia, 46010, Spain
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Hospital Saint Louis
Paris, 75475, France
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Iii Med. Abteilung For Hematologie and Onkologie Hanuscfhkrankenhaus
Vienna, 01140, Austria
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Inselspital - Universitaetsspital Bern
Bern, 03010, Switzerland
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Johannes Wesling Klinikum Minden
Minden, 32429, Germany
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Kindai University Hospital
Osaka, 589-8511, Japan
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Mayo Clinic Arizona
Phoenix, Arizona, 85054, United States
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Md Anderson Cancer Center
Houston, Texas, 77030, United States
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Medical University of Vienna
Vienna, 01090, Austria
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Ntt Medical Center Tokyo
Tokyo, 141-8625, Japan
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Ordensklinikum Krankenhaus Der Barmherzigen Schwestern Linz
Linz, 04010, Austria
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Ospedale Papa Giovanni Xxiii
Bergamo, 24127, Italy
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Oxford University Hospitals Nhs Foundation Trust
Oxford, OX3 7LE, United Kingdom
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Princess Margaret Cancer Center
Toronto, Ontario, M5G 2M9, Canada
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Stanford Cancer Institute
Stanford, California, 94305, United States
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Universitair Ziekenhuis (Uz) Leuven
Leuven, 03000, Belgium
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Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole
Toulouse, 31059, France
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Universitatsklinikum Halle (Saale)
Halle, 06120, Germany
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Universitatsklinikum Jena
Jena, 07740, Germany
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Universitatsklinikum Leipzig
Leipzig, 04103, Germany
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Universitatsspital Zurich
Zurich, 08091, Switzerland
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University Hospital Mannheim
Mannheim, 68167, Germany
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University Medical Center Rwth Aachen
Aachen, D-52074, Germany
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University of Utah
Salt Lake City, Utah, 84112, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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Weill Cornell Medical Centers
New York, New York, 10021, United States
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