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New pill shows promise for rare blood cancer in early trial

NCT ID NCT03011372

Disease control Sponsor: Incyte Corporation Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

47 people

The number who actually took part.

Started

Apr 2017

Finished

Oct 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Documented lymphoid or myeloid neoplasm with 8p11 rearrangement known to lead to FGFR1 activation, based on standard diagnostic cytogenetic evaluation performed locally, before signing informed consent for this study. * Eligible subjects must: * Have relapsed after stem cell transplantation or after other disease modifying therapy, OR * Not be current candidates for stem cell transplantation or other disease modifying therapies. * Note: All relapsed/refractory subjects must have evidence of either cytogenetic or hematological disease and have no evidence of residual toxicity (eg, graft-versus-host disease requiring treatment). * Life expectancy ≥ 12 weeks. * Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2. Exclusion Criteria: * Prior receipt of a selective FGFR inhibitor. * History and/or current evidence of ectopic mineralization/calcification, including but not limited to soft tissue, kidneys, intestine, myocardia, or lung, except calcified lymph nodes and asymptomatic arterial or cartilage/tendon calcifications. * Current evidence of corneal disorder/keratopathy, including but not limited to bullous/band keratopathy, corneal abrasion, inflammation/ulceration, and keratoconjunctivitis, as confirmed by ophthalmologic examination. * Use of any potent cytochrome P450 3A4 inhibitors or inducers within 14 days or 5 half-lives (whichever is shorter) before the first dose of study drug.

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Conditions

The condition(s) this trial relates to.

Eosinophilia myeloid neoplasm associated with FGFR1 rearrangement Myeloproliferative Disorders myeloproliferative neoplasm

As listed by the trial registrant

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

MPN (myeloproliferative neoplasms)

Contacts and locations

Locations

  • Azienda Ospedaliero-Universitaria Careggi (Aouc)

    Florence, 50134, Italy

  • Centre Leon Berard

    Lyon, 69373, France

  • Chu de Nice - Hospital L Archet

    Nice, 06202, France

  • City of Hope National Medical Center

    Duarte, California, 91010, United States

  • Emory University - Winship Cancer Institute

    Atlanta, Georgia, 30322, United States

  • Franciscan St. Francis Health

    Indianapolis, Indiana, 46237, United States

  • Guys and St Thomas Nhs Foundation Trust

    London, SE1 9RT, United Kingdom

  • Hospital Clinico Universitario de Valencia

    Valencia, 46010, Spain

  • Hospital Saint Louis

    Paris, 75475, France

  • Iii Med. Abteilung For Hematologie and Onkologie Hanuscfhkrankenhaus

    Vienna, 01140, Austria

  • Inselspital - Universitaetsspital Bern

    Bern, 03010, Switzerland

  • Johannes Wesling Klinikum Minden

    Minden, 32429, Germany

  • Kindai University Hospital

    Osaka, 589-8511, Japan

  • Mayo Clinic Arizona

    Phoenix, Arizona, 85054, United States

  • Md Anderson Cancer Center

    Houston, Texas, 77030, United States

  • Medical University of Vienna

    Vienna, 01090, Austria

  • Ntt Medical Center Tokyo

    Tokyo, 141-8625, Japan

  • Ordensklinikum Krankenhaus Der Barmherzigen Schwestern Linz

    Linz, 04010, Austria

  • Ospedale Papa Giovanni Xxiii

    Bergamo, 24127, Italy

  • Oxford University Hospitals Nhs Foundation Trust

    Oxford, OX3 7LE, United Kingdom

  • Princess Margaret Cancer Center

    Toronto, Ontario, M5G 2M9, Canada

  • Stanford Cancer Institute

    Stanford, California, 94305, United States

  • Universitair Ziekenhuis (Uz) Leuven

    Leuven, 03000, Belgium

  • Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole

    Toulouse, 31059, France

  • Universitatsklinikum Halle (Saale)

    Halle, 06120, Germany

  • Universitatsklinikum Jena

    Jena, 07740, Germany

  • Universitatsklinikum Leipzig

    Leipzig, 04103, Germany

  • Universitatsspital Zurich

    Zurich, 08091, Switzerland

  • University Hospital Mannheim

    Mannheim, 68167, Germany

  • University Medical Center Rwth Aachen

    Aachen, D-52074, Germany

  • University of Utah

    Salt Lake City, Utah, 84112, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

  • Weill Cornell Medical Centers

    New York, New York, 10021, United States

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