A Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement - (FIGHT-203)

A Phase 2, Open-Label, Monotherapy, Multicenter Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement - (FIGHT-203)

The purpose of this study is to evaluate the efficacy and safety of pemigatinib (INCB054828) in subjects with myeloid/lymphoid neoplasms with fibroblast growth factor receptor (FGFR) 1 rearrangement.

Study Overview

• Status: Completed
• Conditions: MPN (Myeloproliferative Neoplasms)
• Intervention / Treatment: Drug: Pemigatinib

• Study Type: Interventional
• Enrollment (Actual): 47
• Phase: Phase 2

Expanded Access

No longer available outside the clinical trial. See expanded access record.

Contacts and Locations

Study Locations

• Austria
  • Linz, Austria, 04010
    • Ordensklinikum Krankenhaus Der Barmherzigen Schwestern Linz
  • Vienna, Austria, 01090
    • Medical University of Vienna
  • Vienna, Austria, 01140
    • Iii Med. Abteilung For Hematologie and Onkologie Hanuscfhkrankenhaus
• Belgium
  • Leuven, Belgium, 03000
    • Universitair Ziekenhuis (Uz) Leuven
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 2M9
      • Princess Margaret Cancer Center
• France
  • Lyon, France, 69373
    • Centre Leon Berard
  • Nice, France, 06202
    • Chu de Nice - Hospital L Archet
  • Paris, France, 75475
    • Hospital Saint Louis
  • Toulouse, France, 31059
    • Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole
• Germany
  • Aachen, Germany, D-52074
    • University Medical Center Rwth Aachen
  • Halle, Germany, 06120
    • Universitätsklinikum Halle (Saale)
  • Jena, Germany, 07740
    • Universitätsklinikum Jena
  • Leipzig, Germany, 04103
    • Universitätsklinikum Leipzig
  • Mannheim, Germany, 68167
    • University Hospital Mannheim
  • Minden, Germany, 32429
    • Johannes Wesling Klinikum Minden
• Italy
  • Bergamo, Italy, 24127
    • Ospedale Papa Giovanni XXIII
  • Florence, Italy, 50134
    • Azienda Ospedaliero-Universitaria Careggi (AOUC)
• Japan
  • Osaka, Japan, 589-8511
    • Kindai University Hospital
  • Tokyo, Japan, 141-8625
    • NTT Medical Center Tokyo
• Spain
  • Valencia, Spain, 46010
    • Hospital Clinico Universitario de Valencia
• Switzerland
  • Bern, Switzerland, 03010
    • Inselspital - Universitaetsspital Bern
  • Zurich, Switzerland, 08091
    • UniversitätsSpital Zürich
• United Kingdom
  • London, United Kingdom, SE1 9RT
    • Guys and St Thomas NHS Foundation Trust
  • Oxford, United Kingdom, OX3 7LE
    • Oxford University Hospitals NHS Foundation Trust
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85054
      • Mayo Clinic Arizona
  • California
    • Duarte, California, United States, 91010
      • City of Hope National Medical Center
    • Stanford, California, United States, 94305
      • Stanford Cancer Institute
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University - Winship Cancer Institute
  • Indiana
    • Indianapolis, Indiana, United States, 46237
      • Franciscan St. Francis Health
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • New York
    • New York, New York, United States, 10021
      • Weill Cornell Medical Centers
  • Texas
    • Houston, Texas, United States, 77030
      • Md Anderson Cancer Center
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pemigatinib	Drug: Pemigatinib; Pemigatinib once a day by mouth for 2 consecutive weeks and 1 week off therapy. Participants will receive either the intermittent dose (as written) or continuous dosing.; Other Names: INCB054828

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Who Achieved Complete Response (CR) as Determined by Investigator Assessment According to the Response Criteria for Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement	CR was defined as the presence of all of the following improvements: (1) bone marrow: ≤5% myeloblasts (including monocytic blast equivalent) and no lymphoblasts, with normal maturation of all cell lines, and return to age-adjusted normal cellularity; (2) osteomyelofibrosis absent or equal to "mild reticulin fibrosis" (Grade 1 or less fibrosis); (3) peripheral blood: white blood cells (WBC) ≤10 x 10^9 cells/Liter (L); hemoglobin (Hgb) ≥11 grams per deciliter (g/dL); platelets ≥100 x 10^9/L and ≤450 x 10^9/L; neutrophils ≥1.0 x 10^9/L; blasts = 0%; neutrophil precursors reduced to ≤2%; monocytes ≤1 x 10^9/L; eosinophils ≤0.5 x 10^9/L; (4) extramedullary disease: complete resolution of extramedullary disease present before therapy (e.g., lymphadenopathy), including palpable hepatosplenomegaly. Persistent low-level dysplasia was permitted given subjectivity of assignment of dysplasia. Response criteria by investigator assessment were the same for chronic phase (CP) and blast phase (BP).	up to 2513 days (120 21-day treatment cycles)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Who Achieved a Best Overall Response of Complete Response (CR) or Partial Response (PR) as Determined by Investigator Assessment According to the Response Criteria for Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement	CR=all of the following improvements: (1) bone marrow: ≤5% myeloblasts (including monocytic blast equivalent) and no lymphoblasts, with normal maturation of all cell lines, and return to age-adjusted normal cellularity; (2) osteomyelofibrosis absent/equal to "mild reticulin fibrosis"; (3) WBC ≤10 x 10^9 cells/L; Hgb ≥11 g/dL; platelets ≥100 x 10^9/L, ≤450 x 10^9/L; neutrophils ≥1.0 x 10^9/L; blasts=0%; neutrophil precursors reduced to ≤2%; monocytes ≤1 x 10^9/L; eosinophils ≤0.5 x 10^9/L; (4) extramedullary disease: complete resolution of extramedullary disease present pre-therapy, including palpable hepatosplenomegaly. Persistent low-level dysplasia was permitted. PR=all of the following improvements: (1) reduction of bone marrow blasts/blast equivalents by 50%, but remaining >5% of cellularity (except in cases with ≤5% bone marrow blasts at baseline); (2) normalization of peripheral blood indices per CR Criterion 3; (3) extra medullary disease response of CMR/CR or PMR/PR.	up to 2513 days (120 21-day treatment cycles)
Percentage of Participants Who Achieved a Complete Cytogenetic Response (CCyR) as Assessed by Local Analysis and Investigator Evaluation	CCyR was defined as 0% 8p11 translocated metaphases as seen on classic karyotyping with minimal of 20 metaphases, or fluorescence in situ hybridization (FISH). Loss of cytogenetic burden of disease (via FISH or classic karyotyping) was required to reach CCyR.	up to 2513 days (120 21-day treatment cycles)
Percentage of Participants Who Achieved a Partial Cytogenetic Response (PCyR) as Assessed by Local Analysis and Investigator Evaluation	PCyR was defined as the decrease from baseline of 50% or more 8p11 translocated metaphases as seen on classic karyotyping with minimal of 20 metaphases, or FISH.	up to 2513 days (120 21-day treatment cycles)
Percentage of Participants Who Achieved a PCyR as Assessed by CRC Assessment	In addition, responses were assessed by CRC based on MLN IWG response criteria for myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions.	up to 2513 days (120 21-day treatment cycles)
Duration of Complete Response	Duration of complete response was defined as the time from the first assessment of complete response to the earlier of the date of first worsening assessment after complete response or death due to any cause	up to 2513 days (120 21-day treatment cycles)
Duration of Response	Duration of response was defined as the time from the first assessment of complete response or partial response to the earlier of the date of first worsening assessment after response or death due to any cause.	up to 2513 days (120 21-day treatment cycles)
Progression-free Survival (PFS)	PFS was defined as the time from the first date of taking study drug until the date of disease progression or until death due to any cause, whichever was earlier. Disease progression was defined as the combination of 2 major criteria, 1 major and 2 minor criteria, or 3 minor criteria from the following lists. Major criteria: (1) increase in blast count; (2) evidence of cytogenetic evolution (re-appearance of a previously present or appearance of a new cytogenetic abnormality, or increase in cytogenetic burden of disease); (3) new or worsening extramedullary disease (worsening splenomegaly or extramedullary disease outside of the spleen). Minor criteria: (1) transfusion dependence; (2) significant loss of maximal response on cytopenias ≥50% decrement from maximum remission/response in granulocytes or platelets; (3) reduction in Hgb by ≥1.5g/dL from best response or from baseline as noted on complete blood count; (4) evidence of clonal evolution (molecular).	up to 2513 days (120 21-day treatment cycles)
Overall Survival	Overall survival was defined as as the time from the first day of taking study drug until death due to any cause	up to 2513 days (120 21-day treatment cycles)
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)	An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurs after a participant provides informed consent. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug.	up to 2543 days
Number of Participants With Any ≥Grade 3 TEAE	An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurs after a participant provides informed consent. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug. The severity of AEs was assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 Grades 1 through 4. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2: moderate; minimal, local, or noninvasive intervention indicated; limiting age-appropriate activities of daily living. Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent intervention indicated.	up to 2543 days

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Who Achieved CR as Determined by Central Review Committee (CRC) Assessment	In addition, responses were assessed by CRC based on Myeloid/Lymphoid Neoplasm International Working Group (MLN IWG) response criteria for myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions.	up to 2513 days (120 21-day treatment cycles)
Percentage of Participants Who Achieved a Best Overall Response of CR or PR as Determined by CRC Assessment	In addition, responses were assessed by CRC based on MLN IWG response criteria for myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions.	up to 2513 days (120 21-day treatment cycles)
Percentage of Participants Who Achieved a CCyR as Assessed by CRC Assessment	In addition, responses were assessed by CRC based on MLN IWG response criteria for myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions.	up to 2513 days (120 21-day treatment cycles)

Collaborators and Investigators

• Sponsor: Incyte Corporation
• Investigators: Study Director: Philomena Collucci, MD, Incyte Corporation

Publications and helpful links

General Publications

• Subbiah V, Burris HA 3rd, Kurzrock R. Revolutionizing cancer drug development: Harnessing the potential of basket trials. Cancer. 2024 Jan;130(2):186-200. doi: 10.1002/cncr.35085. Epub 2023 Nov 7.
• Al-Bazaz M, Forstreuter A, Hammada I, Hille J, Wagner JN, Reinert J, Wehrhahn J, Bokemeyer C, Fiedler W. Acute Lymphoblastic Leukemia Characterized by Rare BCR::FGFR1 Translocation: A Case Report With Literature Review. Case Rep Hematol. 2025 Oct 23;2025:8892036. doi: 10.1155/crh/8892036. eCollection 2025.
• Verstovsek S, Kiladjian JJ, Vannucchi AM, Patel JL, Rambaldi A, Shomali WE, Oh ST, Usuki K, Harrison CN, Ritchie EK, Akard LP, Hernandez-Boluda JC, Huguet F, Colucci P, Zhen H, Oliveira N, Gilmartin A, Langford C, George TI, Reiter A, Gotlib J. Pemigatinib for Myeloid/Lymphoid Neoplasms with FGFR1 Rearrangement. NEJM Evid. 2025 Sep;4(9):EVIDoa2500017. doi: 10.1056/EVIDoa2500017. Epub 2025 Aug 26.

Study record dates

Study Major Dates

• Study Start (Actual): April 25, 2017
• Primary Completion (Actual): October 30, 2024
• Study Completion (Actual): October 30, 2024

Study Registration Dates

• First Submitted: January 4, 2017
• First Submitted That Met QC Criteria: January 4, 2017
• First Posted (Estimated): January 5, 2017

Study Record Updates

• Last Update Posted (Estimated): November 18, 2025
• Last Update Submitted That Met QC Criteria: November 4, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Keywords: eosinophilia; Myeloid neoplasm; Lymphoid neoplasm; fibroblast growth factor receptor inhibitor; FGFR1 rearrangement; 8p11; eosinophilic syndrome
• Additional Relevant MeSH Terms: Leukocyte Disorders; Hematologic Diseases; Bone Marrow Diseases; Hemic and Lymphatic Diseases; Myeloproliferative Disorders; Eosinophilia; pemigatinib
• Other Study ID Numbers: INCB 54828-203; 2016-002596-10 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency

• IPD Sharing Time Frame: Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
• IPD Sharing Access Criteria: Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No