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

NCT03011372 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: INCB 54828-2032016-002596-10
• Study Type: interventional
• Target: 47
• Locations: 11 countries
• Sites: 33

Brief Summary

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.

Conditions

MPN (Myeloproliferative Neoplasms)

Keywords

Myeloid neoplasm; fibroblast growth factor receptor inhibitor; FGFR1 rearrangement; 8p11; eosinophilia; eosinophilic syndrome; Lymphoid neoplasm

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (10)

• 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

  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

  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

  up to 2513 days (120 21-day treatment cycles)

• Percentage of Participants Who Achieved a PCyR as Assessed by CRC Assessment

  up to 2513 days (120 21-day treatment cycles)

• Duration of Complete Response

  up to 2513 days (120 21-day treatment cycles)

Other Outcomes (3)

• Percentage of Participants Who Achieved CR as Determined by Central Review Committee (CRC) Assessment

  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

  up to 2513 days (120 21-day treatment cycles)

• Percentage of Participants Who Achieved a CCyR as Assessed by CRC Assessment

  up to 2513 days (120 21-day treatment cycles)

Study Arms (1)

Pemigatinib

EXPERIMENTAL

Drug: Pemigatinib

Interventions

Pemigatinib DRUG

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.

Also known as: INCB054828

Pemigatinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (33)

Mayo Clinic Arizona

Phoenix, Arizona, 85054, United States

Location

City of Hope National Medical Center

Duarte, California, 91010, United States

Location

Stanford Cancer Institute

Stanford, California, 94305, United States

Location

Emory University - Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

Franciscan St. Francis Health

Indianapolis, Indiana, 46237, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Weill Cornell Medical Centers

New York, New York, 10021, United States

Location

Md Anderson Cancer Center

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84112, United States

Location

Ordensklinikum Krankenhaus Der Barmherzigen Schwestern Linz

Linz, 04010, Austria

Location

Medical University of Vienna

Vienna, 01090, Austria

Location

Iii Med. Abteilung For Hematologie and Onkologie Hanuscfhkrankenhaus

Vienna, 01140, Austria

Location

Universitair Ziekenhuis (Uz) Leuven

Leuven, 03000, Belgium

Location

Princess Margaret Cancer Center

Toronto, Ontario, M5G 2M9, Canada

Location

Centre Leon Berard

Lyon, 69373, France

Location

Chu de Nice - Hospital L Archet

Nice, 06202, France

Location

Hospital Saint Louis

Paris, 75475, France

Location

Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole

Toulouse, 31059, France

Location

University Medical Center Rwth Aachen

Aachen, D-52074, Germany

Location

Universitatsklinikum Halle (Saale)

Halle, 06120, Germany

Location

Universitatsklinikum Jena

Jena, 07740, Germany

Location

Universitatsklinikum Leipzig

Leipzig, 04103, Germany

Location

University Hospital Mannheim

Mannheim, 68167, Germany

Location

Johannes Wesling Klinikum Minden

Minden, 32429, Germany

Location

Ospedale Papa Giovanni Xxiii

Bergamo, 24127, Italy

Location

Azienda Ospedaliero-Universitaria Careggi (Aouc)

Florence, 50134, Italy

Location

Kindai University Hospital

Osaka, 589-8511, Japan

Location

Ntt Medical Center Tokyo

Tokyo, 141-8625, Japan

Location

Hospital Clinico Universitario de Valencia

Valencia, 46010, Spain

Location

Inselspital - Universitaetsspital Bern

Bern, 03010, Switzerland

Location

Universitatsspital Zurich

Zurich, 08091, Switzerland

Location

Guys and St Thomas Nhs Foundation Trust

London, SE1 9RT, United Kingdom

Location

Oxford University Hospitals Nhs Foundation Trust

Oxford, OX3 7LE, United Kingdom

Location

Related Publications (3)

• 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.

  PMID: 41180818 DERIVED

• 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.

  PMID: 40856555 DERIVED

• 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.

  PMID: 37934000 DERIVED

MeSH Terms

Conditions

Myeloproliferative Disorders; Eosinophilia

Interventions

pemigatinib

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukocyte Disorders

Results Point of Contact

• Title: Study Director
• Organization: Incyte Corporation

medinfo@incyte.com

1-855-463-3463

Study Officials

• Philomena Collucci, MD

  Incyte Corporation

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: January 4, 2017
• First Posted: January 5, 2017
• Study Start: April 25, 2017
• Primary Completion: October 30, 2024
• Study Completion: October 30, 2024
• Last Updated: November 18, 2025
• Results First Posted: November 18, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
• 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.

Locations

CA (1); IT (2); JP (2); ES (1); GB (2); FR (4); AU (3); BE (1); US (9); DE (6); CH (2)