TriangleNCT03011372 · CITED IN SOURCE DOCUMENTS
A Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement - (FIGHT-203)
An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.
- Phase
- PHASE2
- Status at capture
- COMPLETED
- Registry last update
- 2025-11-18
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.
Open the original ClinicalTrials.gov record → · Download preserved record
What did the study report?
Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.
Participant flow
Baseline characteristics
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
Percentage of Participants Who Achieved CR as Determined by Central Review Committee (CRC) Assessment
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
Percentage of Participants Who Achieved a Best Overall Response of CR or PR as Determined by CRC Assessment
Percentage of Participants Who Achieved a Complete Cytogenetic Response (CCyR) as Assessed by Local Analysis and Investigator Evaluation
Percentage of Participants Who Achieved a CCyR as Assessed by CRC Assessment
Percentage of Participants Who Achieved a Partial Cytogenetic Response (PCyR) as Assessed by Local Analysis and Investigator Evaluation
Percentage of Participants Who Achieved a PCyR as Assessed by CRC Assessment
Duration of Complete Response
Duration of Response
Progression-free Survival (PFS)
Overall Survival
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
Number of Participants With Any ≥Grade 3 TEAE
Adverse events
Limitations, agreements, and source contact
Who could take part
- eligibility Criteria
- 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.
- healthy Volunteers
- false
- minimum Age
- 18 Years
- sex
- ALL
- std Ages
- ADULT
- OLDER_ADULT
Treatment arms and interventions
- arm Groups
- intervention Names
- Drug: Pemigatinib
- label
- Pemigatinib
- type
- EXPERIMENTAL
- interventions
- arm Group Labels
- Pemigatinib
- description
- 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.
- name
- Pemigatinib
- other Names
- INCB054828
- type
- DRUG
Study design
- allocation
- NA
- intervention Model
- SINGLE_GROUP
- masking Info
- masking
- NONE
- primary Purpose
- TREATMENT
Enrollment
- count
- 47
- type
- ACTUAL
Registered outcome plans (not posted results)
- other Outcomes
- description
- 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.
- measure
- Percentage of Participants Who Achieved CR as Determined by Central Review Committee (CRC) Assessment
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- description
- In addition, responses were assessed by CRC based on MLN IWG response criteria for myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions.
- measure
- Percentage of Participants Who Achieved a Best Overall Response of CR or PR as Determined by CRC Assessment
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- description
- In addition, responses were assessed by CRC based on MLN IWG response criteria for myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions.
- measure
- Percentage of Participants Who Achieved a CCyR as Assessed by CRC Assessment
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- primary Outcomes
- description
- 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).
- measure
- 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
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- secondary Outcomes
- description
- 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.
- measure
- 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
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- description
- 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.
- measure
- Percentage of Participants Who Achieved a Complete Cytogenetic Response (CCyR) as Assessed by Local Analysis and Investigator Evaluation
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- description
- 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.
- measure
- Percentage of Participants Who Achieved a Partial Cytogenetic Response (PCyR) as Assessed by Local Analysis and Investigator Evaluation
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- description
- In addition, responses were assessed by CRC based on MLN IWG response criteria for myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions.
- measure
- Percentage of Participants Who Achieved a PCyR as Assessed by CRC Assessment
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- description
- 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
- measure
- Duration of Complete Response
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- description
- 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.
- measure
- Duration of Response
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- description
- 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).
- measure
- Progression-free Survival (PFS)
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- description
- Overall survival was defined as as the time from the first day of taking study drug until death due to any cause
- measure
- Overall Survival
- time Frame
- up to 2513 days (120 21-day treatment cycles)
- description
- 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.
- measure
- Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
- time Frame
- up to 2543 days
- description
- 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.
- measure
- Number of Participants With Any ≥Grade 3 TEAE
- time Frame
- up to 2543 days
Full study description
- 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.
Source references
- references
- citation
- 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
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- type
- DERIVED
- citation
- 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
- type
- DERIVED
- citation
- 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.
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- type
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Discovery and provenance
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