<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03011372</url>
  </required_header>
  <id_info>
    <org_study_id>INCB 54828-203</org_study_id>
    <nct_id>NCT03011372</nct_id>
  </id_info>
  <brief_title>A Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement - (FIGHT-203)</brief_title>
  <official_title>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)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Incyte Corporation</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Incyte Corporation</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to evaluate the efficacy and safety of pemigatinib (INCB054828)&#xD;
      in subjects with myeloid/lymphoid neoplasms with fibroblast growth factor receptor (FGFR) 1&#xD;
      rearrangement.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">April 25, 2017</start_date>
  <completion_date type="Anticipated">July 31, 2024</completion_date>
  <primary_completion_date type="Anticipated">July 31, 2024</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>Yes</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>The proportion of participants who achieve Complete Response (CR) based on response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement</measure>
    <time_frame>: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>The proportion of subjects who achieve response, defined as a best response of CR or PR, as determined by investigator assessment according to the response criteria</measure>
    <time_frame>Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The proportion of subjects who achieve a complete cytogenetic response (CCyR) as assessed by local analysis and investigator evaluation</measure>
    <time_frame>Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The proportion of subjects who achieve a partial cytogenetic response (PCyR) as assessed by local analysis and investigator evaluation</measure>
    <time_frame>Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Duration of CR, defined as the time from first assessment of CR to the earlier of disease progression or death due to any cause</measure>
    <time_frame>Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Duration of response, defined as the time from first assessment of CR or PR to the earlier of disease progression or death due to any cause</measure>
    <time_frame>Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Progression-free survival (PFS)</measure>
    <time_frame>From the date of first study drug dose until the date of disease progression or until death due to any cause, whichever is earlier, assessed up to approximately 24 months.</time_frame>
    <description>PFS is defined as the time from the first date of taking study drug until the date of disease progression, as measured by response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement, or until death due to any cause, whichever is earlier.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Overall survival</measure>
    <time_frame>From date of first study drug dose until death due to any cause, assessed up to approximately 24 months.</time_frame>
    <description>Overall survival is defined as the time from the first day of taking study drug until death due to any cause. Subjects without death observed at the time of the analysis will be censored at last date known to be alive.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Safety and tolerability as assessed by frequency, duration, and severity of adverse events</measure>
    <time_frame>From baseline through 30-35 days after end of treatment, up to 7 months per individual subject</time_frame>
    <description>A treatment-emergent AE was defined as an event occurring after exposure to at least 1 dose of study drug. A treatment-related AE was defined as an event with a definite, probable, or possible causality to study medication. A serious AE is an event resulting in death, hospitalization, persistent or significant disability/incapacity, or is life threatening, a congenital anomaly/birth defect or requires medical or surgical intervention to prevent 1 of the outcomes above. The intensity of an AE was graded according to the National Cancer Institute common terminology criteria for adverse events (NCI-CTCAE) version 4.03: Grade 1 (Mild); Grade 2 (Moderate); Grade 3 (Severe); Grade 4 (life-threatening).</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">47</enrollment>
  <condition>MPN (Myeloproliferative Neoplasms)</condition>
  <arm_group>
    <arm_group_label>Pemigatinib</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Pemigatinib</intervention_name>
    <description>Pemigatinib once a day by mouth for 2 consecutive weeks and 1 week off therapy.&#xD;
Participants will receive either the intermittent dose (as written) or continuous dosing.</description>
    <arm_group_label>Pemigatinib</arm_group_label>
    <other_name>INCB054828</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Documented lymphoid or myeloid neoplasm with 8p11 rearrangement known to lead to FGFR1&#xD;
             activation, based on standard diagnostic cytogenetic evaluation performed locally,&#xD;
             before signing informed consent for this study.&#xD;
&#xD;
          -  Eligible subjects must:&#xD;
&#xD;
               -  Have relapsed after stem cell transplantation or after other disease modifying&#xD;
                  therapy, OR&#xD;
&#xD;
               -  Not be current candidates for stem cell transplantation or other disease&#xD;
                  modifying therapies.&#xD;
&#xD;
          -  Note: All relapsed/refractory subjects must have evidence of either cytogenetic or&#xD;
             hematological disease and have no evidence of residual toxicity (eg, graft-versus-host&#xD;
             disease requiring treatment).&#xD;
&#xD;
          -  Life expectancy ≥ 12 weeks.&#xD;
&#xD;
          -  Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Prior receipt of a selective FGFR inhibitor.&#xD;
&#xD;
          -  History and/or current evidence of ectopic mineralization/calcification, including but&#xD;
             not limited to soft tissue, kidneys, intestine, myocardia, or lung, except calcified&#xD;
             lymph nodes and asymptomatic arterial or cartilage/tendon calcifications.&#xD;
&#xD;
          -  Current evidence of corneal disorder/keratopathy, including but not limited to&#xD;
             bullous/band keratopathy, corneal abrasion, inflammation/ulceration, and&#xD;
             keratoconjunctivitis, as confirmed by ophthalmologic examination.&#xD;
&#xD;
          -  Use of any potent cytochrome P450 3A4 inhibitors or inducers within 14 days or 5&#xD;
             half-lives (whichever is shorter) before the first dose of study drug.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Philomena Collucci, MD</last_name>
    <role>Study Director</role>
    <affiliation>Incyte Corporation</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Mayo Clinic Arizona</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85054</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>City of Hope National Medical Center</name>
      <address>
        <city>Duarte</city>
        <state>California</state>
        <zip>91010</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Stanford Cancer Institute</name>
      <address>
        <city>Stanford</city>
        <state>California</state>
        <zip>94305</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Emory University - Winship Cancer Institute</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Franciscan St. Francis Health</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46237</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Washington University School of Medicine</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63110</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Weill Cornell Medical Centers</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10021</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Md Anderson Cancer Center</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Utah</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ordensklinikum Krankenhaus Der Barmherzigen Schwestern Linz</name>
      <address>
        <city>Linz</city>
        <zip>04010</zip>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Medical University of Vienna</name>
      <address>
        <city>Wien</city>
        <zip>01090</zip>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Iii Med. Abteilung For Hematologie and Onkologie Hanuscfhkrankenhaus</name>
      <address>
        <city>Wien</city>
        <zip>01140</zip>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitair Ziekenhuis (Uz) Leuven</name>
      <address>
        <city>Leuven</city>
        <zip>03000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Princess Margaret Cancer Center</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5G 2M9</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre Leon Berard</name>
      <address>
        <city>Lyon</city>
        <zip>69373</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Chu de Nice - Hospital L Archet</name>
      <address>
        <city>Nice Cedex 3</city>
        <zip>06202</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Saint Louis</name>
      <address>
        <city>Paris Cedex 10</city>
        <zip>75475</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole</name>
      <address>
        <city>Toulouse Cedex 9</city>
        <zip>31059</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Medical Center Rwth Aachen</name>
      <address>
        <city>Aachen</city>
        <zip>D-52074</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitatsklinikum Halle (Saale)</name>
      <address>
        <city>Halle</city>
        <zip>06120</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitatsklinikum Jena</name>
      <address>
        <city>Jena</city>
        <zip>07740</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitatsklinikum Leipzig</name>
      <address>
        <city>Leipzig</city>
        <zip>04103</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital Mannheim</name>
      <address>
        <city>Mannheim</city>
        <zip>68167</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Johannes Wesling Klinikum Minden</name>
      <address>
        <city>Minden</city>
        <zip>32429</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ospedale Papa Giovanni Xxiii</name>
      <address>
        <city>Bergamo</city>
        <zip>24127</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Azienda Ospedaliero-Universitaria Careggi (Aouc)</name>
      <address>
        <city>Florence</city>
        <zip>50134</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kindai University Hospital</name>
      <address>
        <city>Osaka</city>
        <zip>589-8511</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ntt Medical Center Tokyo</name>
      <address>
        <city>Tokyo</city>
        <zip>141-8625</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Clinico Universitario de Valencia</name>
      <address>
        <city>Valencia</city>
        <zip>46010</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Inselspital - Universitaetsspital Bern</name>
      <address>
        <city>Bern</city>
        <zip>03010</zip>
        <country>Switzerland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitatsspital Zurich</name>
      <address>
        <city>Zurich</city>
        <zip>08091</zip>
        <country>Switzerland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Guys and St Thomas Nhs Foundation Trust</name>
      <address>
        <city>London</city>
        <zip>SE1 9RT</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oxford University Hospitals Nhs Foundation Trust</name>
      <address>
        <city>Oxford</city>
        <zip>OX3 7LE</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Austria</country>
    <country>Belgium</country>
    <country>Canada</country>
    <country>France</country>
    <country>Germany</country>
    <country>Italy</country>
    <country>Japan</country>
    <country>Spain</country>
    <country>Switzerland</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>June 2023</verification_date>
  <study_first_submitted>January 4, 2017</study_first_submitted>
  <study_first_submitted_qc>January 4, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 5, 2017</study_first_posted>
  <last_update_submitted>June 30, 2023</last_update_submitted>
  <last_update_submitted_qc>June 30, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">July 3, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Myeloid neoplasm</keyword>
  <keyword>fibroblast growth factor receptor inhibitor</keyword>
  <keyword>FGFR1 rearrangement</keyword>
  <keyword>8p11</keyword>
  <keyword>eosinophilia</keyword>
  <keyword>eosinophilic syndrome</keyword>
  <keyword>Lymphoid neoplasm</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neoplasms</mesh_term>
    <mesh_term>Myeloproliferative Disorders</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_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.&#xD;
The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency</ipd_description>
    <ipd_info_type>Study Protocol</ipd_info_type>
    <ipd_info_type>Statistical Analysis Plan (SAP)</ipd_info_type>
    <ipd_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_time_frame>
    <ipd_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_access_criteria>
    <ipd_url>https://www.incyte.com/our-company/compliance-and-transparency</ipd_url>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03011372
org study id: INCB 54828-203
nct id: NCT03011372
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
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.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: The proportion of participants who achieve Complete Response (CR) based on response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement
time frame: : Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
measure: The proportion of subjects who achieve response, defined as a best response of CR or PR, as determined by investigator assessment according to the response criteria
time frame: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
measure: The proportion of subjects who achieve a complete cytogenetic response (CCyR) as assessed by local analysis and investigator evaluation
time frame: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
measure: The proportion of subjects who achieve a partial cytogenetic response (PCyR) as assessed by local analysis and investigator evaluation
time frame: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
measure: Duration of CR, defined as the time from first assessment of CR to the earlier of disease progression or death due to any cause
time frame: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
measure: Duration of response, defined as the time from first assessment of CR or PR to the earlier of disease progression or death due to any cause
time frame: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
measure: Progression-free survival (PFS)
time frame: From the date of first study drug dose until the date of disease progression or until death due to any cause, whichever is earlier, assessed up to approximately 24 months.
description: PFS is defined as the time from the first date of taking study drug until the date of disease progression, as measured by response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement, or until death due to any cause, whichever is earlier.
measure: Overall survival
time frame: From date of first study drug dose until death due to any cause, assessed up to approximately 24 months.
description: Overall survival is defined as the time from the first day of taking study drug until death due to any cause. Subjects without death observed at the time of the analysis will be censored at last date known to be alive.
measure: Safety and tolerability as assessed by frequency, duration, and severity of adverse events
time frame: From baseline through 30-35 days after end of treatment, up to 7 months per individual subject
description: A treatment-emergent AE was defined as an event occurring after exposure to at least 1 dose of study drug. A treatment-related AE was defined as an event with a definite, probable, or possible causality to study medication. A serious AE is an event resulting in death, hospitalization, persistent or significant disability/incapacity, or is life threatening, a congenital anomaly/birth defect or requires medical or surgical intervention to prevent 1 of the outcomes above. The intensity of an AE was graded according to the National Cancer Institute common terminology criteria for adverse events (NCI-CTCAE) version 4.03: Grade 1 (Mild); Grade 2 (Moderate); Grade 3 (Severe); Grade 4 (life-threatening).
arm group label: Pemigatinib
arm group type: Experimental
intervention type: Drug
intervention name: 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.
arm group label: Pemigatinib
other name: INCB054828
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Philomena Collucci, MD
role: Study Director
affiliation: Incyte Corporation
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country: Austria
country: Belgium
country: Canada
country: France
country: Germany
country: Italy
country: Japan
country: Spain
country: Switzerland
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Neoplasms
mesh term: Myeloproliferative Disorders
sharing ipd: Yes
ipd 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 info type: Study Protocol
ipd info type: Statistical Analysis Plan (SAP)
ipd 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 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 url: https://www.incyte.com/our-company/compliance-and-transparency