<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/NCT02261207</url>
  </required_header>
  <id_info>
    <org_study_id>SFT-AX2014</org_study_id>
    <secondary_id>2013-005596-40</secondary_id>
    <nct_id>NCT02261207</nct_id>
  </id_info>
  <brief_title>Phase II Study on Axitinib in Advanced Solitary Fibrous Tumor</brief_title>
  <official_title>Phase II Study on Axitinib in Advanced VEGFR and/or PDGFR Solitary Fibrous Tumor</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Fondazione IRCCS Istituto Nazionale dei Tumori, Milano</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Fondazione IRCCS Istituto Nazionale dei Tumori, Milano</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is an investigator initiated, open label, prospective, non-randomized, phase II trial&#xD;
      aimed at evaluating the activity of Axitinib in progressive VEGFR2 and/or PDGFRB positive&#xD;
      advanced Solitary Fibrous Tumor (SFT) patients. Patients with a documented and centrally&#xD;
      reviewed pathologic and radiologic diagnosis of progressive VEGFR2 and/or PDGFRB positive&#xD;
      advanced SFT may enter in the study. Axitinib will be administered at the dose of 5mg twice a&#xD;
      day, continuously. Treatment will be continued till evidence of progression, or toxicities or&#xD;
      patient withdrawal.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is an investigator initiated, open label, prospective, non-randomized, phase II trial&#xD;
      aimed at evaluating the activity of Axitinib in progressive VEGFR2 and/or PDGFRB positive&#xD;
      advanced Solitary Fibrous Tumor (SFT) patients. Patients with a documented and centrally&#xD;
      reviewed pathologic and radiologic diagnosis of progressive VEGFR2 and/or PDGFRB positive&#xD;
      advanced SFT may enter in the study. Axitinib will be administered at the dose of 5mg twice a&#xD;
      day, continuously. Treatment will be continued till evidence of progression, or toxicities or&#xD;
      patient withdrawal.&#xD;
&#xD;
      The activity of Axitinib will be evaluated in terms of overall response rate according to&#xD;
      Choi Criteria as defined for GIST patients treated with Imatinib, extended even to MRI.&#xD;
&#xD;
      Secondary objectives of the study will be: response rate by RECIST, progression free&#xD;
      survival, overall survival, clinical benefit (RECIST Complete Response + Partial Response +&#xD;
      Stable Disease &gt;6 months). Whenever possible a post-treatment biopsy will be performed to&#xD;
      assess Axitinib targets status after treatment and to correlate their status to the response.&#xD;
&#xD;
      Tumor assessment with CT scan and/or MRI will be performed within 4 weeks prior to first dose&#xD;
      of Axitinib and after 4 weeks from starting treatment, then every 2 months until progression&#xD;
      or toxicity.&#xD;
&#xD;
      As per protocol the experimental treatment can be administered in front or further line.&#xD;
&#xD;
      The study will be considered positive if 30% of response rate by Choi criteria is observed.&#xD;
      To this end a maximum of 16 evaluable patients will be enrolled in two years.&#xD;
&#xD;
      In case of positive study the trial will be re-open to confirm the result in a larger number&#xD;
      of patients.&#xD;
&#xD;
      .&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>November 2014</start_date>
  <completion_date type="Actual">March 2018</completion_date>
  <primary_completion_date type="Actual">October 2017</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</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>Response rate by Choi criteria</measure>
    <time_frame>4 years</time_frame>
    <description>response rate, according to Choi Criteria</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Response rate by RECIST criteria</measure>
    <time_frame>4 years</time_frame>
    <description>response rate according to RECIST</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Overall survival</measure>
    <time_frame>4 years</time_frame>
    <description>Evaluation of overall survival</description>
  </other_outcome>
  <other_outcome>
    <measure>Progression Free Survival</measure>
    <time_frame>4 years</time_frame>
    <description>Evaluation of Progression Free Survival</description>
  </other_outcome>
  <other_outcome>
    <measure>Clinical Benefit</measure>
    <time_frame>4 years</time_frame>
    <description>Evaluation of clinical benefit according to response rate, overall survival, progression free survival</description>
  </other_outcome>
  <other_outcome>
    <measure>Post-treatment Axitinib target status assessment</measure>
    <time_frame>4 years</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Safety essessment</measure>
    <time_frame>4 years</time_frame>
    <description>assessment of the safety profile of Axitinib in agreement with the incidence of related adverse events.</description>
  </other_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">17</enrollment>
  <condition>Solitary Fibrous Tumor</condition>
  <arm_group>
    <arm_group_label>Axitinib</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Axitinib will be administered at the dose of 5mg twice a day, continuously. Treatment will be continued till evidence of progression, or toxicities or patient withdrawal.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Axitinib</intervention_name>
    <description>Axitinib will be administered at the dose of 5mg twice a day, continuously. Treatment will be continued till evidence of progression, or toxicities or patient withdrawal.</description>
    <arm_group_label>Axitinib</arm_group_label>
    <other_name>Inlyta</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Pat centrally confirmed diagnosis of solitary fibrous tumor&#xD;
&#xD;
          -  Expression of PDGFRB and/or VEGFR2 by immunohistochemistry on formalin fixed-paraffin&#xD;
             embedded (FFPE) material as minimal requirement. Activation of PDGFRB and/or VEGFR2 by&#xD;
             real time C reactive protein of PDGFB and VEGFA on FFPE material (if in sufficient&#xD;
             quantity) or by biochemistry on frozen material (if available)&#xD;
&#xD;
          -  Locally advanced disease (i.e. surgical resection of local disease unfeasible&#xD;
             radically, or unaccepted by the patient, or amenable to become less demolitive, or&#xD;
             feasible, or easier, after cytoreduction) and/or metastatic disease&#xD;
&#xD;
          -  Measurable disease&#xD;
&#xD;
          -  Centrally confirmed evidence of progression by RECIST during the 6 months before study&#xD;
             entry&#xD;
&#xD;
          -  1st-line vs 3-rd-line&#xD;
&#xD;
          -  Eastern Cooperative Oncology Group (ECOG) Performance Status = 0, 1, 2&#xD;
&#xD;
          -  Adequate bone marrow function, defined as the following: absolute neutrophil count&#xD;
             (ANC) &gt;1.5 x 109/L, platelets &gt;100 x 109/L, Hb &gt;9 g/dL. Blood transfusions are allowed&#xD;
             to reach the baseline requested Hb level&#xD;
&#xD;
          -  Adequate organ function, defined as the following: total bilirubin within normal&#xD;
             institutional limits (but in case of Gilbert's syndrome), aspartate aminotransferase&#xD;
             (AST) and Serum Glutamic Pyruvic Transaminase (ALT) &lt;2.5 x upper normal limit (UNL),&#xD;
             creatinine &lt;1.5 x upper normal limit (UNL), within normal institutional limits or&#xD;
             creatinine clearance ≥60 mL/min/1.73 m2 for patients with creatinine levels above&#xD;
             institutional normal&#xD;
&#xD;
          -  Cardiac ejection fraction ≥50% as measured by echocardiogram&#xD;
&#xD;
          -  Age &gt; 18 yrs&#xD;
&#xD;
          -  Female patients of child-bearing potential must have negative pregnancy test within 7&#xD;
             days before initiation of study drug dosing. Post-menopausal women must be amenorrheic&#xD;
             for at least 12 months to be considered of non-childbearing potential. Male and female&#xD;
             patients of reproductive potential must agree to employ an effective method of birth&#xD;
             control throughout the study and for up to 3 months following discontinuation of study&#xD;
             drug&#xD;
&#xD;
          -  No history of arterial and/or venous thromboembolic event within the previous 12&#xD;
             months&#xD;
&#xD;
          -  Written, voluntary informed consent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Other primary malignancy with &lt;5 years clinically assessed disease-free interval,&#xD;
             except basal cell skin cancer, cervical carcinoma in situ, or other neoplasms judged&#xD;
             to entail a low risk of relapse&#xD;
&#xD;
          -  Previous treatment with any other investigational or not investigational agents and or&#xD;
             radiation therapy within 28 days of first day of study drug dosing, or patients who&#xD;
             have not recovered from adverse events due to agents administered more than 4 weeks&#xD;
             earlier&#xD;
&#xD;
          -  Major surgery within 2 weeks prior to study entry&#xD;
&#xD;
          -  Previous radiotherapy to ≥25 % of the bone marrow&#xD;
&#xD;
          -  Concomitant other investigational agents or concurrent anticancer therapy. In&#xD;
             addition, all herbal (alternative) medicines are excluded&#xD;
&#xD;
          -  Grade III/IV cardiac problems as defined by the New York Heart Association Criteria&#xD;
             (i.e., history of uncontrolled or symptomatic angina, history of arrhythmias requiring&#xD;
             medications, or clinically significant, with the exception of asymptomatic atrial&#xD;
             fibrillation requiring anticoagulation, myocardial infarction &lt; 6 months from study&#xD;
             entry, uncontrolled or symptomatic congestive heart failure, ejection fraction below&#xD;
             the institutional normal limit)&#xD;
&#xD;
          -  Known chronic liver disease (i.e., chronic active hepatitis, and cirrhosis with&#xD;
             exception of patients with Gilbert's syndrome, asymptomatic gallstones, liver&#xD;
             metastases or stable chronic liver disease per investigator assessment)&#xD;
&#xD;
          -  Known diagnosis of human immunodeficiency virus (HIV) infection&#xD;
&#xD;
          -  History of allergic reactions attributed to compounds of similar chemical or biologic&#xD;
             composition to Axitinib&#xD;
&#xD;
          -  Expected non-compliance to medical regimens&#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>Silvia Stacchiotti, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Fondazione IRCCS Istituto Nazionale dei Tumori, Milano</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Fondazione IRCCS Istituto Naazionale dei Tumori</name>
      <address>
        <city>Milan</city>
        <state>MI</state>
        <zip>20133</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Italy</country>
  </location_countries>
  <verification_date>September 2021</verification_date>
  <study_first_submitted>January 30, 2014</study_first_submitted>
  <study_first_submitted_qc>October 6, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 10, 2014</study_first_posted>
  <last_update_submitted>September 22, 2021</last_update_submitted>
  <last_update_submitted_qc>September 22, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">September 28, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Solitary Fibrous Tumors</mesh_term>
    <mesh_term>Hemangiopericytoma</mesh_term>
    <mesh_term>Neoplasms, Fibrous Tissue</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Axitinib</mesh_term>
  </intervention_browse>
  <!-- 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/NCT02261207
org study id: SFT-AX2014
secondary id: 2013-005596-40
nct id: NCT02261207
lead sponsor: 
has dmc: No
This is an investigator initiated, open label, prospective, non-randomized, phase II trial
      aimed at evaluating the activity of Axitinib in progressive VEGFR2 and/or PDGFRB positive
      advanced Solitary Fibrous Tumor (SFT) patients. Patients with a documented and centrally
      reviewed pathologic and radiologic diagnosis of progressive VEGFR2 and/or PDGFRB positive
      advanced SFT may enter in the study. Axitinib will be administered at the dose of 5mg twice a
      day, continuously. Treatment will be continued till evidence of progression, or toxicities or
      patient withdrawal.
This is an investigator initiated, open label, prospective, non-randomized, phase II trial
      aimed at evaluating the activity of Axitinib in progressive VEGFR2 and/or PDGFRB positive
      advanced Solitary Fibrous Tumor (SFT) patients. Patients with a documented and centrally
      reviewed pathologic and radiologic diagnosis of progressive VEGFR2 and/or PDGFRB positive
      advanced SFT may enter in the study. Axitinib will be administered at the dose of 5mg twice a
      day, continuously. Treatment will be continued till evidence of progression, or toxicities or
      patient withdrawal.

      The activity of Axitinib will be evaluated in terms of overall response rate according to
      Choi Criteria as defined for GIST patients treated with Imatinib, extended even to MRI.

      Secondary objectives of the study will be: response rate by RECIST, progression free
      survival, overall survival, clinical benefit (RECIST Complete Response + Partial Response +
      Stable Disease >6 months). Whenever possible a post-treatment biopsy will be performed to
      assess Axitinib targets status after treatment and to correlate their status to the response.

      Tumor assessment with CT scan and/or MRI will be performed within 4 weeks prior to first dose
      of Axitinib and after 4 weeks from starting treatment, then every 2 months until progression
      or toxicity.

      As per protocol the experimental treatment can be administered in front or further line.

      The study will be considered positive if 30% of response rate by Choi criteria is observed.
      To this end a maximum of 16 evaluable patients will be enrolled in two years.

      In case of positive study the trial will be re-open to confirm the result in a larger number
      of patients.

      .
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Response rate by Choi criteria
time frame: 4 years
description: response rate, according to Choi Criteria
measure: Response rate by RECIST criteria
time frame: 4 years
description: response rate according to RECIST
measure: Overall survival
time frame: 4 years
description: Evaluation of overall survival
measure: Progression Free Survival
time frame: 4 years
description: Evaluation of Progression Free Survival
measure: Clinical Benefit
time frame: 4 years
description: Evaluation of clinical benefit according to response rate, overall survival, progression free survival
measure: Post-treatment Axitinib target status assessment
time frame: 4 years
measure: Safety essessment
time frame: 4 years
description: assessment of the safety profile of Axitinib in agreement with the incidence of related adverse events.
arm group label: Axitinib
arm group type: Experimental
description: Axitinib will be administered at the dose of 5mg twice a day, continuously. Treatment will be continued till evidence of progression, or toxicities or patient withdrawal.
intervention type: Drug
intervention name: Axitinib
description: Axitinib will be administered at the dose of 5mg twice a day, continuously. Treatment will be continued till evidence of progression, or toxicities or patient withdrawal.
arm group label: Axitinib
other name: Inlyta
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Silvia Stacchiotti, MD
role: Principal Investigator
affiliation: Fondazione IRCCS Istituto Nazionale dei Tumori, Milano
facility: 
country: Italy
responsible party type: Sponsor
mesh term: Solitary Fibrous Tumors
mesh term: Hemangiopericytoma
mesh term: Neoplasms, Fibrous Tissue
mesh term: Axitinib