<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/NCT04404569</url>
  </required_header>
  <id_info>
    <org_study_id>BXQ-350.AE</org_study_id>
    <nct_id>NCT04404569</nct_id>
  </id_info>
  <brief_title>Continued Treatment for Participants Enrolled in Studies of BXQ-350</brief_title>
  <acronym>ETERNITI</acronym>
  <official_title>An Open-Label, Multi-Center, Rollover Study to Provide Continued Treatment Access for Participants Enrolled in Studies of BXQ-350</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Bexion Pharmaceuticals, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>CTI Clinical Trial and Consulting Services</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Bexion Pharmaceuticals, Inc.</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>
      This is a study to allow continued treatment for participants enrolled in studies of BXQ-350.&#xD;
      This study is intended only for those who have completed the required study observation&#xD;
      period or are still on treatment upon the closure of their respective BXQ-350 clinical study.&#xD;
      The additional treatment is optional and voluntary.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is an open-label, multi-center, rollover study to allow continued treatment access for&#xD;
      participants enrolled in studies of BXQ-350. This study is intended for subjects who have&#xD;
      completed the required study observation period or are still on treatment upon the closure of&#xD;
      their respective BXQ-350 clinical study, and who are judged by the Investigator to benefit&#xD;
      from continued treatment with BXQ-350. The additional treatment is optional and voluntary.&#xD;
&#xD;
      This clinical trial information was submitted voluntarily under the applicable law and,&#xD;
      therefore, certain submission deadlines may not apply. (That is, clinical trial information&#xD;
      for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public&#xD;
      Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by&#xD;
      sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.)&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Enrolling by invitation</overall_status>
  <start_date type="Actual">June 8, 2020</start_date>
  <completion_date type="Anticipated">July 2024</completion_date>
  <primary_completion_date type="Anticipated">May 2024</primary_completion_date>
  <phase>Phase 1</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>Percentage of subjects with clinical benefit</measure>
    <time_frame>Day 1 up to approximately 5 years</time_frame>
    <description>Subjects will be assessed at every visit for continued clinical benefit as per Investigator's assessment.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Incidence of Treatment Emergent Adverse Events as Assessed by CTCAE v5.0</measure>
    <time_frame>Day 1 up to approximately 5 years</time_frame>
    <description>Subjects will be assessed at every visit for adverse events</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to Disease Progression</measure>
    <time_frame>From Day 1 until the date of first documented disease progression or date of death from any cause, whichever occurs first; up to approximately 5 years</time_frame>
    <description>Subjects will be assessed for disease progression throughout the study per institutional standard of care.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Overall Survival</measure>
    <time_frame>From date of BXQ-350 treatment completion until time of death from any cause; up to approximately 5 years</time_frame>
    <description>Subjects will be followed monthly for survival upon completion of BXQ-350 treatment.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">50</enrollment>
  <condition>Neoplasms</condition>
  <arm_group>
    <arm_group_label>Continued Treatment</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Subjects who have completed the required study observation period or are still on treatment upon the closure of their respective BXQ-350 clinical study, and who are judged by the Investigator to benefit from continued treatment with BXQ-350. Treatment will begin after completion of the End of Study visit of the prior BXQ-350 clinical study.&#xD;
The established safe dose of BXQ-350 from previous adult and pediatric phase 1 studies is 2.4 mg/kg and 3.2 mg/kg respectively once every 28 days (± 3 days). BXQ-350 will be administered intravenously at the same dose level and frequency the subject was receiving at the end of the prior BXQ-350 clinical study. Subjects receiving a reduced dose at the end of the prior BXQ-350 clinical study due to toxicity may continue to receive a reduced dose.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>BXQ-350</intervention_name>
    <description>BXQ-350 is a novel anti-neoplastic therapeutic agent configured from two components: Saposin C (SapC), an expressed (human) lysosomal protein, and the phospholipid dioleoylphosphatidyl-serine (DOPS), a phospholipid located on cell membranes (clinical formulation BXQ-350). BXQ-350 is administered by intravenous (IV) infusion in 28-day cycles</description>
    <arm_group_label>Continued Treatment</arm_group_label>
    <other_name>SapC-DOPS</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Each subject must meet the following criteria:&#xD;
&#xD;
               1. Has participated in and completed all protocol-specified treatments through the&#xD;
                  required study observation period or time of closure for a prior Bexion-sponsored&#xD;
                  BXQ-350 clinical study&#xD;
&#xD;
               2. Has completed the End of Study visit of the prior Bexion-sponsored BXQ-350&#xD;
                  clinical study&#xD;
&#xD;
               3. Did not meet any treatment discontinuation criteria of the original prior Bexion-&#xD;
                  sponsored BXQ-350 clinical study&#xD;
&#xD;
               4. Investigator opinion indicates that continued treatment with BXQ-350 is&#xD;
                  clinically appropriate for the subject&#xD;
&#xD;
               5. Provide signed, written informed consent prior to the initiation of any&#xD;
                  study-specific procedures (Consent from Guardians for minor children and patient&#xD;
                  assent according to Institution and Institutional Review Board (IRB) standards)&#xD;
&#xD;
               6. Have a negative serum pregnancy test result within 28 days prior to the first&#xD;
                  continuing treatment assessment for females of child bearing potential (FCBP);&#xD;
                  not applicable to subjects who are unable to become pregnant, including those&#xD;
                  with tubal ligation, bilateral oophorectomy and/or hysterectomy)&#xD;
&#xD;
               7. FCBP and male subjects whose sexual partner(s) are FCBP must agree to abstain&#xD;
                  from heterosexual activity or use a double barrier method of contraception (e.g.,&#xD;
                  condom and occlusive cap with spermicide) or highly effective contraception&#xD;
                  (intrauterine device or system, established hormonal contraceptive methods on a&#xD;
                  stable dose from the time of the last menstrual cycle, or vasectomized partner&#xD;
                  with confirmed azoospermia) from the time of study entry to 1 month after the&#xD;
                  last day of treatment&#xD;
&#xD;
                  Exclusion Criteria:&#xD;
&#xD;
          -  Subjects must not meet any of the following criteria:&#xD;
&#xD;
               1. Has any ongoing adverse event that could impact tolerability to BXQ-350&#xD;
&#xD;
               2. Receiving any other non-BXQ-350 study treatment modalities with curative intent,&#xD;
                  including investigational products other than BXQ-350&#xD;
&#xD;
               3. Are pregnant or nursing (lactating), where pregnancy is defined as the state of a&#xD;
                  female after conception and until the termination of gestation, confirmed by a&#xD;
                  positive serum human chorionic gonadotropin (hCG) laboratory test&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Year</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>University of Kentucky Markey Cancer Center</name>
      <address>
        <city>Lexington</city>
        <state>Kentucky</state>
        <zip>40536</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Cincinnati Barrett Center</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45219</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Ohio State University Comprehensive Cancer Center</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43210</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2023</verification_date>
  <study_first_submitted>May 20, 2020</study_first_submitted>
  <study_first_submitted_qc>May 22, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">May 27, 2020</study_first_posted>
  <last_update_submitted>May 23, 2023</last_update_submitted>
  <last_update_submitted_qc>May 23, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">May 24, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>High-grade Glioma</keyword>
  <keyword>Glioblastoma</keyword>
  <keyword>Solid Tumor</keyword>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </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/NCT04404569
org study id: BXQ-350.AE
nct id: NCT04404569
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
This is a study to allow continued treatment for participants enrolled in studies of BXQ-350.
      This study is intended only for those who have completed the required study observation
      period or are still on treatment upon the closure of their respective BXQ-350 clinical study.
      The additional treatment is optional and voluntary.
This is an open-label, multi-center, rollover study to allow continued treatment access for
      participants enrolled in studies of BXQ-350. This study is intended for subjects who have
      completed the required study observation period or are still on treatment upon the closure of
      their respective BXQ-350 clinical study, and who are judged by the Investigator to benefit
      from continued treatment with BXQ-350. The additional treatment is optional and voluntary.

      This clinical trial information was submitted voluntarily under the applicable law and,
      therefore, certain submission deadlines may not apply. (That is, clinical trial information
      for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public
      Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by
      sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.)
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Percentage of subjects with clinical benefit
time frame: Day 1 up to approximately 5 years
description: Subjects will be assessed at every visit for continued clinical benefit as per Investigator's assessment.
measure: Incidence of Treatment Emergent Adverse Events as Assessed by CTCAE v5.0
time frame: Day 1 up to approximately 5 years
description: Subjects will be assessed at every visit for adverse events
measure: Time to Disease Progression
time frame: From Day 1 until the date of first documented disease progression or date of death from any cause, whichever occurs first; up to approximately 5 years
description: Subjects will be assessed for disease progression throughout the study per institutional standard of care.
measure: Overall Survival
time frame: From date of BXQ-350 treatment completion until time of death from any cause; up to approximately 5 years
description: Subjects will be followed monthly for survival upon completion of BXQ-350 treatment.
arm group label: Continued Treatment
arm group type: Experimental
description: Subjects who have completed the required study observation period or are still on treatment upon the closure of their respective BXQ-350 clinical study, and who are judged by the Investigator to benefit from continued treatment with BXQ-350. Treatment will begin after completion of the End of Study visit of the prior BXQ-350 clinical study.
The established safe dose of BXQ-350 from previous adult and pediatric phase 1 studies is 2.4 mg/kg and 3.2 mg/kg respectively once every 28 days (± 3 days). BXQ-350 will be administered intravenously at the same dose level and frequency the subject was receiving at the end of the prior BXQ-350 clinical study. Subjects receiving a reduced dose at the end of the prior BXQ-350 clinical study due to toxicity may continue to receive a reduced dose.
intervention type: Drug
intervention name: BXQ-350
description: BXQ-350 is a novel anti-neoplastic therapeutic agent configured from two components: Saposin C (SapC), an expressed (human) lysosomal protein, and the phospholipid dioleoylphosphatidyl-serine (DOPS), a phospholipid located on cell membranes (clinical formulation BXQ-350). BXQ-350 is administered by intravenous (IV) infusion in 28-day cycles
arm group label: Continued Treatment
other name: SapC-DOPS
criteria: 
gender: All
minimum age: 1 Year
maximum age: N/A
healthy volunteers: No
facility: 
facility: 
facility: 
country: United States
responsible party type: Sponsor
sharing ipd: No