xml: <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/NCT05987124</url>
    </required_header>
    <id_info>
      <org_study_id>NYMC 600</org_study_id>
      <nct_id>NCT05987124</nct_id>
    </id_info>
    <brief_title>Defibrotide Dose-escalation for SOS Post-HSCT</brief_title>
    <official_title>A Phase II Intrapatient Open-Label Dose Escalation Trial of Defibrotide in Hematopoietic Cell Transplantation (HCT) Recipients With Sinusoidal Obstructive Syndrome (SOS) Post-HCT Associated With Either Renal and/or Pulmonary Dysfunction With Either Refractory or Progressive Disease Following Defibrotide Therapy</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>New York Medical College</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>New York Medical College</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 research study is being done to determine the safety and tolerability of increasing&#xD;
        doses of defibrotide within a single patient with sinusoidal obstructive syndrome&#xD;
        (SOS)/veno-occlusive disease (VOD) after hematopoietic cell transplantation (HCT) associated&#xD;
        with either kidney and/or lung impairment that has not obtained a complete response (CR) or&#xD;
        progressed in severity with standard doses of defibrotide.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">August 1, 2023</start_date>
    <completion_date type="Anticipated">August 1, 2027</completion_date>
    <primary_completion_date type="Anticipated">August 1, 2026</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>To determine the incidence of grade 3 or 4 adverse events related to defibrotide</measure>
      <time_frame>100 days</time_frame>
      <description>grade 3 and 4 adverse events possible or probably related to defibrotide will be collected</description>
    </primary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">20</enrollment>
    <condition>Sinusoidal Obstruction Syndrome</condition>
    <condition>Veno-occlusive Disease</condition>
    <arm_group>
      <arm_group_label>Defibrotide</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>7.1.1 HCT recipients with SOS/VOD and renal and/or pulmonary dysfunction with either PR after 21 days of standard doses of defibrotide (25mg/kg/day) or SD, after 14 days of standard doses of defibrotide (25mg/kg/day) progressive disease after 7 days on defibrotide (25mg/kg/day) will undergo intra-patient dose escalation every 4 days until a complete response is obtained up until the highest dose level of 100mg/kg/day at which point an endpoint of CR, PR or SD will be sought (see 7.2 for definition of response) (Maximum of 4 dose levels) (7.1.2):</description>
    </arm_group>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Defibrotide</intervention_name>
      <description>HCT recipients with SOS/VOD and renal and/or pulmonary dysfunction with either PR after 21 days of standard doses of defibrotide (25mg/kg/day) or SD, after 14 days of standard doses of defibrotide (25mg/kg/day) progressive disease after 7 days on defibrotide (25mg/kg/day) will undergo intra-patient dose escalation every 4 days until a complete response is obtained up until the highest dose level of 100mg/kg/day at which point an endpoint of CR, PR or SD will be sought(Maximum of 4 dose levels). Defibrotide will be administered in D5W or 0.9% NaCl via IV infusion over 2 hours q6 hours.</description>
      <arm_group_label>Defibrotide</arm_group_label>
      <other_name>Defitelio</other_name>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  HCT recipients (Auto or Allograft)&#xD;
  &#xD;
            -  SOS/VOD as defined by Cairo/Cooke Diagnostic criteria (1) (Table 3) with either renal&#xD;
               and/or pulmonary dysfunction as defined by Cairo/Cooke Grading criteria (1) (Appendix&#xD;
               I).&#xD;
  &#xD;
            -  Unresponsive to standard defibrotide therapy as defined by at least one of the&#xD;
               following:&#xD;
  &#xD;
            -  Patients with SOS/VOD failing to obtain a complete response (CR) defined by Grade I or&#xD;
               less by Cairo/Cooke Grading criteria (1) (Appendix I). This would therefore include&#xD;
               patients with stable disease after at least 14 days of defibrotide or partial response&#xD;
               after at least 21 days of defibrotide (25mg/kg/day).&#xD;
  &#xD;
            -  Progressive disease defined by progression of at least one grade or more from&#xD;
               diagnostic grade as defined by Cairo/Cooke Grading criteria (1) (Appendix I) following&#xD;
               at least 7 days of defibrotide (25mg/kg/day).&#xD;
  &#xD;
            -  Age 1 month - 75 years&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Patients who did not receive HCT.&#xD;
  &#xD;
            -  Concomitant systemic anticoagulation (excluding central venous line management,&#xD;
               fibrinolytic instillation for central venous line occlusion, management of&#xD;
               intermittent dialysis or ultrafiltration of CVVH).&#xD;
  &#xD;
            -  Active bleeding and/or hemorrhage of at least grade 2 and above.&#xD;
  &#xD;
            -  History of development of Grade III/IV anaphylaxis probably or directly secondary to&#xD;
               defibrotide.&#xD;
  &#xD;
            -  Female patients who are pregnant or breast feeding.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>1 Month</minimum_age>
      <maximum_age>75 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Mitchell Cairo, MD</last_name>
      <role>Principal Investigator</role>
      <affiliation>New York Medical College</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Mitchell Cairo, MD</last_name>
      <phone>914-594-2150</phone>
      <email>mitchell_cairo@nymc.edu</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Lauren Harrison, MSN</last_name>
      <phone>617-285-7844</phone>
      <email>lauren_harrison@nymc.edu</email>
    </overall_contact_backup>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 4, 2023</study_first_submitted>
    <study_first_submitted_qc>August 4, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 14, 2023</study_first_posted>
    <last_update_submitted>August 4, 2023</last_update_submitted>
    <last_update_submitted_qc>August 4, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 14, 2023</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>Hepatic Veno-Occlusive Disease</mesh_term>
      <mesh_term>Syndrome</mesh_term>
    </condition_browse>
    <intervention_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Defibrotide</mesh_term>
    </intervention_browse>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT05987124
  org study id: NYMC 600
  nct id: NCT05987124
  lead sponsor: 
  has dmc: No
  is fda regulated drug: Yes
  is fda regulated device: No
  This research study is being done to determine the safety and tolerability of increasing
        doses of defibrotide within a single patient with sinusoidal obstructive syndrome
        (SOS)/veno-occlusive disease (VOD) after hematopoietic cell transplantation (HCT) associated
        with either kidney and/or lung impairment that has not obtained a complete response (CR) or
        progressed in severity with standard doses of defibrotide.
  allocation: N/A
  intervention model: Single Group Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: To determine the incidence of grade 3 or 4 adverse events related to defibrotide
  time frame: 100 days
  description: grade 3 and 4 adverse events possible or probably related to defibrotide will be collected
  arm group label: Defibrotide
  arm group type: Experimental
  description: 7.1.1 HCT recipients with SOS/VOD and renal and/or pulmonary dysfunction with either PR after 21 days of standard doses of defibrotide (25mg/kg/day) or SD, after 14 days of standard doses of defibrotide (25mg/kg/day) progressive disease after 7 days on defibrotide (25mg/kg/day) will undergo intra-patient dose escalation every 4 days until a complete response is obtained up until the highest dose level of 100mg/kg/day at which point an endpoint of CR, PR or SD will be sought (see 7.2 for definition of response) (Maximum of 4 dose levels) (7.1.2):
  intervention type: Drug
  intervention name: Defibrotide
  description: HCT recipients with SOS/VOD and renal and/or pulmonary dysfunction with either PR after 21 days of standard doses of defibrotide (25mg/kg/day) or SD, after 14 days of standard doses of defibrotide (25mg/kg/day) progressive disease after 7 days on defibrotide (25mg/kg/day) will undergo intra-patient dose escalation every 4 days until a complete response is obtained up until the highest dose level of 100mg/kg/day at which point an endpoint of CR, PR or SD will be sought(Maximum of 4 dose levels). Defibrotide will be administered in D5W or 0.9% NaCl via IV infusion over 2 hours q6 hours.
  arm group label: Defibrotide
  other name: Defitelio
  criteria: 
  gender: All
  minimum age: 1 Month
  maximum age: 75 Years
  healthy volunteers: No
  last name: Mitchell Cairo, MD
  role: Principal Investigator
  affiliation: New York Medical College
  last name: Mitchell Cairo, MD
  phone: 914-594-2150
  email: mitchell_cairo@nymc.edu
  last name: Lauren Harrison, MSN
  phone: 617-285-7844
  email: lauren_harrison@nymc.edu
  responsible party type: Sponsor
  mesh term: Hepatic Veno-Occlusive Disease
  mesh term: Syndrome
  mesh term: Defibrotide