<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/NCT03805581</url>
  </required_header>
  <id_info>
    <org_study_id>NYMC-192</org_study_id>
    <nct_id>NCT03805581</nct_id>
  </id_info>
  <brief_title>Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome</brief_title>
  <official_title>A Phase II Study to Determine the Safety of Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>New York Medical College</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Johns Hopkins University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </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 study evaluates the safety of defibrotide in subjects with sickle cell disease&#xD;
      (SCD)-associated acute chest syndrome (ACS).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">January 12, 2018</start_date>
  <completion_date type="Anticipated">July 2023</completion_date>
  <primary_completion_date type="Actual">July 1, 2022</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>Number of patients with grade III/IV allergic reaction to defibrotide</measure>
    <time_frame>30 days</time_frame>
    <description>All patients will be monitored for allergic reaction probably or definitely related to defibrotide administration.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of patients with grade III/IV hemorrhage</measure>
    <time_frame>30 days</time_frame>
    <description>All patients will be monitored for hemorrhage probably or definitely related to defibrotide.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of patients with improvement in clinical signs of Acute Chest Syndrome</measure>
    <time_frame>30 days</time_frame>
    <description>Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of ACS</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">20</enrollment>
  <condition>Sickle Cell Disease</condition>
  <condition>Acute Chest Syndrome</condition>
  <arm_group>
    <arm_group_label>Interventional</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Defibrotide 6.25 mg/kg IV q6h</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Defibrotide</intervention_name>
    <description>Defibrotide 6.25 mg/kg IV q6h up to 7 days</description>
    <arm_group_label>Interventional</arm_group_label>
    <other_name>Defitelio®</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  SCD-associated ACS with the presence of any two or more of the following signs not&#xD;
             explained by other etiologies: Fever, Chest pain, Cough, Dyspnea, Tachypnea for age,&#xD;
             Pulmonary infiltrate on CXR and/or Chest CT scan, Decreased O2 saturation with or&#xD;
             without oxygen supplement;&#xD;
&#xD;
          -  Age 2 to 40 years of age;&#xD;
&#xD;
          -  Homozygous Hemoglobin S Disease, Hemoglobin SC Disease or Hemoglobin S 0/+&#xD;
             thalassemia;&#xD;
&#xD;
          -  Informed consent/assent;&#xD;
&#xD;
          -  Consent of patient/parent within ≤72 hours after inpatient admission for&#xD;
             SCD-associated ACS.&#xD;
&#xD;
          -  Females of childbearing age will have a negative pregnancy test.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Current Grade III or IV hemorrhage;&#xD;
&#xD;
          -  Previous hypersensitivity reaction to defibrotide;&#xD;
&#xD;
          -  Current systemic anti-coagulant therapy and/or fibrinolytic therapy;&#xD;
&#xD;
          -  Consent of patient/parent greater than 72 hours of inpatient admission for&#xD;
             SCD-associated ACS;&#xD;
&#xD;
          -  No signed informed consent&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>40 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Mitchell S Cairo, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>New York Medical College</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>New York Medical College</name>
      <address>
        <city>Valhalla</city>
        <state>New York</state>
        <zip>10595</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>October 2022</verification_date>
  <study_first_submitted>December 18, 2018</study_first_submitted>
  <study_first_submitted_qc>January 14, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 15, 2019</study_first_posted>
  <last_update_submitted>October 24, 2022</last_update_submitted>
  <last_update_submitted_qc>October 24, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">October 25, 2022</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>Acute Chest Syndrome</mesh_term>
    <mesh_term>Anemia, Sickle Cell</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>


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/NCT03805581
org study id: NYMC-192
nct id: NCT03805581
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
This study evaluates the safety of defibrotide in subjects with sickle cell disease
      (SCD)-associated acute chest syndrome (ACS).
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of patients with grade III/IV allergic reaction to defibrotide
time frame: 30 days
description: All patients will be monitored for allergic reaction probably or definitely related to defibrotide administration.
measure: Number of patients with grade III/IV hemorrhage
time frame: 30 days
description: All patients will be monitored for hemorrhage probably or definitely related to defibrotide.
measure: Number of patients with improvement in clinical signs of Acute Chest Syndrome
time frame: 30 days
description: Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of ACS
arm group label: Interventional
arm group type: Experimental
description: Defibrotide 6.25 mg/kg IV q6h
intervention type: Drug
intervention name: Defibrotide
description: Defibrotide 6.25 mg/kg IV q6h up to 7 days
arm group label: Interventional
other name: Defitelio®
criteria: 
gender: All
minimum age: 2 Years
maximum age: 40 Years
healthy volunteers: No
last name: Mitchell S Cairo, MD
role: Principal Investigator
affiliation: New York Medical College
facility: 
country: United States
responsible party type: Sponsor
mesh term: Acute Chest Syndrome
mesh term: Anemia, Sickle Cell
mesh term: Syndrome
mesh term: Defibrotide