<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/NCT01688401</url>
  </required_header>
  <id_info>
    <org_study_id>J11164</org_study_id>
    <secondary_id>NA_00069122</secondary_id>
    <nct_id>NCT01688401</nct_id>
  </id_info>
  <brief_title>Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).</brief_title>
  <official_title>Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Solving Kids' Cancer</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The goal of this pilot study is to determine if intra-arterial (IA) chemotherapy is safe in&#xD;
      the treatment of progressive diffuse intrinsic pontine gliomas (DIPG). IA administration of&#xD;
      the chemotherapeutic agent enhances the regional distribution of the drug, thereby increasing&#xD;
      the local delivered dose while minimizing systemic toxicity. It also provides a treatment&#xD;
      option for these patients at the time of tumor recurrence.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Delivering the chemotherapeutic agent directly to the tumor via the arterial system avoids&#xD;
      the complications and adverse events associated with toxicity from systemic chemotherapy.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">March 8, 2013</start_date>
  <completion_date type="Actual">November 26, 2018</completion_date>
  <primary_completion_date type="Actual">May 2017</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>Other</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Technical safety as determined by number of participants with toxicity</measure>
    <time_frame>60 days</time_frame>
    <description>Number of participants with grades 3-5 intracranial hemorrhage, grades 3-5 stroke, as defined by the Nervous system disorder CTCAE, and requirement of blood transfusion.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Long-term Efficacy as assessed by progression free survival</measure>
    <time_frame>2 years</time_frame>
    <description>Number of months until disease progression.</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Immediate Efficacy as assessed by number of participants with decrease in required steroid dose</measure>
    <time_frame>60 days</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Immediate Efficacy as assessed by number of participants with decrease in tumor size on MRI</measure>
    <time_frame>60 days</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Immediate Efficacy as assessed by number of participants with decrease in the degree of enhancement on MRI</measure>
    <time_frame>60 days</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Immediate Efficacy as assessed by number of participants with improved neurological examination</measure>
    <time_frame>60 days</time_frame>
  </other_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">3</enrollment>
  <condition>Diffuse Intrinsic Pontine Glioma (DIPG)</condition>
  <arm_group>
    <arm_group_label>IA melphalan</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>IA melphalan is administered via the basilar artery.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Melphalan hydrochloride</intervention_name>
    <description>Drug administered intra-arterially (injection in the artery).&#xD;
Standard dose: Cycle 1: 1 mg, intra-arterial (IA) delivery. Cycle 2: 2 mg, intra-arterial (IA) delivery.&#xD;
Duration of treatment: Eight weeks total - two cycles of IA chemotherapy, separated by four weeks.</description>
    <arm_group_label>IA melphalan</arm_group_label>
    <other_name>Alkeran</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Pediatric patients of all ages with progressive DIPG.&#xD;
&#xD;
          -  Consensus following presentation of the case at the multidisciplinary Pediatric&#xD;
             Neuro-Oncology conference, which includes participation of neuro-oncology,&#xD;
             neurosurgery, radiation oncology, interventional neuroradiology and neurology.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Documented hypercoagulable disorders or vasculopathies&#xD;
&#xD;
               -  INR value more than a Grade 1 toxicity by CTCAE v 4.0 criteria (&gt;1 - 1.5 x ULN;&#xD;
                  &gt;1 - 1.5 times above baseline if on anticoagulation).&#xD;
&#xD;
               -  APTT value more than a Grade 1 toxicity by CTCAE v 4.0 criteria (&gt;ULN - 1.5 x&#xD;
                  ULN).&#xD;
&#xD;
          -  Platelets less than 50 x 103/mm3&#xD;
&#xD;
          -  Absolute neutrophil count less than 500/ mm3&#xD;
&#xD;
          -  Pregnancy&#xD;
&#xD;
          -  Documented severe allergic reaction to IV iodinated contrast, specifically&#xD;
             bronchospasm and anaphylaxis.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Month</minimum_age>
    <maximum_age>17 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Monica Pearl, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Johns Hopkins University</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>The Johns Hopkins Hospital</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21287</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>March 2019</verification_date>
  <study_first_submitted>September 10, 2012</study_first_submitted>
  <study_first_submitted_qc>September 14, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 19, 2012</study_first_posted>
  <last_update_submitted>March 26, 2019</last_update_submitted>
  <last_update_submitted_qc>March 26, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">March 28, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>diffuse intrinsic pontine glioma (DIPG)</keyword>
  <keyword>intra-arterial chemotherapy</keyword>
  <keyword>angiography</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Glioma</mesh_term>
    <mesh_term>Diffuse Intrinsic Pontine Glioma</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Melphalan</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/NCT01688401
org study id: J11164
secondary id: NA_00069122
nct id: NCT01688401
lead sponsor: 
collaborator: 
has dmc: Yes
The goal of this pilot study is to determine if intra-arterial (IA) chemotherapy is safe in
      the treatment of progressive diffuse intrinsic pontine gliomas (DIPG). IA administration of
      the chemotherapeutic agent enhances the regional distribution of the drug, thereby increasing
      the local delivered dose while minimizing systemic toxicity. It also provides a treatment
      option for these patients at the time of tumor recurrence.
Delivering the chemotherapeutic agent directly to the tumor via the arterial system avoids
      the complications and adverse events associated with toxicity from systemic chemotherapy.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Other
masking: None (Open Label)
measure: Technical safety as determined by number of participants with toxicity
time frame: 60 days
description: Number of participants with grades 3-5 intracranial hemorrhage, grades 3-5 stroke, as defined by the Nervous system disorder CTCAE, and requirement of blood transfusion.
measure: Long-term Efficacy as assessed by progression free survival
time frame: 2 years
description: Number of months until disease progression.
measure: Immediate Efficacy as assessed by number of participants with decrease in required steroid dose
time frame: 60 days
measure: Immediate Efficacy as assessed by number of participants with decrease in tumor size on MRI
time frame: 60 days
measure: Immediate Efficacy as assessed by number of participants with decrease in the degree of enhancement on MRI
time frame: 60 days
measure: Immediate Efficacy as assessed by number of participants with improved neurological examination
time frame: 60 days
arm group label: IA melphalan
arm group type: Experimental
description: IA melphalan is administered via the basilar artery.
intervention type: Drug
intervention name: Melphalan hydrochloride
description: Drug administered intra-arterially (injection in the artery).
Standard dose: Cycle 1: 1 mg, intra-arterial (IA) delivery. Cycle 2: 2 mg, intra-arterial (IA) delivery.
Duration of treatment: Eight weeks total - two cycles of IA chemotherapy, separated by four weeks.
arm group label: IA melphalan
other name: Alkeran
criteria: 
gender: All
minimum age: 1 Month
maximum age: 17 Years
healthy volunteers: No
last name: Monica Pearl, M.D.
role: Principal Investigator
affiliation: Johns Hopkins University
facility: 
country: United States
responsible party type: Sponsor
mesh term: Glioma
mesh term: Diffuse Intrinsic Pontine Glioma
mesh term: Melphalan