<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/NCT02257684</url>
  </required_header>
  <id_info>
    <org_study_id>13-011</org_study_id>
    <nct_id>NCT02257684</nct_id>
  </id_info>
  <brief_title>A Dose Confirmation and Pharmacokinetic Study of Pegcrisantaspase Administered as Intravenous (IV) Infusion in Children and Young Adults With Acute Lymphoblastic Leukemia (ALL) /Lymphoblastic Lymphoma (LBL). Following Hypersensitivity to Pegaspargase (Oncaspar)</brief_title>
  <official_title>A Dose Confirmation and Pharmacokinetic Study of Pegcrisantaspase Administered as Intravenous (IV) Infusion in Children and Young Adults With Acute Lymphoblastic Leukemia (ALL) /Lymphoblastic Lymphoma (LBL). Following Hypersensitivity to Pegaspargase (Oncaspar)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Jazz Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Jazz Pharmaceuticals</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to evaluate the effectiveness,safety, and dosage of&#xD;
      pegcrisantaspase in patients with Acute Lymphoblastic Leukemia (ALL) / Lymphoblastic Lymphoma&#xD;
      (LBL).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The purpose of the study was to assess the response rate in children and young adults with&#xD;
      ALL/LBL and hypersensitivity to pegaspargase defined as the proportion of subjects having a&#xD;
      serum asparaginase activity (SAA) level of ≥ 0.1 IU/mL 14 days following the first IV&#xD;
      pegcrisantaspase dose in Course 1. Also, to assess the safety of IV pegcrisantaspase therapy&#xD;
      in children and young adults with ALL/LBL with hypersensitivity to pegaspargase.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Sponsor decision&#xD;
  </why_stopped>
  <start_date>September 2014</start_date>
  <completion_date type="Actual">January 2016</completion_date>
  <primary_completion_date type="Actual">February 2015</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>The Response Rate in Children &amp; Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of &gt;= 0.1 IU/mL Following the First IV Dose in Course 1</measure>
    <time_frame>15 days during Course 1</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>The Serum Asparaginase Activity 14 Days After the First Infusion of Study Drug and the Adverse Events in All Participants.</measure>
    <time_frame>1 Year</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>The Pharmakokinetic (PK) Profile of IV Pegcrisantaspase in Children and Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase. Pharmakokinetic Profiles to be Assessed Are: Half Life, Elimination Rate, Tmax, Cmax, AUC.</measure>
    <time_frame>14 Days</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The SAA Levels Over Time Following Repeated Administration in Children and Young Adults ALL/LBL and Hypersensitivity to Pegaspargase</measure>
    <time_frame>30 Days</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The Immunogenicity of IV Pegcristaspase by Testing Anti-pegcrisantaspase and Anti-PEG Binding and Neutralizing Antibodies</measure>
    <time_frame>30 Days</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">4</enrollment>
  <condition>Acute Lymphoblastic Leukemia</condition>
  <condition>Lymphoblastic Lymphoma</condition>
  <arm_group>
    <arm_group_label>Pegcrisantaspase</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>pegcrisantaspase</intervention_name>
    <arm_group_label>Pegcrisantaspase</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Have a diagnosis of ALL/LBL&#xD;
&#xD;
          2. Be &gt; 1 to ≤ 21 years of age at study enrollment&#xD;
&#xD;
          3. Have had a ≥ Grade 2 allergic reaction (Common Terminology Criteria for Adverse Events&#xD;
             [CTCAE] v4.03) to pegaspargase&#xD;
&#xD;
          4. Have ≥ 1 dose(s) of pegaspargase remaining in his/her treatment plan&#xD;
&#xD;
          5. Have a documented SAA level that is below the limit of quantitation per the analytical&#xD;
             method.&#xD;
&#xD;
          6. Subjects must have, in the opinion of the investigator, fully recovered from prior&#xD;
             allergic reaction to pegaspargase. Subjects must have completed antihistamine,&#xD;
             epinephrine, and/or corticosteroid treatment for the allergic reaction ≥ 24 hours&#xD;
             prior to pegcrisantaspase administration.&#xD;
&#xD;
          7. Subjects must have a performance status corresponding to:&#xD;
&#xD;
               -  Karnofsky ≥ 50 (for subjects &gt; 16 years of age)&#xD;
&#xD;
               -  Lansky ≥ 50 (for subjects ≤ 16 years of age)&#xD;
&#xD;
          8. Adequate Renal Function Defined as:&#xD;
&#xD;
               -  Creatinine clearance or radioisotope GFR ≥ 70 mL/min/1.73 m2 or&#xD;
&#xD;
               -  A serum creatinine based on age/gender as follows:&#xD;
&#xD;
             Age Maximum Serum Creatinine (mg/dL) Male Female 1 to &lt; 2 years 0.6 0.6 2 to &lt; 6 years&#xD;
             0.8 0.8 6 to &lt; 10 years 1 1 10 to &lt; 13 years 1.2 1.2 13 to &lt; 16 years 1.5 1.4&#xD;
&#xD;
             ≥ 16 years 1.7 1.4&#xD;
&#xD;
             The threshold creatinine values in this table were derived from the Schwartz formula&#xD;
             for estimating GFR (Schwartz &amp; Gauthier 1985) utilizing child length and stature data&#xD;
             published by the CDC.&#xD;
&#xD;
          9. Adequate Liver Function defined as:&#xD;
&#xD;
             Bilirubin levels ≤ 2.5x ULN for age, and Direct (conjugated) Bilirubin &lt; 0.5 mg/dLSGPT&#xD;
             (ALT) ≤ 225 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L.&#xD;
&#xD;
         10. Subjects who are sexually active must agree to use a medically acceptable method of&#xD;
             contraception throughout the entire study period and for 4 weeks after the study is&#xD;
             completed. Medically acceptable methods of contraception that may be used by the&#xD;
             subject and/or the partner include abstinence, birth control pills or patches,&#xD;
             diaphragm and spermicide, condom and vaginal spermicide, surgical sterilization,&#xD;
             postmenopausal, vasectomy (&gt;6 months prior to baseline), and progestin implant or&#xD;
             injection.&#xD;
&#xD;
         11. Able to understand and to sign a written informed consent. All subjects and/or their&#xD;
             parent or a legally authorized representative must sign a written informed consent.&#xD;
             Assent, when appropriate, will be obtained according to institutional guidelines.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Have previously received Erwinia asparaginase&#xD;
&#xD;
          2. Are receiving another investigational agent or will receive an investigational agent&#xD;
             in subsequent phases of protocol therapy that include asparaginase&#xD;
&#xD;
          3. Have a history of ≥ Grade 3 pancreatitis (CTCAE v4.03)&#xD;
&#xD;
          4. Prior history of a CNS thrombotic event or ≥ Grade 3 (CTCAE v4.03) thrombotic event,&#xD;
             excluding catheter-associated clots&#xD;
&#xD;
          5. Prior history of asparaginase-associated ≥ Grade 3 (CTCAE v4.03) hemorrhagic event or&#xD;
             asparaginase-associated thrombus requiring anticoagulation therapy&#xD;
&#xD;
          6. Blood triglyceride levels &gt; 500 mg/dL or &gt; 5.6 mmol/L&#xD;
&#xD;
          7. Hyperglycemia requiring insulin therapy&#xD;
&#xD;
          8. QTc prolongation ≥ 550 msec&#xD;
&#xD;
          9. Subjects who in the opinion of the investigator may not be able to comply with the&#xD;
             safety monitoring requirements of the study&#xD;
&#xD;
         10. Subjects who have any serious active disease or co-morbid medical condition (according&#xD;
             to investigator's decision), or psychiatric illness that would prevent the subject&#xD;
             from signing the informed consent form, assent form or informed consent form by&#xD;
             parents, pending institutional requirements, or per investigator's opinion, would&#xD;
             prevent the subject from completing one course of pegcrisantaspase.&#xD;
&#xD;
        Pregnant or lactating females or females of childbearing potential not willing to use an&#xD;
        adequate method of birth control for the duration of the study. Female subjects who are&#xD;
        lactating who do not agree to stop breast-feeding.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Year</minimum_age>
    <maximum_age>21 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Roman Skowronski, MD, PhD</last_name>
    <role>Study Director</role>
    <affiliation>Jazz Pharmaceuticals</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Phoenix Children's Hospital</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85016</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Arkansas Children's Hospital</name>
      <address>
        <city>Little Rock</city>
        <state>Arkansas</state>
        <zip>72202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Miller Children's Hospital</name>
      <address>
        <city>Long Beach</city>
        <state>California</state>
        <zip>9080</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Los Angeles</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90027-6016</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital Central California</name>
      <address>
        <city>Madera</city>
        <state>California</state>
        <zip>93636</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kaiser Permanente</name>
      <address>
        <city>Oakland</city>
        <state>California</state>
        <zip>94611</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Orange County</name>
      <address>
        <city>Orange</city>
        <state>California</state>
        <zip>92868</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UCSF Benioff Children's Hospital / UCSF Benioff Children's Hospital</name>
      <address>
        <city>San Francisco</city>
        <state>California</state>
        <zip>94143</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital Colorado</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's National Medical Center Center for Cancer &amp; Blood Disorders</name>
      <address>
        <city>Washington</city>
        <state>District of Columbia</state>
        <zip>20010</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nemours Children's Clinic</name>
      <address>
        <city>Jacksonville</city>
        <state>Florida</state>
        <zip>32207</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>All Children's Hospital</name>
      <address>
        <city>St. Petersburg</city>
        <state>Florida</state>
        <zip>33701</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ann &amp; Robert H. Lurie Children's Hospital of Chicago</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60611</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Riley Hospital for Children / Indiana University</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kosair Children's Hospital</name>
      <address>
        <city>Louisville</city>
        <state>Kentucky</state>
        <zip>40202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital Main Campus</name>
      <address>
        <city>New Orleans</city>
        <state>Louisiana</state>
        <zip>70118</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Johns Hopkins University</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21218</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>C.S. Mott / University of Michigan</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109-5718</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Wayne State University c/o Children's Hospital of Michigan</name>
      <address>
        <city>Detroit</city>
        <state>Michigan</state>
        <zip>48201</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Minnesota Medical Center - Fairview</name>
      <address>
        <city>Minneaplois</city>
        <state>Minnesota</state>
        <zip>55455</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospitals &amp; Clinics of Minnesota</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55404</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Mississippi Medical Center</name>
      <address>
        <city>Jackson</city>
        <state>Mississippi</state>
        <zip>39216</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Mercy Hospital - Kansas City</name>
      <address>
        <city>Kansas City</city>
        <state>Missouri</state>
        <zip>64108</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Washington University School of Medicine</name>
      <address>
        <city>St. Louis</city>
        <state>Missouri</state>
        <zip>37212</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital &amp; Medical Center of Omaha</name>
      <address>
        <city>Omaha</city>
        <state>Nebraska</state>
        <zip>68114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hackensack University Medical Center</name>
      <address>
        <city>Hackensack</city>
        <state>New Jersey</state>
        <zip>07601</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rutgers Cancer Institute of New Jersey</name>
      <address>
        <city>New Brunswick</city>
        <state>New Jersey</state>
        <zip>08903</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Steven and Alexandra Cohen Children's Medical Center of New York</name>
      <address>
        <city>New Hyde Park</city>
        <state>New York</state>
        <zip>11040</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of North Carolina at Chapel Hill</name>
      <address>
        <city>Chapel Hill</city>
        <state>North Carolina</state>
        <zip>27599</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Carolinas Medical Center, Levine Cancer Institute, Levine Children's Hospital</name>
      <address>
        <city>Charlotte</city>
        <state>North Carolina</state>
        <zip>28203</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cincinnati Children's Hospital Medical</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45229</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nationwide Children's Hospital</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The University of Oklahoma Health Sciences Center</name>
      <address>
        <city>Oklahoma</city>
        <state>Oklahoma</state>
        <zip>73104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Penn State Children's Hospital</name>
      <address>
        <city>Hershey</city>
        <state>Pennsylvania</state>
        <zip>17033</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Bi-Lo Charities Children's Cancer Center</name>
      <address>
        <city>Greenville</city>
        <state>South Carolina</state>
        <zip>29681</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vanderbilt University Ingram Cancer Center</name>
      <address>
        <city>Nashville</city>
        <state>Tennessee</state>
        <zip>37212</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Dell Children's Medical Center</name>
      <address>
        <city>Austin</city>
        <state>Texas</state>
        <zip>78723</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The University of Texas Southwestern Medical Center</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75390</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Texas Children's Hospital / Baylor College of Medicine</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Seattle Children's Hospital</name>
      <address>
        <city>Seatlle</city>
        <state>Washington</state>
        <zip>98105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Wisconsin / American Family Children's Hospital</name>
      <address>
        <city>Madison</city>
        <state>Wisconsin</state>
        <zip>53792</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Wisconsin / Midwest Children's Cancer Center</name>
      <address>
        <city>Milwaukee</city>
        <state>Wisconsin</state>
        <zip>53226</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>March 2017</verification_date>
  <study_first_submitted>September 22, 2014</study_first_submitted>
  <study_first_submitted_qc>October 2, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 6, 2014</study_first_posted>
  <results_first_submitted>January 5, 2017</results_first_submitted>
  <results_first_submitted_qc>January 5, 2017</results_first_submitted_qc>
  <results_first_posted type="Actual">February 23, 2017</results_first_posted>
  <last_update_submitted>March 6, 2017</last_update_submitted>
  <last_update_submitted_qc>March 6, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">April 4, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>ALL</keyword>
  <keyword>LBL</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Lymphoma</mesh_term>
    <mesh_term>Leukemia</mesh_term>
    <mesh_term>Precursor Cell Lymphoblastic Leukemia-Lymphoma</mesh_term>
    <mesh_term>Leukemia, Lymphoid</mesh_term>
    <mesh_term>Lymphoma, Non-Hodgkin</mesh_term>
    <mesh_term>Hypersensitivity</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Pegcrisantaspase</title>
          <description>IV administration of pegcrisantaspase in Course 1</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="4"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="4"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lack of Efficacy</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Sponsor's Decision</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Pegcrisantaspase</title>
          <description>pegcrisantaspase</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="4"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="12.95" lower_limit="7.8" upper_limit="20.3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>The Response Rate in Children &amp; Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of &gt;= 0.1 IU/mL Following the First IV Dose in Course 1</title>
        <time_frame>15 days during Course 1</time_frame>
        <population>Only 1 of the first 4 patients dosed achieved the predefined serum asparaginase activity (SAA) level above the 0.1 IU/mL therapeutic threshold 14 days following the first IV pegcrisantaspase dose in Course 1 (Primary Objective of the study).</population>
        <group_list>
          <group group_id="O1">
            <title>Pegcrisantaspase</title>
            <description>IV administration of pegcrisantaspase in Course 1</description>
          </group>
        </group_list>
        <measure>
          <title>The Response Rate in Children &amp; Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of &gt;= 0.1 IU/mL Following the First IV Dose in Course 1</title>
          <population>Only 1 of the first 4 patients dosed achieved the predefined serum asparaginase activity (SAA) level above the 0.1 IU/mL therapeutic threshold 14 days following the first IV pegcrisantaspase dose in Course 1 (Primary Objective of the study).</population>
          <units>SAA Level IU/mL</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Course 1; Day 0 (Screening)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.013"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Course 1; Day 1 (Pre-dose)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.013"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Course 1; Day 1 (1 hour post)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.50"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Course 1; Day 1 (3 hour post)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.44"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Course 1; Day 1 (5 hour post)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.41"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Course 1; Day 2 (Visit 2)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.36"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Course 1; Day 8 (Visit 3)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.28"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Course 1; Day 11 (Visit 4)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.24"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Course 1; Day 15 (Visit 5)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.26"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>The Serum Asparaginase Activity 14 Days After the First Infusion of Study Drug and the Adverse Events in All Participants.</title>
        <time_frame>1 Year</time_frame>
        <population>Not Applicable, as the study was terminated before this endpoint was analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Pegcrisantaspase</title>
            <description>IV administration of pegcrisantaspase in Course 1</description>
          </group>
        </group_list>
        <measure>
          <title>The Serum Asparaginase Activity 14 Days After the First Infusion of Study Drug and the Adverse Events in All Participants.</title>
          <population>Not Applicable, as the study was terminated before this endpoint was analyzed.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>The Pharmakokinetic (PK) Profile of IV Pegcrisantaspase in Children and Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase. Pharmakokinetic Profiles to be Assessed Are: Half Life, Elimination Rate, Tmax, Cmax, AUC.</title>
        <time_frame>14 Days</time_frame>
        <population>Not Applicable, as the study was terminated before this endpoint was analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Pegcrisantaspase</title>
            <description>IV administration of pegcrisantaspase in Course 1</description>
          </group>
        </group_list>
        <measure>
          <title>The Pharmakokinetic (PK) Profile of IV Pegcrisantaspase in Children and Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase. Pharmakokinetic Profiles to be Assessed Are: Half Life, Elimination Rate, Tmax, Cmax, AUC.</title>
          <population>Not Applicable, as the study was terminated before this endpoint was analyzed.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>The SAA Levels Over Time Following Repeated Administration in Children and Young Adults ALL/LBL and Hypersensitivity to Pegaspargase</title>
        <time_frame>30 Days</time_frame>
        <population>Not Applicable, as the study was terminated before this endpoint was analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Pegcrisantaspase</title>
            <description>IV administration of pegcrisantaspase in Course 1</description>
          </group>
        </group_list>
        <measure>
          <title>The SAA Levels Over Time Following Repeated Administration in Children and Young Adults ALL/LBL and Hypersensitivity to Pegaspargase</title>
          <population>Not Applicable, as the study was terminated before this endpoint was analyzed.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>The Immunogenicity of IV Pegcristaspase by Testing Anti-pegcrisantaspase and Anti-PEG Binding and Neutralizing Antibodies</title>
        <time_frame>30 Days</time_frame>
        <population>Not Applicable, as the study was terminated before this endpoint was analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Pegcrisantaspase</title>
            <description>IV administration of pegcrisantaspase in Course 1</description>
          </group>
        </group_list>
        <measure>
          <title>The Immunogenicity of IV Pegcristaspase by Testing Anti-pegcrisantaspase and Anti-PEG Binding and Neutralizing Antibodies</title>
          <population>Not Applicable, as the study was terminated before this endpoint was analyzed.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <group_list>
        <group group_id="E1">
          <title>Pegcrisantaspase</title>
          <description>IV administration of pegcrisantaspase in Course 1</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA 17.1</default_vocab>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Febrile neutropenia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
              <event>
                <sub_title>Pancytopenia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Immune system disorders</title>
            <event_list>
              <event>
                <sub_title>Anaphylactic reaction</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Infusion related reaction</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Neoplasms benign, malignant and unspecified (incl cysts and polyps)</title>
            <event_list>
              <event>
                <sub_title>T-cell lymphoma recurrent</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <default_vocab>MedDRA 17.1</default_vocab>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Anaemia</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="4"/>
              </event>
              <event>
                <sub_title>Neutropenia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Eye disorders</title>
            <event_list>
              <event>
                <sub_title>Periorbital oedema</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Mucosal inflammation</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Alanine aminotransferase increased</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
              <event>
                <sub_title>Platelet count decreased</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Muscle spasms</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Nasal congestion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
              <event>
                <sub_title>Rhinorrhoea</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Alopecia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="4"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <limitations_and_caveats>Only 1 patient achieved the predefined SAA level above the 0.1 IU/mL therapeutic threshold 14 days following IV pegcrisantaspase dose in Course 1. Therefore, the corresponding PK parameters for repeated administration were not calculated.</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Head of Clinical Development</name_or_title>
      <organization>Jazz Pharmaceuticals</organization>
      <phone>650-496-3777</phone>
    </point_of_contact>
  </clinical_results>
</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/NCT02257684
org study id: 13-011
nct id: NCT02257684
lead sponsor: 
has dmc: Yes
The purpose of this study is to evaluate the effectiveness,safety, and dosage of
      pegcrisantaspase in patients with Acute Lymphoblastic Leukemia (ALL) / Lymphoblastic Lymphoma
      (LBL).
The purpose of the study was to assess the response rate in children and young adults with
      ALL/LBL and hypersensitivity to pegaspargase defined as the proportion of subjects having a
      serum asparaginase activity (SAA) level of ≥ 0.1 IU/mL 14 days following the first IV
      pegcrisantaspase dose in Course 1. Also, to assess the safety of IV pegcrisantaspase therapy
      in children and young adults with ALL/LBL with hypersensitivity to pegaspargase.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: The Response Rate in Children & Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase Defined as the Proportion of Subjects Having a Serum Asparaginase Activity (SAA) Level of >= 0.1 IU/mL Following the First IV Dose in Course 1
time frame: 15 days during Course 1
measure: The Serum Asparaginase Activity 14 Days After the First Infusion of Study Drug and the Adverse Events in All Participants.
time frame: 1 Year
measure: The Pharmakokinetic (PK) Profile of IV Pegcrisantaspase in Children and Young Adults With ALL/LBL and Hypersensitivity to Pegaspargase. Pharmakokinetic Profiles to be Assessed Are: Half Life, Elimination Rate, Tmax, Cmax, AUC.
time frame: 14 Days
measure: The SAA Levels Over Time Following Repeated Administration in Children and Young Adults ALL/LBL and Hypersensitivity to Pegaspargase
time frame: 30 Days
measure: The Immunogenicity of IV Pegcristaspase by Testing Anti-pegcrisantaspase and Anti-PEG Binding and Neutralizing Antibodies
time frame: 30 Days
arm group label: Pegcrisantaspase
arm group type: Experimental
intervention type: Drug
intervention name: pegcrisantaspase
arm group label: Pegcrisantaspase
criteria: 
gender: All
minimum age: 1 Year
maximum age: 21 Years
healthy volunteers: No
last name: Roman Skowronski, MD, PhD
role: Study Director
affiliation: Jazz Pharmaceuticals
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: United States
responsible party type: Sponsor
mesh term: Lymphoma
mesh term: Leukemia
mesh term: Precursor Cell Lymphoblastic Leukemia-Lymphoma
mesh term: Leukemia, Lymphoid
mesh term: Lymphoma, Non-Hodgkin
mesh term: Hypersensitivity
sharing ipd: No
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
limitations and caveats: Only 1 patient achieved the predefined SAA level above the 0.1 IU/mL therapeutic threshold 14 days following IV pegcrisantaspase dose in Course 1. Therefore, the corresponding PK parameters for repeated administration were not calculated.
point of contact: 