<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/NCT02968108</url>
  </required_header>
  <id_info>
    <org_study_id>CR108233</org_study_id>
    <secondary_id>CNTO1275CRD1001</secondary_id>
    <secondary_id>2016-001956-22</secondary_id>
    <nct_id>NCT02968108</nct_id>
  </id_info>
  <brief_title>A Pharmacokinetic Study of Ustekinumab in Pediatric Subjects With Moderately to Severely Active Crohn's Disease</brief_title>
  <acronym>STELARA</acronym>
  <official_title>A Randomized Double-blind Pharmacokinetic Study of Ustekinumab in Pediatric Subjects With Moderately to Severely Active Crohn's Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Janssen Research &amp; Development, LLC</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Janssen Research &amp; Development, LLC</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>
      The purpose of this study is to evaluate the pharmacokinetics (PK) of ustekinumab in subjects&#xD;
      from 2 through less than (&lt;) 18 years old in the USA, or 6 through less than (&lt;) 18 years old&#xD;
      in other countries and determine if it is similar to that observed in adults with moderately&#xD;
      to severely active Crohn's disease (CD). Also to assess the safety, immunogenicity and&#xD;
      efficacy of ustekinumab in the treatment of moderately to severely active CD. The main part&#xD;
      of the study continues to Week 16, at which point all subjects who are receiving benefit from&#xD;
      ustekinumab maintenance therapy (as determined by the investigator) are eligible to enter the&#xD;
      long-term extension (LTE) and continue to receive ustekinumab. The study extension ends at&#xD;
      Week 268 or upon availability of the LTE basket study (CNTO1275ISD3001) whichever occurs&#xD;
      first. If participants do not consent/assent to the LTE basket study, they will continue&#xD;
      safety follow-up for approximately 20 weeks after the last study agent administration.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">January 18, 2017</start_date>
  <completion_date type="Actual">March 18, 2022</completion_date>
  <primary_completion_date type="Actual">September 19, 2018</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Serum Ustekinumab Concentrations Over Time</measure>
    <time_frame>Up to Week 16</time_frame>
    <description>Serum samples will be collected to measure seum concentrations of Ustekinumab.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Clinical Response as Measured by the Pediatric Crohn's Disease Activity Index (PCDAI) Score</measure>
    <time_frame>Week 6</time_frame>
    <description>Clinical Response is defined as greater than or equal to (&gt;=) 15-point reduction from baseline in the total Pediatric Crohn's Disease Activity Index (PCDAI) score, accompanied by a total PDCAI score of less than or equal to (&lt;=) 30 points. PCDAI is an index used to measure disease activity of pediatric patients with Crohn's Disease assessing abdominal pain, stool frequency, patient functioning, hematocrit, erythrocyte sedimentation rate, albumin, weight, height, abdominal tenderness or mass, perirectal disease, and extraintestinal manifestations. It ranges from 0 to 100; higher scores indicate more active disease.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Clinical Remission as Measured by the Pediatric Crohn's Disease Activity Index (PCDAI) Score</measure>
    <time_frame>Week 8</time_frame>
    <description>Clinical remission is defined as PCDAI score of less than or equal to (&lt;=) 10. PCDAI is an index used to measure disease activity of pediatric patients with Crohn's Disease assessing abdominal pain, stool frequency, patient functioning, hematocrit, erythrocyte, sedimentation rate, albumin, weight, height, abdominal tenderness or mass, perirectal disease, and extraintestinal manifestations. It ranges from 0 to 100; higher scores indicate more active disease.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">45</enrollment>
  <condition>Crohn Disease</condition>
  <arm_group>
    <arm_group_label>Group1: Ustekinumab Dose Regimen 1</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Subjects will receive a single intravenous (IV) induction dose of 3 milligram per kilogram (mg/kg) for subjects less than &lt; 40 kilogram (kg) or 130 milligram (mg) for subjects greater than or equal to &gt;= 40 kg at Week 0 followed by subcutaneous (SC) maintenance dose of 2 mg/kg for subjects &lt; 40 kg or 90 mg for subjects &gt;= 40 kg at week 8.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Group2: Ustekinumab Dose Regimen 2</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Subjects will receive a single Intravenous (IV) dose of 9 mg/kg for subjects &lt;40 kg or 390 mg for subjects &gt;= 40 kg at Week 0 followed by SC maintenance dose of 2 mg/kg for subjects &lt;40 kg or 90 mg for subjects &gt;= 40 kg at week 8.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Ustekinumab</intervention_name>
    <description>Subjects will receive a single IV administration of ustekinumab (3 mg/kg for subjects &lt;40 kg or 130 mg for subjects &gt;= 40 kg in Group 1 and 9 mg/kg for subjects &lt; 40 kg or 390 mg for subjects &gt;= 40 kg in group 2) at week 0 followed by SC administration of ustekinumab (2 mg/kg for subjects &lt; 40 kg or 90 mg for subjects &gt;= 40 kg at Week 8.</description>
    <arm_group_label>Group1: Ustekinumab Dose Regimen 1</arm_group_label>
    <arm_group_label>Group2: Ustekinumab Dose Regimen 2</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Be a pediatric subject 2 to less than (&lt;) 18 years old in the US, 6 to &lt;18 years old&#xD;
             elsewhere, of either gender with a body weight of greater than or equal to (&gt;=) 10&#xD;
             kilogram (kg)&#xD;
&#xD;
          -  Have Crohn's disease (CD) or fistulizing CD of at least 3 months duration, with active&#xD;
             colitis, ileitis, or ileocolitis, confirmed at any time in the past by radiography,&#xD;
             histology, and/or endoscopy&#xD;
&#xD;
          -  Must have moderately to severely active CD defined by: Baseline pediatric Crohn's&#xD;
             disease activity index (PCDAI) score of greater than (&gt;)30 and at least one of the&#xD;
             following: An abnormal C-reactive protein (CRP) &gt;0.3 milligram per deciliter (mg/dL)&#xD;
             or 3.0 milligram per liter (mg/L) at screening) or fecal calprotectin &gt;250 milligram&#xD;
             per kilogram (mg/kg) at screening or ileocolonoscopy with evidence of active CD&#xD;
             (defined as ulcerations in the ileum and/or colon) during screening into this study&#xD;
             including at the baseline visit&#xD;
&#xD;
          -  Prior or current medication for CD must include at least 1 of the following: Current&#xD;
             treatment with at least 1 of the following therapies: oral corticosteroids, the&#xD;
             immunomodulators azathioprine, 6-MP, or methotrexate, or currently have or have had a&#xD;
             history of corticosteroid dependency, or have a history of failure to respond to, or&#xD;
             tolerate, at least 1 of the following therapies including oral or IV corticosteroids&#xD;
             or the immunomodulators 6-mercaptopurine, azathioprine, or methotrexate,or have&#xD;
             required more than 3 courses of oral or IV corticosteroids in the past year&#xD;
&#xD;
          -  Have negative stool results for enteric pathogens. Stool studies must include a stool&#xD;
             culture and Clostridium difficile toxin assay. These must have been performed during&#xD;
             screening or the current episode of disease exacerbation as long as the stool studies&#xD;
             were performed within 4 months prior to the first administration of study agent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Has complications of CD such as symptomatic strictures or stenosis, short gut&#xD;
             syndrome, or any other manifestation that might be anticipated to require surgery,&#xD;
             could preclude the use of the PCDAI to assess response to therapy, or would possibly&#xD;
             confound the ability to assess the effect of treatment with ustekinumab&#xD;
&#xD;
          -  Currently has or is suspected to have an abscess. Recent cutaneous and perianal&#xD;
             abscesses are not exclusionary if drained and adequately treated at least 3 weeks&#xD;
             prior to baseline, or 8 weeks prior to baseline for intra-abdominal abscesses,&#xD;
             provided that there is no anticipated need for any further surgery. Participant with&#xD;
             active fistulas may be included if there is no anticipation of a need for surgery and&#xD;
             there are currently no abscesses identified&#xD;
&#xD;
          -  Has had any kind of bowel resection within 6 months or any other intra-abdominal&#xD;
             surgery within 3 months prior to baseline&#xD;
&#xD;
          -  Has a draining (that is (i.e.), functioning) stoma or ostomy&#xD;
&#xD;
          -  Presence or history of any malignancy including presence or history of&#xD;
             lymphoproliferative disease including lymphoma, or signs and symptoms suggestive of&#xD;
             possible lymphoproliferative disease, such as lymphadenopathy of unusual size or&#xD;
             location (example, nodes in the posterior triangle of the neck, infraclavicular,&#xD;
             epitrochlear, or periaortic areas), or clinically significant hepatomegaly or&#xD;
             splenomegaly&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>17 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Janssen Research &amp; Development, LLC Clinical Trial</last_name>
    <role>Study Director</role>
    <affiliation>Janssen Research &amp; Development, LLC</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Cedars-Sinai Medical Center</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90048</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Connecticut Childrens Medical Center</name>
      <address>
        <city>Hartford</city>
        <state>Connecticut</state>
        <zip>06106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Emory University</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Center For Digestive Healthcare, Llc</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30342</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Methodist Medical Center of Illinois</name>
      <address>
        <city>Peoria</city>
        <state>Illinois</state>
        <zip>61602</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mayo Clinic</name>
      <address>
        <city>Rochester</city>
        <state>Minnesota</state>
        <zip>55905</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mount Sinai</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10029</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Childrens Hospital Of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Center For Digestive Health Systems-Greenville</name>
      <address>
        <city>Greenville</city>
        <state>South Carolina</state>
        <zip>29615</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitair Kinderziekenhuis Koningin Fabiola</name>
      <address>
        <city>Brussel</city>
        <zip>1020</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cliniques Universitaires Saint-Luc</name>
      <address>
        <city>Brussel</city>
        <zip>1200</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UZ Gent</name>
      <address>
        <city>Gent</city>
        <zip>9000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UZ Brussel</name>
      <address>
        <city>Jette</city>
        <zip>1090</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UZ Leuven</name>
      <address>
        <city>Leuven</city>
        <zip>3000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Stollery Children's Hospital</name>
      <address>
        <city>Edmonton</city>
        <state>Alberta</state>
        <zip>T6G 2B7</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Western Ontario</name>
      <address>
        <city>London</city>
        <state>Ontario</state>
        <zip>N6A 5W9</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital For Sick Children</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5G 1X8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre Hospitalier Sainte Justine</name>
      <address>
        <city>Montreal</city>
        <state>Quebec</state>
        <zip>H3T 1C5</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>British Columbia Children's Hospital</name>
      <address>
        <city>Vancouver</city>
        <zip>V6H3V4</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hôpital Necker</name>
      <address>
        <city>Paris</city>
        <zip>75015</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hôpital Robert Debré</name>
      <address>
        <city>Paris</city>
        <zip>75019</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Dr. von Haunersches Kinderspital</name>
      <address>
        <city>Munich</city>
        <zip>80337</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>HELIOS Klinikum Wuppertal GmbH</name>
      <address>
        <city>Wuppertal</city>
        <zip>42283</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Instytut Centrum Zdrowia Matki Polki</name>
      <address>
        <city>Lodz</city>
        <zip>93-338</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>WIP Warsaw IBD Point Profesor Kierkus</name>
      <address>
        <city>Warszawa</city>
        <zip>00-728</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu</name>
      <address>
        <city>Wroclaw</city>
        <zip>50-369</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Belgium</country>
    <country>Canada</country>
    <country>France</country>
    <country>Germany</country>
    <country>Poland</country>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2023</verification_date>
  <study_first_submitted>October 24, 2016</study_first_submitted>
  <study_first_submitted_qc>November 16, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 18, 2016</study_first_posted>
  <last_update_submitted>May 1, 2023</last_update_submitted>
  <last_update_submitted_qc>May 1, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">May 3, 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>Crohn Disease</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Ustekinumab</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/NCT02968108
org study id: CR108233
secondary id: CNTO1275CRD1001
secondary id: 2016-001956-22
nct id: NCT02968108
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this study is to evaluate the pharmacokinetics (PK) of ustekinumab in subjects
      from 2 through less than (<) 18 years old in the USA, or 6 through less than (<) 18 years old
      in other countries and determine if it is similar to that observed in adults with moderately
      to severely active Crohn's disease (CD). Also to assess the safety, immunogenicity and
      efficacy of ustekinumab in the treatment of moderately to severely active CD. The main part
      of the study continues to Week 16, at which point all subjects who are receiving benefit from
      ustekinumab maintenance therapy (as determined by the investigator) are eligible to enter the
      long-term extension (LTE) and continue to receive ustekinumab. The study extension ends at
      Week 268 or upon availability of the LTE basket study (CNTO1275ISD3001) whichever occurs
      first. If participants do not consent/assent to the LTE basket study, they will continue
      safety follow-up for approximately 20 weeks after the last study agent administration.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: Serum Ustekinumab Concentrations Over Time
time frame: Up to Week 16
description: Serum samples will be collected to measure seum concentrations of Ustekinumab.
measure: Clinical Response as Measured by the Pediatric Crohn's Disease Activity Index (PCDAI) Score
time frame: Week 6
description: Clinical Response is defined as greater than or equal to (>=) 15-point reduction from baseline in the total Pediatric Crohn's Disease Activity Index (PCDAI) score, accompanied by a total PDCAI score of less than or equal to (<=) 30 points. PCDAI is an index used to measure disease activity of pediatric patients with Crohn's Disease assessing abdominal pain, stool frequency, patient functioning, hematocrit, erythrocyte sedimentation rate, albumin, weight, height, abdominal tenderness or mass, perirectal disease, and extraintestinal manifestations. It ranges from 0 to 100; higher scores indicate more active disease.
measure: Clinical Remission as Measured by the Pediatric Crohn's Disease Activity Index (PCDAI) Score
time frame: Week 8
description: Clinical remission is defined as PCDAI score of less than or equal to (<=) 10. PCDAI is an index used to measure disease activity of pediatric patients with Crohn's Disease assessing abdominal pain, stool frequency, patient functioning, hematocrit, erythrocyte, sedimentation rate, albumin, weight, height, abdominal tenderness or mass, perirectal disease, and extraintestinal manifestations. It ranges from 0 to 100; higher scores indicate more active disease.
arm group label: Group1: Ustekinumab Dose Regimen 1
arm group type: Experimental
description: Subjects will receive a single intravenous (IV) induction dose of 3 milligram per kilogram (mg/kg) for subjects less than < 40 kilogram (kg) or 130 milligram (mg) for subjects greater than or equal to >= 40 kg at Week 0 followed by subcutaneous (SC) maintenance dose of 2 mg/kg for subjects < 40 kg or 90 mg for subjects >= 40 kg at week 8.
arm group label: Group2: Ustekinumab Dose Regimen 2
arm group type: Experimental
description: Subjects will receive a single Intravenous (IV) dose of 9 mg/kg for subjects <40 kg or 390 mg for subjects >= 40 kg at Week 0 followed by SC maintenance dose of 2 mg/kg for subjects <40 kg or 90 mg for subjects >= 40 kg at week 8.
intervention type: Drug
intervention name: Ustekinumab
description: Subjects will receive a single IV administration of ustekinumab (3 mg/kg for subjects <40 kg or 130 mg for subjects >= 40 kg in Group 1 and 9 mg/kg for subjects < 40 kg or 390 mg for subjects >= 40 kg in group 2) at week 0 followed by SC administration of ustekinumab (2 mg/kg for subjects < 40 kg or 90 mg for subjects >= 40 kg at Week 8.
arm group label: Group1: Ustekinumab Dose Regimen 1
arm group label: Group2: Ustekinumab Dose Regimen 2
criteria: 
gender: All
minimum age: 2 Years
maximum age: 17 Years
healthy volunteers: No
last name: Janssen Research & Development, LLC Clinical Trial
role: Study Director
affiliation: Janssen Research & Development, LLC
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country: Belgium
country: Canada
country: France
country: Germany
country: Poland
country: United States
responsible party type: Sponsor
mesh term: Crohn Disease
mesh term: Ustekinumab