<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/NCT01724580</url>
  </required_header>
  <id_info>
    <org_study_id>14559</org_study_id>
    <secondary_id>I4V-MC-JAGA</secondary_id>
    <nct_id>NCT01724580</nct_id>
  </id_info>
  <brief_title>Compassionate Use Protocol for the Treatment of Autoinflammatory Syndromes</brief_title>
  <official_title>Compassionate Use Treatment Protocol I4V-MC-JAGA: Treatment of Conditions Expected to Benefit From JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI and Severe Juvenile Dermatomyositis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Eli Lilly and Company</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Eli Lilly and Company</source>
  <brief_summary>
    <textblock>
      The Requesting Physician/Investigator contacts Lilly when, based on their medical opinion, a&#xD;
      patient meets the criteria for inclusion in the expanded access program.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>No longer available</overall_status>
  <study_type>Expanded Access</study_type>
  <has_expanded_access>No</has_expanded_access>
  <condition>Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE)</condition>
  <condition>Juvenile Dermatomyositis (JDM)</condition>
  <condition>Stimulator of Interferon Genes (STING)-Associated Vasculopathy With Onset During Infancy (SAVI)</condition>
  <condition>Aicardi-Goutières Syndrome (AGS)</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Baricitinib</intervention_name>
    <description>Administered orally</description>
    <other_name>LY3009104</other_name>
    <other_name>INCB 028050</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Are ≥17.5 months of age (or are ≥6 months of age with Aicardi-Goutières Syndrome&#xD;
             [AGS]). Participants younger than 17.5 months of age can be considered for enrollment&#xD;
             after discussion with the sponsor.&#xD;
&#xD;
          -  Have systemic signs and symptoms of inflammation as manifested by the presence of two&#xD;
             or more of the following symptoms: rash, fever, musculoskeletal pain, headache,&#xD;
             fatigue, weakness, respiratory/breathing symptoms, or ulcers/ischemic lesions.&#xD;
&#xD;
          -  Have an average daily Diary Score of ≥0.5 (CANDLE Diary; used also for CANDLE-related&#xD;
             conditions, AGS Diary) or ≥1.0 (SAVI) or ≥1.0 exclusive of headache and fever symptoms&#xD;
             (JDM Diary) assessed over at least 2 weeks prior to entry, if available. Otherwise,&#xD;
             participants can complete the diary after study consent is signed during the screening&#xD;
             period and meet the inclusion criteria for enrollment into the study.&#xD;
&#xD;
          -  Are ≥8.5 kilogram (kg) in body weight. Participants weighing less than 8.5 kg can be&#xD;
             considered for enrollment after discussion with the sponsor.&#xD;
&#xD;
          -  Have been previously treated with at least 1 biologic therapy and, in the opinion of&#xD;
             the investigator, did not respond or are no longer responding to therapy. If the&#xD;
             participant has been diagnosed with CANDLE, Nakajo-Nishimura Syndrome (NNS), SAVI,&#xD;
             AGS, or an equivalent syndrome, the need for previous biologic therapy is not&#xD;
             required.&#xD;
&#xD;
          -  Require treatment with oral corticosteroids (≥0.15 milligrams per kilogram per day&#xD;
             [mg/kg/d] of prednisone or its equivalent) for control of systemic signs and symptoms&#xD;
             of their chronic inflammatory disease for at least 2 weeks prior to study entry, or in&#xD;
             the opinion of the investigator, have failed an adequate course of steroids. Treatment&#xD;
             with or failure of treatment with steroids is not required for participants with AGS&#xD;
             or a confirmed genetic diagnosis of CANDLE or SAVI.&#xD;
&#xD;
          -  Have had previous documented elevations in acute-phase reactants (for example, high&#xD;
             sensitivity C-reactive protein) considered to be the result of the inflammatory&#xD;
             disease (participants with CANDLE or CANDLE-related conditions only).&#xD;
&#xD;
          -  Have the ability to provide informed consent or have legal representative who is&#xD;
             willing and able to provide written informed consent, provided that assent is obtained&#xD;
             from participants at an age-appropriate level.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Have received an immunosuppressive biologic agent/monoclonal antibody within 4&#xD;
             half-lives prior to entry, for example, anakinra (4 half-lives=18 hours); etanercept&#xD;
             (4 half-lives=18 days); infliximab; adalimumab (4 half-lives=36 days); use of&#xD;
             intravenous immune globulin (IVIg) is permitted.&#xD;
&#xD;
          -  Are pregnant or nursing at the time of entry.&#xD;
&#xD;
          -  Are females of childbearing potential (women &gt;12 or who have had at least 1 menstrual&#xD;
             period regardless of age) who are sexually active and who do not agree to use 2 forms&#xD;
             of highly effective methods of birth control or remain abstinent during the study and&#xD;
             for at least 28 days following the last dose of investigational product.&#xD;
&#xD;
          -  Are sexually active males who do not agree to use 2 forms of highly effective birth&#xD;
             control with female partners of childbearing potential or remain abstinent during the&#xD;
             study and for at least 28 days following the last dose of investigational product.&#xD;
&#xD;
          -  Have had symptomatic herpes zoster infection within 12 weeks prior to entry or during&#xD;
             the screening period.&#xD;
&#xD;
          -  Have a history of disseminated/complicated herpes zoster (for example, multidermatomal&#xD;
             involvement, ophthalmic zoster, central nervous system [CNS] involvement, postherpetic&#xD;
             neuralgia).&#xD;
&#xD;
          -  Have evidence of active infection, at the time of entry or during the screening&#xD;
             period, that in the opinion of the investigator, would pose an unacceptable risk for&#xD;
             participating in the study.&#xD;
&#xD;
          -  Have a history of active hepatitis B (HBV), hepatitis C (HCV), or human&#xD;
             immunodeficiency virus (HIV).&#xD;
&#xD;
          -  Have documented high titer autoantibodies suggestive clinically of autoimmune diseases&#xD;
             other than severe JDM.&#xD;
&#xD;
          -  Are immunocompromised and, in the opinion of the investigator, are at an unacceptable&#xD;
             risk for participating in the study.&#xD;
&#xD;
          -  Have had a serious systemic or local infection (including an infectious&#xD;
             mononucleosis-like illness or herpes zoster) within 12 weeks prior to entry or during&#xD;
             the screening period.&#xD;
&#xD;
          -  Have been exposed to a live vaccine within 12 weeks prior to entry or are expected to&#xD;
             need/receive a live vaccine (including herpes zoster vaccination) during the course of&#xD;
             the study.&#xD;
&#xD;
        Note: Investigators should review the vaccination status of the participants and follow the&#xD;
        local guidelines for vaccination with nonlive vaccines intended to prevent infectious&#xD;
        disease prior to entering participants into the study.&#xD;
&#xD;
          -  Have had household contact with a person with active tuberculosis (TB) and did not&#xD;
             receive appropriate and documented prophylaxis for TB.&#xD;
&#xD;
          -  Have a serious and/or unstable illness that, in the opinion of the investigator, poses&#xD;
             an unacceptable risk for the participant's participation in the study.&#xD;
&#xD;
          -  Have an estimated glomerular filtration rate (eGFR) based on the most recent available&#xD;
             serum creatinine of &lt;40 milliliters/minute/1.73 per square meter.&#xD;
&#xD;
          -  Have or have had a history of lymphoproliferative disease; or signs or symptoms&#xD;
             suggestive of possible lymphoproliferative disease, or active primary or recurrent&#xD;
             malignant disease; or been in remission from clinically significant malignancy for &lt;5&#xD;
             years.&#xD;
&#xD;
        Note: Participants with resolved cervical dysplasia, or no more than 3 successfully treated&#xD;
        basal-cell carcinoma of the skin, may participate in this study.&#xD;
&#xD;
          -  Have a history of chronic alcohol abuse or intravenous (IV) drug abuse within the 2&#xD;
             years prior to entry.&#xD;
&#xD;
          -  Are unable or unwilling to make themselves available for the duration of the study&#xD;
             and/or are unwilling to follow study restrictions/procedures.&#xD;
&#xD;
          -  Are currently enrolled in, or discontinued within the last 30 days from a clinical&#xD;
             trial involving an investigational product or non-approved use of a drug or device&#xD;
             (other than the investigational product used in this study), or concurrently enrolled&#xD;
             in any other type of medical research judged not to be scientifically or medically&#xD;
             compatible with this study.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Months</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST)</last_name>
    <role>Study Director</role>
    <affiliation>Eli Lilly and Company</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Translational Autoinflammatory Disease (TADS)</name>
      <address>
        <city>Bethesda</city>
        <state>Maryland</state>
        <zip>20892</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>Great Ormond Street Hospital</name>
      <address>
        <city>London</city>
        <zip>WC1N 1EH</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>August 1, 2023</verification_date>
  <study_first_submitted>November 5, 2012</study_first_submitted>
  <study_first_submitted_qc>November 9, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 12, 2012</study_first_posted>
  <last_update_submitted>August 8, 2023</last_update_submitted>
  <last_update_submitted_qc>August 8, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">August 9, 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>Dermatomyositis</mesh_term>
    <mesh_term>Lipodystrophy</mesh_term>
    <mesh_term>Skin Diseases</mesh_term>
    <mesh_term>Syndrome</mesh_term>
    <mesh_term>Hyperthermia</mesh_term>
    <mesh_term>Fever</mesh_term>
  </condition_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/NCT01724580
org study id: 14559
secondary id: I4V-MC-JAGA
nct id: NCT01724580
lead sponsor: 
The Requesting Physician/Investigator contacts Lilly when, based on their medical opinion, a
      patient meets the criteria for inclusion in the expanded access program.
intervention type: Drug
intervention name: Baricitinib
description: Administered orally
other name: LY3009104
other name: INCB 028050
criteria: 
gender: All
minimum age: 6 Months
maximum age: N/A
healthy volunteers: No
last name: Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST)
role: Study Director
affiliation: Eli Lilly and Company
facility: 
facility: 
facility: 
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Dermatomyositis
mesh term: Lipodystrophy
mesh term: Skin Diseases
mesh term: Syndrome
mesh term: Hyperthermia
mesh term: Fever