<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/NCT04498871</url>
  </required_header>
  <id_info>
    <org_study_id>CAMN107A2413</org_study_id>
    <nct_id>NCT04498871</nct_id>
  </id_info>
  <brief_title>MAP to Provide Access to Nilotinib, for Patients With HES</brief_title>
  <official_title>Managed Access Program (MAP) to Provide Access to Nilotinib, for Patients With Hypereosinophilic Syndrome (HES)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Novartis Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Novartis</source>
  <brief_summary>
    <textblock>
      The purpose of this program is to allow access to nilotinib for eligible patients diagnosed&#xD;
      with Hypereosinophilic syndrome (HES). The patient's Treating Physician should follow the&#xD;
      suggested treatment guidelines and comply with all local health authority regulations. The&#xD;
      requesting Treating Physician submitted a request for access to drug (often referred to as&#xD;
      Compassionate Use) to Novartis which was reviewed and approved by the medical team&#xD;
      experienced with the drug and indication. Please refer to the latest Investigator's Brochure&#xD;
      (IB) or approved label for overview of drug including: non-clinical and clinical experience,&#xD;
      risk and benefits. Novartis will continue to provide any new safety information to the&#xD;
      Treating Physician as they emerge.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>No longer available</overall_status>
  <study_type>Expanded Access</study_type>
  <has_expanded_access>No</has_expanded_access>
  <expanded_access_info>
    <expanded_access_type_individual>Yes</expanded_access_type_individual>
  </expanded_access_info>
  <condition>Hypereosinophilic Syndrome (HES)</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Nilotinib</intervention_name>
    <description>Nilotinib is recommended to be administered orally on a continuous schedule at a dose of 400 mg twice daily. The patient should be instructed to take each morning dose of nilotinib 2 hours after a light breakfast (e.g., toast and jam), with no oral intake other than water for 1 hour after dosing. It is recommended that the patient take nilotinib doses every 12 hours with a glass of water. The evening dose of nilotinib should be taken at least 2 hours after dinner with no oral intake other than water for 1 hour after dosing.</description>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion criteria&#xD;
&#xD;
          1. ≥ 18 years of age&#xD;
&#xD;
          2. Hypereosinophilic syndrome/chronic eosinophilic leukemia who have a clinical&#xD;
             indication for treatment and meet the following criteria (Vandenberghe, et al 2004):&#xD;
&#xD;
               -  eosinophilia greater than 1500/mm3 which persists greater than 6 months&#xD;
&#xD;
               -  exclusion of other causes of eosinophilia including clonal or abnormal T-cell&#xD;
                  populations, exclusion of reactive eosinophilia, and malignancies or T-cell&#xD;
                  disorders associated with eosinophilia&#xD;
&#xD;
               -  signs and symptoms of organ involvement&#xD;
&#xD;
          3. WHO Performance status of ≤ 2&#xD;
&#xD;
          4. Patient must have the following laboratory values:&#xD;
&#xD;
               -  Potassium ≥ LLN (lower limit of normal) or corrected to within normal limits with&#xD;
                  supplements prior to the first dose of AMN107&#xD;
&#xD;
               -  Total calcium (corrected for serum albumin) ≥ LLN or correctable with supplements&#xD;
&#xD;
               -  Magnesium ≥ LLN or corrected to within normal limits with supplements prior to&#xD;
                  the first dose of AMN107&#xD;
&#xD;
               -  Phosphorus ≥ LLN or correctable with supplements&#xD;
&#xD;
               -  ALT and AST ≤ 2.5 x ULN or ≤ 5.0 x ULN if considered due to tumor&#xD;
&#xD;
               -  Alkaline phosphatase ≤ 2.5 x ULN unless considered due to tumor&#xD;
&#xD;
               -  Serum bilirubin ≤ 1.5 x ULN&#xD;
&#xD;
               -  Serum creatinine ≤ 1.5 x ULN&#xD;
&#xD;
               -  Serum amylase ≤ 1.5 x ULN and serum lipase ≤ 1.5 x ULNWritten patient informed&#xD;
                  consent must be obtained prior to start of treatment.&#xD;
&#xD;
        Written patient informed consent must be obtained prior to start of treatment.&#xD;
&#xD;
        Exclusion criteria&#xD;
&#xD;
        Patients eligible for this Treatment Plan must not meet any of the following criteria:&#xD;
&#xD;
          1. History of hypersensitivity to any drugs or metabolites of similar chemical classes as&#xD;
             nilotinib. Any exclusionary sites of disease (e.g., brain metastases).&#xD;
&#xD;
          2. Impaired cardiac function&#xD;
&#xD;
          3. Patients who have undergone major surgery ≤ 2 weeks prior to Visit 1 or who have not&#xD;
             recovered from side effects of such surgery&#xD;
&#xD;
          4. Known Cytopathologically confirmed CNS infiltration.&#xD;
&#xD;
          5. Use of therapeutic warfarin.&#xD;
&#xD;
          6. Acute or chronic liver or renal disease considered unrelated to tumor.&#xD;
&#xD;
          7. Treatment with any hematopoietic colony-stimulating growth factors ≤ 1 week prior to&#xD;
             starting Nilotinib. Erythropoietin is allowed.&#xD;
&#xD;
          8. Patient who has not recovered from side effects of prior chemotherapy, immunotherapy,&#xD;
             other investigational drugs, wide field radiotherapy, or major surgery. Patient who&#xD;
             has received imatinib &lt; 5 days prior to AMN107 or has not recovered from side effects&#xD;
             of therapy. Hydroxyurea is permitted during the first 28 days of treatment (up to 5&#xD;
             g/day) for a maximum of 7 days.&#xD;
&#xD;
          9. Patient with a history of another primary malignancy that is currently clinically&#xD;
             significant or requires active intervention.&#xD;
&#xD;
         10. Known diagnosis of human immunodeficiency virus (HIV).&#xD;
&#xD;
         11. Known ongoing alcohol or drug abuse&#xD;
&#xD;
         12. Unwillingness or inability to comply with the treatment protocol including returning&#xD;
             for scheduled visits&#xD;
&#xD;
         13. Participation in a prior investigational study within 30 days prior to enrollment or&#xD;
             within 5-half lives of the investigational product, whichever is longer.&#xD;
&#xD;
         14. Pregnant or nursing (lactating) women,&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>99 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <verification_date>September 2021</verification_date>
  <study_first_submitted>July 31, 2020</study_first_submitted>
  <study_first_submitted_qc>July 31, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">August 5, 2020</study_first_posted>
  <last_update_submitted>September 14, 2021</last_update_submitted>
  <last_update_submitted_qc>September 14, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">September 20, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Nilotinib</keyword>
  <keyword>Hypereosinophilic syndrome</keyword>
  <keyword>HES</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hypereosinophilic Syndrome</mesh_term>
    <mesh_term>Syndrome</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/NCT04498871
org study id: CAMN107A2413
nct id: NCT04498871
lead sponsor: 
The purpose of this program is to allow access to nilotinib for eligible patients diagnosed
      with Hypereosinophilic syndrome (HES). The patient's Treating Physician should follow the
      suggested treatment guidelines and comply with all local health authority regulations. The
      requesting Treating Physician submitted a request for access to drug (often referred to as
      Compassionate Use) to Novartis which was reviewed and approved by the medical team
      experienced with the drug and indication. Please refer to the latest Investigator's Brochure
      (IB) or approved label for overview of drug including: non-clinical and clinical experience,
      risk and benefits. Novartis will continue to provide any new safety information to the
      Treating Physician as they emerge.
expanded access type individual: Yes
intervention type: Drug
intervention name: Nilotinib
description: Nilotinib is recommended to be administered orally on a continuous schedule at a dose of 400 mg twice daily. The patient should be instructed to take each morning dose of nilotinib 2 hours after a light breakfast (e.g., toast and jam), with no oral intake other than water for 1 hour after dosing. It is recommended that the patient take nilotinib doses every 12 hours with a glass of water. The evening dose of nilotinib should be taken at least 2 hours after dinner with no oral intake other than water for 1 hour after dosing.
criteria: 
gender: All
minimum age: 18 Years
maximum age: 99 Years
healthy volunteers: No
responsible party type: Sponsor
mesh term: Hypereosinophilic Syndrome
mesh term: Syndrome