<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/NCT04461886</url>
  </required_header>
  <id_info>
    <org_study_id>NPC-12G-5</org_study_id>
    <nct_id>NCT04461886</nct_id>
  </id_info>
  <brief_title>A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I</brief_title>
  <official_title>A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Nobelpharma</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Nobelpharma</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this tria is to evaluate the safety and efficacy of long-term treatment with&#xD;
      NPC-12G gel to patients with neurofibromatosis type I.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      A Phase 3, open-label, uncontrolled, multicenter study to evaluate the safety and efficacy of&#xD;
      long-term treatment with NPC-12G gel for patients with neurofibromatosis type I. Patients who&#xD;
      meet all entry criteria for this trial apply NPC-12G gel twice a day for 52 weeks or more.&#xD;
      Approximately 100 eligible patients will be enrolled.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Sponsor decision&#xD;
  </why_stopped>
  <start_date type="Actual">July 8, 2020</start_date>
  <completion_date type="Actual">December 7, 2021</completion_date>
  <primary_completion_date type="Actual">December 7, 2021</primary_completion_date>
  <phase>Phase 3</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>Discontinuation rate associated with adverse events (Kaplan-Meier method)</measure>
    <time_frame>52 weeks</time_frame>
    <description>Number of Adverse events leading to discontinuation</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">100</enrollment>
  <condition>Neurofibromatosis</condition>
  <arm_group>
    <arm_group_label>NPC-12G gel</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>NPC-12G gel is containing 0.2% Sirolimus</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>NPC-12G gel</intervention_name>
    <description>NPC-12G gel is containing 0.2% Sirolimus</description>
    <arm_group_label>NPC-12G gel</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Patients diagnosed as neurofibromatosis type 1 based on the clinical diagnostic&#xD;
             criteria in guideline of Japanese Dermatological Association&#xD;
&#xD;
          2. Patients participated in a Phase II/III investigator-initiated clinical trial for&#xD;
             neurofibromatosis type I (OSD-001-004) who wish to continue treatment of the&#xD;
             investigational drug&#xD;
&#xD;
          3. At the time of enrollment, patients who are able to choose ten evaluable target&#xD;
             lesions for efficacy (at least 5)..&#xD;
&#xD;
          4. Patients who have evaluable skin neurofibromas at baseline.&#xD;
&#xD;
          5. Males and females who are 3 years old or elder at the time of informed consent.&#xD;
&#xD;
          6. Patients who (or whose guardian) give a written informed consent in understanding and&#xD;
             willingness after having received enough explanation regarding the study&#xD;
             participation.&#xD;
&#xD;
          7. Patients whose use or continued use of the investigational drug is judged to be&#xD;
             reasonable by the principal investigator or sub-investigator.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Patients who occurred serious adverse effects, who discontinued the sudy due to&#xD;
             adverse effects, or who discontinued the trial due to withdrew of their consent in the&#xD;
             Phase II/III investigator-initiated study of neurofibromatosis type I (OSD-001-004)&#xD;
&#xD;
          2. Patients with abnormal findings (e.g., pneumonia) in the results of chest x-ray at the&#xD;
             time of screening&#xD;
&#xD;
          3. Patients with creatinine clearance of less than 50 mL/min&#xD;
&#xD;
          4. Patients with poorly controlled dyslipidemia (serum triglycerides &gt;500 mg/dL or LDL&#xD;
             cholesterol &gt;190 mg/dL even with treatment)&#xD;
&#xD;
          5. Patients who have complications such as infection, cardiac, hepatic, pulmonary, renal,&#xD;
             or hematological diseases and malignant tumors that are considered unsuitable for&#xD;
             participation in this trial.&#xD;
&#xD;
          6. Patients with alcohol sensitivity or allergy to an ingredient of the study drug&#xD;
             (Sirolimus)&#xD;
&#xD;
          7. Female patients who are pregnant, may be pregnant, or are lactating&#xD;
&#xD;
          8. Patients (including male patients with a fertile partner) who cannot consent to use&#xD;
             adequate contraception from the date of consent to 12 weeks after the end of treatment&#xD;
&#xD;
          9. Patients who participated in any other clinical trial or clinical study, other than&#xD;
             the OSD-001-004 study, and have taken an investigational or investigational drug&#xD;
             within 6 months prior to the date of the consent&#xD;
&#xD;
         10. Patients who are participating in an observational study during this trial&#xD;
&#xD;
         11. Patients who are considered by the investigator as unsuitable for participation in&#xD;
             this trial&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>3 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Osaka University Hospital</name>
      <address>
        <city>Suita-shi</city>
        <state>Osaka</state>
        <zip>565-0871</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Jikei University Hospital</name>
      <address>
        <city>Minato-ku</city>
        <state>Tokyo</state>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Fukuoka University Hospital</name>
      <address>
        <city>Fukuoka</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tottori University Hospital</name>
      <address>
        <city>Tottori</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Japan</country>
  </location_countries>
  <verification_date>May 2022</verification_date>
  <study_first_submitted>July 3, 2020</study_first_submitted>
  <study_first_submitted_qc>July 7, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 8, 2020</study_first_posted>
  <last_update_submitted>May 17, 2022</last_update_submitted>
  <last_update_submitted_qc>May 17, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">May 24, 2022</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>Neurofibromatoses</mesh_term>
    <mesh_term>Neurofibromatosis 1</mesh_term>
    <mesh_term>Neurofibroma</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </patient_data>
  <!-- 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/NCT04461886
org study id: NPC-12G-5
nct id: NCT04461886
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The purpose of this tria is to evaluate the safety and efficacy of long-term treatment with
      NPC-12G gel to patients with neurofibromatosis type I.
A Phase 3, open-label, uncontrolled, multicenter study to evaluate the safety and efficacy of
      long-term treatment with NPC-12G gel for patients with neurofibromatosis type I. Patients who
      meet all entry criteria for this trial apply NPC-12G gel twice a day for 52 weeks or more.
      Approximately 100 eligible patients will be enrolled.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Discontinuation rate associated with adverse events (Kaplan-Meier method)
time frame: 52 weeks
description: Number of Adverse events leading to discontinuation
arm group label: NPC-12G gel
arm group type: Experimental
description: NPC-12G gel is containing 0.2% Sirolimus
intervention type: Drug
intervention name: NPC-12G gel
description: NPC-12G gel is containing 0.2% Sirolimus
arm group label: NPC-12G gel
criteria: 
gender: All
minimum age: 3 Years
maximum age: N/A
healthy volunteers: No
facility: 
facility: 
facility: 
facility: 
country: Japan
responsible party type: Sponsor
mesh term: Neurofibromatoses
mesh term: Neurofibromatosis 1
mesh term: Neurofibroma
sharing ipd: Undecided