<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/NCT02993133</url>
  </required_header>
  <id_info>
    <org_study_id>I14027 / PEMPA</org_study_id>
    <nct_id>NCT02993133</nct_id>
  </id_info>
  <brief_title>Pharmacokinetics Study of Mycophenolic Acid in Patients With an Autoimmune Bullous Dermatose, Pemphigus or Cicatricial Pemphigoid.</brief_title>
  <acronym>PEMPA</acronym>
  <official_title>Pharmacokinetics Study of Mycophenolic Acid in Patients With an Autoimmune Bullous Dermatose, Pemphigus or Cicatricial Pemphigoid.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University Hospital, Limoges</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>University Hospital, Limoges</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The main autoimmune bullous dermatoses are pemphigus and cicatricial pemphigoid. Pemphigus is&#xD;
      an autoimmune dermatological disease characterized by the production of anti-desmoclesin&#xD;
      antibodies 1 and 3, affecting the skin and mucous membranes.The cicatricial pemphigoid is an&#xD;
      autoimmune dermatological disease, characterized by the production of anti-zone antibodies of&#xD;
      the basal membrane and characterized by a predominant mucosal involvement. Mycophenolic acid&#xD;
      (MPA) is an increasingly used form of corticosteroid. Despite its increasing use,&#xD;
      pharmacokinetics in autoimmune bullous dermatosis remain little studied.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">December 2016</start_date>
  <completion_date type="Actual">April 2019</completion_date>
  <primary_completion_date type="Actual">January 2019</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>Evaluation of the Bayesian estimator performance</measure>
    <time_frame>8 hours</time_frame>
    <description>The evaluation of the Bayesian estimator performance will be based on its capacity to predict MPA AUC (Area Under the Curve), expressed as the bias (%) and the precision (root mean square error; RMSE) between the predicted AUC calculated using a limited number of samples performed in the first 4 hours post dosing and the observed AUC estimated using the reference method (trapezoidal rule method). A Bland Altman curve will be constructed.</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">53</enrollment>
  <condition>Autoimmune Bullous Dermatose</condition>
  <arm_group>
    <arm_group_label>60 patients will be used to build and validate the system</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>The first 30 patients will be used to build and validate the pharmacokinetic modeling of MPA in pemphigus.The 30 patients included later will provide additional data.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Cellcept® in autoimmune bullous dermatoses</intervention_name>
    <description>The administration will follow the recommendations for the use of Cellcept® in autoimmune bullous dermatoses</description>
    <arm_group_label>60 patients will be used to build and validate the system</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Age &gt; or = 18 years.&#xD;
&#xD;
          -  Patient treated by Mycophénolate Mofétil (MMF) per os (Cellcept®) for an autoimmune&#xD;
             bullous dermatose (pemphigus or cicatricial pemphigoid) for at least 30 days according&#xD;
             to the recommendations (PNDS Pemphigus and cicatricial pemphigoid HAS 2011)&#xD;
&#xD;
          -  Patient able to understand the nature, purpose and methodology of the study&#xD;
&#xD;
          -  Patient affiliated to the French social security system or equivalent&#xD;
&#xD;
          -  Patient who have signed an informed consent form&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Pregnant or breast-feeding women or women of childbearing potential without efficient&#xD;
             contraception (based on a declaration)&#xD;
&#xD;
          -  Patient under legal protection.&#xD;
&#xD;
          -  Patient deprived of freedom&#xD;
&#xD;
          -  Patient with any altered mental status or any psychiatric condition that would&#xD;
             interfere with the understanding of the study.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>CHU de Bordeaux</name>
      <address>
        <city>Bordeaux</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hôpital Avicenne - AP-HP</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>CHU de Rouen</name>
      <address>
        <city>Rouen</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>June 2018</verification_date>
  <study_first_submitted>December 12, 2016</study_first_submitted>
  <study_first_submitted_qc>December 12, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 15, 2016</study_first_posted>
  <last_update_submitted>April 19, 2019</last_update_submitted>
  <last_update_submitted_qc>April 19, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">April 22, 2019</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>Pemphigoid, Benign Mucous Membrane</mesh_term>
    <mesh_term>Pemphigoid, Bullous</mesh_term>
    <mesh_term>Pemphigus</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Mycophenolic Acid</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</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/NCT02993133
org study id: I14027 / PEMPA
nct id: NCT02993133
lead sponsor: 
has dmc: No
The main autoimmune bullous dermatoses are pemphigus and cicatricial pemphigoid. Pemphigus is
      an autoimmune dermatological disease characterized by the production of anti-desmoclesin
      antibodies 1 and 3, affecting the skin and mucous membranes.The cicatricial pemphigoid is an
      autoimmune dermatological disease, characterized by the production of anti-zone antibodies of
      the basal membrane and characterized by a predominant mucosal involvement. Mycophenolic acid
      (MPA) is an increasingly used form of corticosteroid. Despite its increasing use,
      pharmacokinetics in autoimmune bullous dermatosis remain little studied.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Evaluation of the Bayesian estimator performance
time frame: 8 hours
description: The evaluation of the Bayesian estimator performance will be based on its capacity to predict MPA AUC (Area Under the Curve), expressed as the bias (%) and the precision (root mean square error; RMSE) between the predicted AUC calculated using a limited number of samples performed in the first 4 hours post dosing and the observed AUC estimated using the reference method (trapezoidal rule method). A Bland Altman curve will be constructed.
arm group label: 60 patients will be used to build and validate the system
arm group type: Experimental
description: The first 30 patients will be used to build and validate the pharmacokinetic modeling of MPA in pemphigus.The 30 patients included later will provide additional data.
intervention type: Drug
intervention name: Cellcept® in autoimmune bullous dermatoses
description: The administration will follow the recommendations for the use of Cellcept® in autoimmune bullous dermatoses
arm group label: 60 patients will be used to build and validate the system
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
facility: 
facility: 
facility: 
country: France
responsible party type: Sponsor
mesh term: Pemphigoid, Benign Mucous Membrane
mesh term: Pemphigoid, Bullous
mesh term: Pemphigus
mesh term: Mycophenolic Acid
sharing ipd: No