<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/NCT03258801</url>
  </required_header>
  <id_info>
    <org_study_id>NIS-ML-29952</org_study_id>
    <nct_id>NCT03258801</nct_id>
  </id_info>
  <brief_title>Pirfenidone as Bridging Therapy for Lung Transplant in Patients Suffering From Idiopathic Pulmonary Fibrosis</brief_title>
  <official_title>Pirfenidone as Bridging Therapy for Lung Transplant in Patients Suffering From Idiopathic Pulmonary Fibrosis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Dr. Christopher Lambers</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Clinical Trials Coordination Centre, Medical University of Vienna</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Medical University of Vienna</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 diagnosis of idiopathic pulmonary fibrosis (IPF) is currently one of the most common&#xD;
      diagnoses for patients under evaluation for lung transplantation. In recent years, an&#xD;
      absolute increase in prevalence/ incidence of IPF has been observed. There is evidence that&#xD;
      patients with IPF on waiting list for lung transplantation might benefit from pirfenidone&#xD;
      treatment. Until now, no data are published regarding this important issue in lung&#xD;
      transplantation.&#xD;
&#xD;
      Primary objective is to determine whether there is a difference in the duration time of&#xD;
      mechanical ventilation (weaning) directly after lung transplantation between patients treated&#xD;
      with pirfenidone and patients without pirfenidone treatment. The Secondary objectives are to&#xD;
      determine whether there are differences between the pirfenidone treatment group and the&#xD;
      control group regarding survival after LUTX, the score on the Saint Georges Respiratory&#xD;
      Questionnaire and the decline in forced vital capacity (FVC%) In this Investigator initiated,&#xD;
      non- interventional single center study , patients on the waiting list for transplant&#xD;
      pirfenidone treatment receive oral pirfenidone at the standard dose of 2403 mg per day. The&#xD;
      treatment duration will range from 6 to 12 months. A control group will be used to correlate&#xD;
      the outcome-parameters for a descriptive comparison. The control group includes patients with&#xD;
      IPF on the waiting list who were on another IPF specific (or no) treatment for IPF The Study&#xD;
      Population are Patients aged between 40-70 years who are admitted to the lung transplantation&#xD;
      department and fulfill the international criteria for idiopathic pulmonary fibrosis (&#xD;
      existence of a usual interstitial pneumonia (UIP) pattern in the high-resolution computed&#xD;
      tomography (HRCT) is necessary).&#xD;
&#xD;
      Variables: Duration of mechanical ventilation after LUTX (hours), Forced Vital capacity&#xD;
      relative to reference value at baseline (FVC0%), Forced Vital capacity relative to reference&#xD;
      value after 6 months (FVC6%),Forced Vital capacity relative to reference value after 12&#xD;
      months (FVC12%) Study Size: 30 patients in the Pirfenidone group, 20 patients in the control&#xD;
      group.&#xD;
&#xD;
      For the primary Endpoint, the mean, standard deviation, median, minimum and maximum of the&#xD;
      weaning time of patients who received a pirfenidone treatment, as well as of patients from&#xD;
      the control group will be computed and presented in a table. Additionally, a Kaplan-Meier&#xD;
      curve will be estimated and plotted alongside the respective 95% CI calculated using the&#xD;
      method of Brookmeyer and Crowley. Furthermore, a stepwise linear regression using forward&#xD;
      selection and Age, RBMI, FVC0%, (FVC6%-FVC0%), TLC, FEV1% and ECMO, as well as the&#xD;
      pirfenidone treatment as predictors will be computed. The null hypothesis is that the&#xD;
      pirfenidone treatment has no influence on the weaning time. The according model coefficient&#xD;
      estimate and standard error will be used to test the null hypothesis using a t-test at&#xD;
      significance level α=0.05.&#xD;
&#xD;
      For the secondary endpoints, the mean, standard deviation, median, minimum and maximum of&#xD;
      patients who received a pirfenidone treatment, as well as of patients from the control group&#xD;
      will be computed and presented in a tableStepwise Cox Regression using forward selection and&#xD;
      Age, RBMI, FVC0%, (FVC6%-FVC0%) and ECMO, as well as the pirfenidone treatment as predictors&#xD;
      will be computed in order to compare the treatment and the control group a . If p-values are&#xD;
      calculated for the secondary endpoint analysis, they serve only descriptive purposes.&#xD;
      Therefore no multiple testing corrections are applied.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    Clinical evaluation plan outdated and obsolete due to altered clinical routine&#xD;
  </why_stopped>
  <start_date type="Actual">October 29, 2018</start_date>
  <completion_date type="Actual">December 2, 2019</completion_date>
  <primary_completion_date type="Actual">December 2, 2019</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Duration time of mechanical ventilation (weaning) directly after lung transplantation</measure>
    <time_frame>First two weeks after lung transplantation</time_frame>
    <description>Duration of mechanical ventilation after LUTX measured in days</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Days of survival after LUTX</measure>
    <time_frame>First 90 days after LUTX</time_frame>
    <description>Days of survival after LUTX measured in days</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>decline in forced vital capacity (FVC%) from baseline to 6 months</measure>
    <time_frame>from baseline to 6 months</time_frame>
    <description>decline in forced vital capacity (FVC%) from baseline to 6 months</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>decline in forced vital capacity (FVC%) from baseline to 12 months</measure>
    <time_frame>from baseline to 12 months</time_frame>
    <description>decline in forced vital capacity (FVC%) from baseline to 12 months</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>decline in forced vital capacity (FVC%) from 6 months to 12 months</measure>
    <time_frame>from 6 months to 12 months</time_frame>
    <description>decline in forced vital capacity (FVC%) from 6 months to 12 months</description>
  </secondary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Actual">0</enrollment>
  <condition>Idiopathic Pulmonary Fibrosis</condition>
  <condition>Lung Transplant; Complications</condition>
  <arm_group>
    <arm_group_label>Pirfenidone</arm_group_label>
    <description>Patients who are admitted to the lung transplantation department and fulfill the international criteria for idiopathic pulmonary fibrosis and are treated with Pirfenidone as bridging therapy.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Control</arm_group_label>
    <description>Patients who are admitted to the lung transplantation department and fulfill the international criteria for idiopathic pulmonary fibrosis and are not treated with Pirfenidone.</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        All patients who are admitted to the lung transplantation department and fulfill the&#xD;
        international criteria for idiopathic pulmonary fibrosis will be evaluated (existence of a&#xD;
        usual interstitial pneumonia (UIP) pattern in the high-resolution computed tomography&#xD;
        (HRCT) is necessary).&#xD;
&#xD;
        The assignment of a patient to a particular therapeutic strategy remains in the sole&#xD;
        responsibility of the treating physician and must not be dictated by this study-specific&#xD;
        observation- and evaluation plan&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        For Pirfenidone Group&#xD;
&#xD;
        Patients must meet all of the following inclusion criteria to be eligible for inclusion in&#xD;
        the study&#xD;
&#xD;
          -  mild to moderate idiopathic pulmonary fibrosis (IPF).&#xD;
&#xD;
          -  Current or intended treatment with Pirfenidone&#xD;
&#xD;
          -  Diagnosis of Interstitial Lung disease&#xD;
&#xD;
          -  Evaluation for Lung Transplantation&#xD;
&#xD;
          -  Age 40-70&#xD;
&#xD;
        For Control Group&#xD;
&#xD;
        Patients must meet all of the following inclusion criteria to be eligible for inclusion in&#xD;
        the study&#xD;
&#xD;
          -  mild to moderate idiopathic pulmonary fibrosis (IPF).&#xD;
&#xD;
          -  Diagnosis of Interstitial Lung disease&#xD;
&#xD;
          -  Evaluation for Lung Transplantation&#xD;
&#xD;
          -  Age 40-70&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        For Pirfenidone Group&#xD;
&#xD;
          -  Other lung diseases (such as cystic fibrosis, COPD)&#xD;
&#xD;
          -  Infection with Hepatitis C,&#xD;
&#xD;
          -  Liver cirrhosis CHILD C&#xD;
&#xD;
          -  Coronary heart disease (3VD)&#xD;
&#xD;
        For Control Group&#xD;
&#xD;
          -  Other lung diseases (such as cystic fibrosis, COPD)&#xD;
&#xD;
          -  Infection with Hepatitis C,&#xD;
&#xD;
          -  Liver cirrhosis CHILD C&#xD;
&#xD;
          -  Coronary heart disease (3VD)&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>40 Years</minimum_age>
    <maximum_age>70 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Medical University of Vienna, Department of Surgery</name>
      <address>
        <city>Vienna</city>
        <zip>1090</zip>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Austria</country>
  </location_countries>
  <verification_date>January 2020</verification_date>
  <study_first_submitted>August 18, 2017</study_first_submitted>
  <study_first_submitted_qc>August 18, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 23, 2017</study_first_posted>
  <last_update_submitted>January 23, 2020</last_update_submitted>
  <last_update_submitted_qc>January 23, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">January 27, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Medical University of Vienna</investigator_affiliation>
    <investigator_full_name>Dr. Christopher Lambers</investigator_full_name>
    <investigator_title>Ass. Prof.</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Pulmonary Fibrosis</mesh_term>
    <mesh_term>Idiopathic Pulmonary Fibrosis</mesh_term>
    <mesh_term>Fibrosis</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/NCT03258801
org study id: NIS-ML-29952
nct id: NCT03258801
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The diagnosis of idiopathic pulmonary fibrosis (IPF) is currently one of the most common
      diagnoses for patients under evaluation for lung transplantation. In recent years, an
      absolute increase in prevalence/ incidence of IPF has been observed. There is evidence that
      patients with IPF on waiting list for lung transplantation might benefit from pirfenidone
      treatment. Until now, no data are published regarding this important issue in lung
      transplantation.

      Primary objective is to determine whether there is a difference in the duration time of
      mechanical ventilation (weaning) directly after lung transplantation between patients treated
      with pirfenidone and patients without pirfenidone treatment. The Secondary objectives are to
      determine whether there are differences between the pirfenidone treatment group and the
      control group regarding survival after LUTX, the score on the Saint Georges Respiratory
      Questionnaire and the decline in forced vital capacity (FVC%) In this Investigator initiated,
      non- interventional single center study , patients on the waiting list for transplant
      pirfenidone treatment receive oral pirfenidone at the standard dose of 2403 mg per day. The
      treatment duration will range from 6 to 12 months. A control group will be used to correlate
      the outcome-parameters for a descriptive comparison. The control group includes patients with
      IPF on the waiting list who were on another IPF specific (or no) treatment for IPF The Study
      Population are Patients aged between 40-70 years who are admitted to the lung transplantation
      department and fulfill the international criteria for idiopathic pulmonary fibrosis (
      existence of a usual interstitial pneumonia (UIP) pattern in the high-resolution computed
      tomography (HRCT) is necessary).

      Variables: Duration of mechanical ventilation after LUTX (hours), Forced Vital capacity
      relative to reference value at baseline (FVC0%), Forced Vital capacity relative to reference
      value after 6 months (FVC6%),Forced Vital capacity relative to reference value after 12
      months (FVC12%) Study Size: 30 patients in the Pirfenidone group, 20 patients in the control
      group.

      For the primary Endpoint, the mean, standard deviation, median, minimum and maximum of the
      weaning time of patients who received a pirfenidone treatment, as well as of patients from
      the control group will be computed and presented in a table. Additionally, a Kaplan-Meier
      curve will be estimated and plotted alongside the respective 95% CI calculated using the
      method of Brookmeyer and Crowley. Furthermore, a stepwise linear regression using forward
      selection and Age, RBMI, FVC0%, (FVC6%-FVC0%), TLC, FEV1% and ECMO, as well as the
      pirfenidone treatment as predictors will be computed. The null hypothesis is that the
      pirfenidone treatment has no influence on the weaning time. The according model coefficient
      estimate and standard error will be used to test the null hypothesis using a t-test at
      significance level α=0.05.

      For the secondary endpoints, the mean, standard deviation, median, minimum and maximum of
      patients who received a pirfenidone treatment, as well as of patients from the control group
      will be computed and presented in a tableStepwise Cox Regression using forward selection and
      Age, RBMI, FVC0%, (FVC6%-FVC0%) and ECMO, as well as the pirfenidone treatment as predictors
      will be computed in order to compare the treatment and the control group a . If p-values are
      calculated for the secondary endpoint analysis, they serve only descriptive purposes.
      Therefore no multiple testing corrections are applied.
observational model: Cohort
time perspective: Prospective
measure: Duration time of mechanical ventilation (weaning) directly after lung transplantation
time frame: First two weeks after lung transplantation
description: Duration of mechanical ventilation after LUTX measured in days
measure: Days of survival after LUTX
time frame: First 90 days after LUTX
description: Days of survival after LUTX measured in days
measure: decline in forced vital capacity (FVC%) from baseline to 6 months
time frame: from baseline to 6 months
description: decline in forced vital capacity (FVC%) from baseline to 6 months
measure: decline in forced vital capacity (FVC%) from baseline to 12 months
time frame: from baseline to 12 months
description: decline in forced vital capacity (FVC%) from baseline to 12 months
measure: decline in forced vital capacity (FVC%) from 6 months to 12 months
time frame: from 6 months to 12 months
description: decline in forced vital capacity (FVC%) from 6 months to 12 months
arm group label: Pirfenidone
description: Patients who are admitted to the lung transplantation department and fulfill the international criteria for idiopathic pulmonary fibrosis and are treated with Pirfenidone as bridging therapy.
arm group label: Control
description: Patients who are admitted to the lung transplantation department and fulfill the international criteria for idiopathic pulmonary fibrosis and are not treated with Pirfenidone.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 40 Years
maximum age: 70 Years
healthy volunteers: No
facility: 
country: Austria
responsible party type: Sponsor-Investigator
investigator affiliation: Medical University of Vienna
investigator full name: Dr. Christopher Lambers
investigator title: Ass. Prof.
mesh term: Pulmonary Fibrosis
mesh term: Idiopathic Pulmonary Fibrosis
mesh term: Fibrosis