<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/NCT03265288</url>
  </required_header>
  <id_info>
    <org_study_id>LAU-14-01</org_study_id>
    <nct_id>NCT03265288</nct_id>
  </id_info>
  <brief_title>Study of LAU-7b in the Treatment of Cystic Fibrosis in Adults</brief_title>
  <acronym>APPLAUD</acronym>
  <official_title>APPLAUD: A Double-Blind, Randomized, Placebo-Controlled, Phase II Study of the Efficacy and Safety of LAU-7b in the Treatment of Cystic Fibrosis in Adults</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Laurent Pharmaceuticals Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Cystic Fibrosis Foundation</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Laurent Pharmaceuticals Inc.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the&#xD;
      safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the&#xD;
      safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF.&#xD;
      All patients will remain on their CF standard-of-care treatments over the trial duration.&#xD;
&#xD;
      The goal for the treatment with LAU-7b in CF is to preserve lung function by reducing the&#xD;
      persistent inflammation in the lung and to improve its capacity to defend against resistant&#xD;
      bacteria such as Pseudomonas aeruginosa.&#xD;
&#xD;
      The treatment regimen will consist of 6 consecutive &quot;dosing cycles&quot; of 21 days each, spaced&#xD;
      by study drug-free periods of 7 days. A total of 136 eligible adult patients with CF will be&#xD;
      randomized to receive 300 mg LAU-7b or placebo in a 1:1 ratio. The participation in the study&#xD;
      will last about 7 months.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">October 10, 2018</start_date>
  <completion_date type="Actual">September 15, 2021</completion_date>
  <primary_completion_date type="Actual">September 15, 2021</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <intervention_model_description>Double-blind, randomized, parallel groups and placebo-controlled trial.</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
    <masking_description>Patients will be randomly assigned to take either the active drug (fenretinide capsule) or a matching inactive placebo (inactive capsule).</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Absolute change in percent predicted forced expiratory volume in 1 second (FEV1%)</measure>
    <time_frame>From baseline to 24 weeks</time_frame>
    <description>Standardized, serial FEV1 measurements will be performed during the trial</description>
  </primary_outcome>
  <primary_outcome>
    <measure>The safety and tolerability of LAU-7b will be assessed by the incidence of treatment emergent adverse events compared to placebo</measure>
    <time_frame>From Baseline to 28 weeks</time_frame>
    <description>This will be assessed through serial assessments and ad-hoc assessments</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>The proportion of patients achieving normalization of the arachidonic acid, docosahexaenoic acid and their ratio in phospholipids</measure>
    <time_frame>From baseline to 28 weeks</time_frame>
    <description>This will be assessed through serial blood sampling during the trial</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The absolute and relative (%) change in FEV1 percent predicted at 3, 7, 11, 15 and 28 weeks into the trial</measure>
    <time_frame>From baseline to 3, 7, 11, 15 and 28 weeks into the trial</time_frame>
    <description>Standardized, serial FEV1 measurements will be performed during the trial</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The time to first protocol-defined pulmonary exacerbation</measure>
    <time_frame>From baseline to 28 weeks</time_frame>
    <description>Reports of pulmonary exacerbation during the trial</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The incidence of protocol-defined pulmonary exacerbation</measure>
    <time_frame>From baseline to 28 weeks</time_frame>
    <description>Reports of pulmonary exacerbation during the trial</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The time to first change and usage of antibiotic (other than chronic inhaled antibiotics already started prior to trial or oral chronic azithromycin)</measure>
    <time_frame>From baseline to 28 weeks</time_frame>
    <description>Reports of pulmonary exacerbation and their treatment during the trial</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The change from baseline of systemic markers of inflammation in blood</measure>
    <time_frame>From baseline to 28 weeks</time_frame>
    <description>This will be assessed through serial blood sampling during the trial</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The change from screening of the body weight and calculated Body Mass Index (BMI)</measure>
    <time_frame>From screening to 28 weeks</time_frame>
    <description>This will be assessed through serial weighing during the trial</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The overall change from screening of the Pseudomonas aeruginosa density (colony forming units) in the sputum</measure>
    <time_frame>From screening to Weeks 11 and 24</time_frame>
    <description>This will be assessed through induced sputum on 3 occasions during the trial</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The impact (from baseline) on overall health, daily life, perceived well-being and symptoms measured with the Cystic Fibrosis Questionnaire-Revised (CFQ-R)</measure>
    <time_frame>From baseline to 28 weeks</time_frame>
    <description>This will be assessed through administration of the questionnaire at planned times during the trial</description>
  </secondary_outcome>
  <other_outcome>
    <measure>The change in metabolipidomic profile and in markers of oxidative stress in blood</measure>
    <time_frame>From baseline to 28 weeks</time_frame>
    <description>This will be assessed through serial blood sampling during the trial</description>
  </other_outcome>
  <other_outcome>
    <measure>The change in metabolipidomic profile in blood, the systemic markers of inflammation in blood, the FEV1, the body weight and calculated BMI</measure>
    <time_frame>From baseline to 28 weeks</time_frame>
    <description>Only in patients who experience a pulmonary exacerbation requiring intravenous antibiotics, this will be assessed prior to- and after the intravenous antibiotic course.</description>
  </other_outcome>
  <other_outcome>
    <measure>The change from baseline of systemic bone formation and resorption biomarkers</measure>
    <time_frame>Baseline and 24 weeks</time_frame>
    <description>This will be assessed through blood sampling on 2 occasions during the trial</description>
  </other_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">166</enrollment>
  <condition>Cystic Fibrosis</condition>
  <arm_group>
    <arm_group_label>LAU-7b</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Active drug fenretinide (as LAU-7b capsules)</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Placebo oral capsule (as inactive capsules identical to active arm)</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>LAU-7b</intervention_name>
    <description>LAU-7b will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.</description>
    <arm_group_label>LAU-7b</arm_group_label>
    <other_name>fenretinide</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo oral capsule</intervention_name>
    <description>Placebo will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Screening FEV1 between 40% and 100% predicted value for age, gender and height, in&#xD;
             patients capable of properly performing the test;&#xD;
&#xD;
          -  History of pulmonary exacerbation, defined as at least one (1) pulmonary exacerbation&#xD;
             in the year prior to Screening which resulted in documented intravenous or Oral&#xD;
             antibiotics;&#xD;
&#xD;
          -  Patients are eligible independently of their history of pulmonary Pseudomonas&#xD;
             aeruginosa (PsA) infection and their PsA status at screening;&#xD;
&#xD;
          -  If taking Kalydeco® (ivacaftor), Orkambi® (ivacaftor/lumacaftor), Symdeko®&#xD;
             (ivacaftor/tezacaftor) or other commercially available CFTR modulator products,&#xD;
             patients must be taking it for a minimum of 3 months prior to screening if naïve to&#xD;
             CFTR modulators and 1 month if switched from another CFTR modulator product and deemed&#xD;
             to tolerate it;&#xD;
&#xD;
          -  No change in CF and allowed systemic chronic therapy for a minimum of 5 weeks prior to&#xD;
             randomization, of which 2 weeks minimum are prior to screening;&#xD;
&#xD;
          -  Female patients of child bearing potential should be on highly effective contraceptive&#xD;
             methods during the study;&#xD;
&#xD;
          -  Male patients with spouse or partner of child bearing potential, or pregnant, are&#xD;
             eligible if they use an appropriate method of contraception.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Pregnancy: due to the potential teratogenic effects of retinoids, pregnant women are&#xD;
             NOT eligible;&#xD;
&#xD;
          -  Breast milk feeding by study patient is NOT allowed;&#xD;
&#xD;
          -  Clinically abnormal renal function: serum creatinine &gt; 132 μM (1.5 mg/dL);&#xD;
&#xD;
          -  Clinically abnormal liver function: Total bilirubin &gt;1.5 x ULN (in the absence of&#xD;
             demonstrated Gilbert's syndrome), alanine aminotransferase (ALT) and/or aspartate&#xD;
             aminotransferase (AST) &gt; 2.5 x ULN;&#xD;
&#xD;
          -  Patients with plasma retinol levels below 0.7 µM;&#xD;
&#xD;
          -  Presence of nyctalopia or hemeralopia at enrolment, or any other serious retinal,&#xD;
             ophthalmological condition;&#xD;
&#xD;
          -  Presence of serious dermatological conditions at entry, including inflammatory or&#xD;
             xerotic skin pathologies such as psoriasis or ichthyosis;&#xD;
&#xD;
          -  Intake of chronic systemic steroids in the month prior to screening and during the&#xD;
             study;&#xD;
&#xD;
          -  History of acute infections (viral/bacterial/fungal) within 5 weeks prior to&#xD;
             randomization, of which 2 weeks minimum are prior to screening, whether or not treated&#xD;
             and resolved;&#xD;
&#xD;
          -  Presence of infection with Burkholderia cepacia (including all species within the&#xD;
             Burkholderia cepacia complex group, and Burkholderia gladioli) in the 12 months prior&#xD;
             to screening;&#xD;
&#xD;
          -  Patients with a confirmed diagnosis (as per the Cystic Fibrosis Foundation diagnostic&#xD;
             criteria) of Allergic BronchoPulmonary Aspergillosis (ABPA) and actively being treated&#xD;
             with corticosteroids and/or anti fungal agents.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Larry C Lands, MD PhD</last_name>
    <role>Study Chair</role>
    <affiliation>McGill Uinversity Health Centre</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Michael W Konstan, MD</last_name>
    <role>Study Chair</role>
    <affiliation>Rainbow Babies and Children's Hospital/ University Hospitals Cleveland Medical Center</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Long Beach Memorial Medical Center</name>
      <address>
        <city>Long Beach</city>
        <state>California</state>
        <zip>90806</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital Los Angeles</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90227</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UC Davis Medical Center, Division of Pulmonary &amp; Critical Care Medicine</name>
      <address>
        <city>Sacramento</city>
        <state>California</state>
        <zip>95817</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's National Medical Center</name>
      <address>
        <city>Washington</city>
        <state>District of Columbia</state>
        <zip>20010</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Division of pulmonary, critical care and sleep medicine, University of Florida</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32610</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Memorial Healthcare System, Joe DiMaggio Children's Hospital Cystic Fibrosis &amp; Pulmonary Center</name>
      <address>
        <city>Hollywood</city>
        <state>Florida</state>
        <zip>33021</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Avanza Medical Research Center</name>
      <address>
        <city>Pensacola</city>
        <state>Florida</state>
        <zip>32603</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>St-Luke's CF Center of Idaho</name>
      <address>
        <city>Boise</city>
        <state>Idaho</state>
        <zip>83712</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Riley Hospital for Children</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Kansas Medical Center</name>
      <address>
        <city>Kansas City</city>
        <state>Kansas</state>
        <zip>66103</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Maine Medical Center Cystic Fibrosis Research</name>
      <address>
        <city>Portland</city>
        <state>Maine</state>
        <zip>04102</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Michigan Health System</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Wayne State University, Harper University Hospital</name>
      <address>
        <city>Detroit</city>
        <state>Michigan</state>
        <zip>48201</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Minnesota Cystic Fibrosis Center, University of Minnesota</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55455</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Washington University Medical School</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63110</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Morristown Medical Center, NJ Adult Cystic Fibrosis Center</name>
      <address>
        <city>Morristown</city>
        <state>New Jersey</state>
        <zip>07962</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rutgers University Clinical Research Center, RW Johnson University Hospital</name>
      <address>
        <city>New Brunswick</city>
        <state>New Jersey</state>
        <zip>08901</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Albany Medical College</name>
      <address>
        <city>Albany</city>
        <state>New York</state>
        <zip>12208</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospitals Cleveland Medical Center, Rainbow Babies and Children's Hospital</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nationwide Children's Hospital</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Oklahoma Health Sciences Center</name>
      <address>
        <city>Oklahoma City</city>
        <state>Oklahoma</state>
        <zip>73104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cystic Fibrosis Center, Doernbecher Children's Hospital, Oregon Health &amp; Science University</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Pittsburgh of UPMC</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15224</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Medical University of South Carolina</name>
      <address>
        <city>Charleston</city>
        <state>South Carolina</state>
        <zip>29425</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Utah</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84132</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Virginia Commonwealth University</name>
      <address>
        <city>Richmond</city>
        <state>Virginia</state>
        <zip>23298</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Medical College of Wisconsin, Div of Pulmonary and Critical Care Medicine</name>
      <address>
        <city>Milwaukee</city>
        <state>Wisconsin</state>
        <zip>53226</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Respiratory Medicine, John Hunter Hospital</name>
      <address>
        <city>New Lambton Heights</city>
        <state>New South Wales</state>
        <zip>2305</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Department of Respiratory Medicine, Royal Prince Alfred Hospital</name>
      <address>
        <city>Sydney</city>
        <state>New South Wales</state>
        <zip>2050</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Department of Respiratory and Sleep Medicine, Westmead Hospital</name>
      <address>
        <city>Westmead</city>
        <state>New South Wales</state>
        <zip>2145</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mater Misericordiae Ltd</name>
      <address>
        <city>Brisbane</city>
        <state>Queensland</state>
        <zip>4101</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Monash Lung and Sleep, Monash Health</name>
      <address>
        <city>Clayton</city>
        <state>Victoria</state>
        <zip>3168</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Alfred Hospital</name>
      <address>
        <city>Melbourne</city>
        <state>Victoria</state>
        <zip>3004</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Institute of Respiratory Health, Harry Perkins Institute</name>
      <address>
        <city>Nedlands</city>
        <state>Western Australia</state>
        <zip>6009</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Pacific Lung Research Institute at St. Paul's Hospital</name>
      <address>
        <city>Vancouver</city>
        <state>British Columbia</state>
        <zip>V6Z 1Y6</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Ottawa Hospital Center for Practice-Changing Research</name>
      <address>
        <city>Ottawa</city>
        <state>Ontario</state>
        <zip>K1H 8L6</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre d'études cliniques CIUSS SLJ, Hôpital Chicoutimi</name>
      <address>
        <city>Chicoutimi</city>
        <state>Quebec</state>
        <zip>G7H 5H6</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre Hospitalier de l'Université de Montréal</name>
      <address>
        <city>Montréal</city>
        <state>Quebec</state>
        <zip>H2X2P1</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>McGill University Health Center</name>
      <address>
        <city>Montréal</city>
        <state>Quebec</state>
        <zip>H4A 3J1</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre de recherche de l'institut Universitaire de Cardiologie et de Pneumologie de Québec</name>
      <address>
        <city>Québec City</city>
        <state>Quebec</state>
        <zip>G1V 4G5</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Canada</country>
    <country>United States</country>
  </location_countries>
  <verification_date>October 2021</verification_date>
  <study_first_submitted>February 21, 2017</study_first_submitted>
  <study_first_submitted_qc>August 25, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 29, 2017</study_first_posted>
  <last_update_submitted>October 3, 2021</last_update_submitted>
  <last_update_submitted_qc>October 3, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">October 5, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Inflammation</keyword>
  <keyword>Essential Fatty Acids</keyword>
  <keyword>Inflammation resolution</keyword>
  <keyword>Docosahexaenoic acid</keyword>
  <keyword>Lung function</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cystic Fibrosis</mesh_term>
    <mesh_term>Fibrosis</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Fenretinide</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/NCT03265288
org study id: LAU-14-01
nct id: NCT03265288
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
is us export: No
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the
      safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF.
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the
      safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF.
      All patients will remain on their CF standard-of-care treatments over the trial duration.

      The goal for the treatment with LAU-7b in CF is to preserve lung function by reducing the
      persistent inflammation in the lung and to improve its capacity to defend against resistant
      bacteria such as Pseudomonas aeruginosa.

      The treatment regimen will consist of 6 consecutive "dosing cycles" of 21 days each, spaced
      by study drug-free periods of 7 days. A total of 136 eligible adult patients with CF will be
      randomized to receive 300 mg LAU-7b or placebo in a 1:1 ratio. The participation in the study
      will last about 7 months.
allocation: Randomized
intervention model: Parallel Assignment
intervention model description: Double-blind, randomized, parallel groups and placebo-controlled trial.
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
masking description: Patients will be randomly assigned to take either the active drug (fenretinide capsule) or a matching inactive placebo (inactive capsule).
measure: Absolute change in percent predicted forced expiratory volume in 1 second (FEV1%)
time frame: From baseline to 24 weeks
description: Standardized, serial FEV1 measurements will be performed during the trial
measure: The safety and tolerability of LAU-7b will be assessed by the incidence of treatment emergent adverse events compared to placebo
time frame: From Baseline to 28 weeks
description: This will be assessed through serial assessments and ad-hoc assessments
measure: The proportion of patients achieving normalization of the arachidonic acid, docosahexaenoic acid and their ratio in phospholipids
time frame: From baseline to 28 weeks
description: This will be assessed through serial blood sampling during the trial
measure: The absolute and relative (%) change in FEV1 percent predicted at 3, 7, 11, 15 and 28 weeks into the trial
time frame: From baseline to 3, 7, 11, 15 and 28 weeks into the trial
description: Standardized, serial FEV1 measurements will be performed during the trial
measure: The time to first protocol-defined pulmonary exacerbation
time frame: From baseline to 28 weeks
description: Reports of pulmonary exacerbation during the trial
measure: The incidence of protocol-defined pulmonary exacerbation
time frame: From baseline to 28 weeks
description: Reports of pulmonary exacerbation during the trial
measure: The time to first change and usage of antibiotic (other than chronic inhaled antibiotics already started prior to trial or oral chronic azithromycin)
time frame: From baseline to 28 weeks
description: Reports of pulmonary exacerbation and their treatment during the trial
measure: The change from baseline of systemic markers of inflammation in blood
time frame: From baseline to 28 weeks
description: This will be assessed through serial blood sampling during the trial
measure: The change from screening of the body weight and calculated Body Mass Index (BMI)
time frame: From screening to 28 weeks
description: This will be assessed through serial weighing during the trial
measure: The overall change from screening of the Pseudomonas aeruginosa density (colony forming units) in the sputum
time frame: From screening to Weeks 11 and 24
description: This will be assessed through induced sputum on 3 occasions during the trial
measure: The impact (from baseline) on overall health, daily life, perceived well-being and symptoms measured with the Cystic Fibrosis Questionnaire-Revised (CFQ-R)
time frame: From baseline to 28 weeks
description: This will be assessed through administration of the questionnaire at planned times during the trial
measure: The change in metabolipidomic profile and in markers of oxidative stress in blood
time frame: From baseline to 28 weeks
description: This will be assessed through serial blood sampling during the trial
measure: The change in metabolipidomic profile in blood, the systemic markers of inflammation in blood, the FEV1, the body weight and calculated BMI
time frame: From baseline to 28 weeks
description: Only in patients who experience a pulmonary exacerbation requiring intravenous antibiotics, this will be assessed prior to- and after the intravenous antibiotic course.
measure: The change from baseline of systemic bone formation and resorption biomarkers
time frame: Baseline and 24 weeks
description: This will be assessed through blood sampling on 2 occasions during the trial
arm group label: LAU-7b
arm group type: Experimental
description: Active drug fenretinide (as LAU-7b capsules)
arm group label: Placebo
arm group type: Placebo Comparator
description: Placebo oral capsule (as inactive capsules identical to active arm)
intervention type: Drug
intervention name: LAU-7b
description: LAU-7b will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.
arm group label: LAU-7b
other name: fenretinide
intervention type: Drug
intervention name: Placebo oral capsule
description: Placebo will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.
arm group label: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Larry C Lands, MD PhD
role: Study Chair
affiliation: McGill Uinversity Health Centre
last name: Michael W Konstan, MD
role: Study Chair
affiliation: Rainbow Babies and Children's Hospital/ University Hospitals Cleveland Medical Center
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country: Australia
country: Canada
country: United States
responsible party type: Sponsor
mesh term: Cystic Fibrosis
mesh term: Fibrosis
mesh term: Fenretinide
sharing ipd: No