<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/NCT01710644</url>
  </required_header>
  <id_info>
    <org_study_id>NM-BL-101</org_study_id>
    <secondary_id>207862</secondary_id>
    <nct_id>NCT01710644</nct_id>
  </id_info>
  <brief_title>Efficacy and Tolerability of NM-BL in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis</brief_title>
  <official_title>A Randomized, Double-blind, Multicenter, Two-period Crossover Study to Assess the Efficacy and Tolerability of NM-BL (Burlulipase) in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Nordmark Arzneimittel GmbH &amp; Co. KG</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Parexel</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Nordmark Arzneimittel GmbH &amp; Co. KG</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this Randomized, Double-blind, Multicenter, Two-period Crossover Study is to&#xD;
      Assess the Efficacy and Tolerability of Burlulipase (NM-BL) in Patients with Exocrine&#xD;
      Pancreatic Insufficiency due to Cystic Fibrosis&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>May 2013</start_date>
  <completion_date type="Actual">August 2014</completion_date>
  <primary_completion_date type="Actual">July 2014</primary_completion_date>
  <phase>Phase 1/Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Crossover Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>To evaluate the efficacy of NM-BL compared to placebo in patients with Exocrine Pancreatic Insufficiency (EPI) due to CF, where the primary variable is coefficient of fat absorption (CFA%)</measure>
    <time_frame>72 hrs</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>To evaluate the efficacy of NM-BL compared to placebo in patients with Exocrine Pancreatic Insufficiency (EPI)due to CF, where the primary variable is coefficient of nitrogen absorption (CNA%)</measure>
    <time_frame>72 hrs</time_frame>
  </secondary_outcome>
  <other_outcome>
    <measure>Symptomatology / Symptom Questionnaire</measure>
    <time_frame>7 days</time_frame>
    <description>Comparison gastrointestinal parameters, recorded by the patient/carer in the Symptom Questionnaire each day during the Treatment Periods</description>
  </other_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">35</enrollment>
  <condition>Pancreatic Insufficiency</condition>
  <condition>Cystic Fibrosis</condition>
  <condition>Digestive System Diseases</condition>
  <condition>Lung Diseases</condition>
  <condition>Respiratory Tract Diseases</condition>
  <arm_group>
    <arm_group_label>Burlulipase</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Burlulipase orally, per meal</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo (Caramel in sterile water)</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Placebo orally, per meal</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Burlulipase</intervention_name>
    <description>Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days</description>
    <arm_group_label>Burlulipase</arm_group_label>
    <other_name>NM-BL</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo (Caramel in sterile water)</intervention_name>
    <description>Placebo will be taken with meals and snacks for 5 to 7 days</description>
    <arm_group_label>Placebo (Caramel in sterile water)</arm_group_label>
    <other_name>Placebo</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Male and female patients aged ≥12 years from the date of informed consent&#xD;
&#xD;
          -  Confirmed diagnosis of CF at screening&#xD;
&#xD;
          -  Confirmed EPI by historical (within past 12 months) CFA &lt;70% without use of PERTs or&#xD;
             current fecal elastase &lt;50 µg/g stool at screening&#xD;
&#xD;
          -  Currently receiving PERT with a commercially available pancreatic enzyme&#xD;
&#xD;
          -  Currently on stable treatment with proton pump inhibitors or H2 receptor antagonists&#xD;
&#xD;
          -  Clinically stable condition without evidence of acute respiratory disease or any other&#xD;
             acute condition&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  History of fibrosing colonopathy&#xD;
&#xD;
          -  History of significant bowel resection, in the opinion of the investigator, or solid&#xD;
             organ transplant&#xD;
&#xD;
          -  History of being refractory to pancreatic enzyme replacement&#xD;
&#xD;
          -  Current diagnosis or history of distal intestinal obstruction syndrome&#xD;
&#xD;
          -  Current diagnosis of small intestinal bacterial overgrowth, ileus or acute abdomen&#xD;
&#xD;
          -  A body mass index percentile &lt;10%&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>12 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Kristin Forssmann, MD</last_name>
    <role>Study Director</role>
    <affiliation>Nordmark Arzneimittel GmbH &amp; Co. KG</affiliation>
  </overall_official>
  <overall_official>
    <last_name>James E. Heubi, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Hospital Medical Center, Cincinnati</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Nemours Children's Clinic</name>
      <address>
        <city>Jacksonville</city>
        <state>Florida</state>
        <zip>32207</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nemours Children's Clinic</name>
      <address>
        <city>Pensacola</city>
        <state>Florida</state>
        <zip>32504</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The University of Iowa</name>
      <address>
        <city>Iowa City</city>
        <state>Iowa</state>
        <zip>52242</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Via Cristi Hospitals Wichita, Inc.</name>
      <address>
        <city>Wichita</city>
        <state>Kansas</state>
        <zip>67214</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cincinnati Children's Hospital Medical Center</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45229</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospitals Case Medical Center</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Children's Medical Center of Dayton</name>
      <address>
        <city>Dayton</city>
        <state>Ohio</state>
        <zip>45404</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Penn State Milton S. Hershey Medical Center</name>
      <address>
        <city>Hershey</city>
        <state>Pennsylvania</state>
        <zip>17033</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>West Virginia University Research Corporation</name>
      <address>
        <city>Morgantown</city>
        <state>West Virginia</state>
        <zip>26506</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>June 2016</verification_date>
  <study_first_submitted>October 17, 2012</study_first_submitted>
  <study_first_submitted_qc>October 17, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 19, 2012</study_first_posted>
  <disposition_first_submitted>April 4, 2016</disposition_first_submitted>
  <disposition_first_submitted_qc>May 4, 2016</disposition_first_submitted_qc>
  <disposition_first_posted type="Estimate">June 6, 2016</disposition_first_posted>
  <last_update_submitted>June 3, 2016</last_update_submitted>
  <last_update_submitted_qc>June 3, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">June 7, 2016</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>Cystic Fibrosis</mesh_term>
    <mesh_term>Digestive System Diseases</mesh_term>
    <mesh_term>Gastrointestinal Diseases</mesh_term>
    <mesh_term>Exocrine Pancreatic Insufficiency</mesh_term>
    <mesh_term>Lung Diseases</mesh_term>
    <mesh_term>Respiratory Tract Diseases</mesh_term>
    <mesh_term>Fibrosis</mesh_term>
  </condition_browse>
  <!-- Results have not yet been posted, but a results disposition has been submitted           -->
</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/NCT01710644
org study id: NM-BL-101
secondary id: 207862
nct id: NCT01710644
lead sponsor: 
collaborator: 
has dmc: Yes
The purpose of this Randomized, Double-blind, Multicenter, Two-period Crossover Study is to
      Assess the Efficacy and Tolerability of Burlulipase (NM-BL) in Patients with Exocrine
      Pancreatic Insufficiency due to Cystic Fibrosis
allocation: Randomized
intervention model: Crossover Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: To evaluate the efficacy of NM-BL compared to placebo in patients with Exocrine Pancreatic Insufficiency (EPI) due to CF, where the primary variable is coefficient of fat absorption (CFA%)
time frame: 72 hrs
measure: To evaluate the efficacy of NM-BL compared to placebo in patients with Exocrine Pancreatic Insufficiency (EPI)due to CF, where the primary variable is coefficient of nitrogen absorption (CNA%)
time frame: 72 hrs
measure: Symptomatology / Symptom Questionnaire
time frame: 7 days
description: Comparison gastrointestinal parameters, recorded by the patient/carer in the Symptom Questionnaire each day during the Treatment Periods
arm group label: Burlulipase
arm group type: Experimental
description: Burlulipase orally, per meal
arm group label: Placebo (Caramel in sterile water)
arm group type: Placebo Comparator
description: Placebo orally, per meal
intervention type: Drug
intervention name: Burlulipase
description: Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days
arm group label: Burlulipase
other name: NM-BL
intervention type: Drug
intervention name: Placebo (Caramel in sterile water)
description: Placebo will be taken with meals and snacks for 5 to 7 days
arm group label: Placebo (Caramel in sterile water)
other name: Placebo
criteria: 
gender: All
minimum age: 12 Years
maximum age: N/A
healthy volunteers: No
last name: Kristin Forssmann, MD
role: Study Director
affiliation: Nordmark Arzneimittel GmbH & Co. KG
last name: James E. Heubi, MD
role: Principal Investigator
affiliation: Children's Hospital Medical Center, Cincinnati
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: United States
responsible party type: Sponsor
mesh term: Cystic Fibrosis
mesh term: Digestive System Diseases
mesh term: Gastrointestinal Diseases
mesh term: Exocrine Pancreatic Insufficiency
mesh term: Lung Diseases
mesh term: Respiratory Tract Diseases
mesh term: Fibrosis