<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/NCT03797131</url>
  </required_header>
  <id_info>
    <org_study_id>K013-118</org_study_id>
    <nct_id>NCT03797131</nct_id>
  </id_info>
  <brief_title>Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle Disorders</brief_title>
  <official_title>An Open Label Pilot Study to Evaluate the Effect of KB195, a Novel Prebiotic Gluco-Oligosaccharide Mixture, in the Diet of Patients With Urea Cycle Disorders on Gut Nitrogen Metabolism</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Kaleido Biosciences</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Kaleido Biosciences</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>
      This clinical food study aims to explore the effect of KB195, a novel mixture of&#xD;
      oligosaccharides, on the metabolism of nitrogen by the microbiome in patients with urea cycle&#xD;
      disorders (UCDs). This will be done using a stable isotope to assess nitrogen metabolism in&#xD;
      the blood, urine, and stool. The study will also assess the safety and tolerability of KB195&#xD;
      in patients with UCDs.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">January 6, 2019</start_date>
  <completion_date type="Actual">September 1, 2019</completion_date>
  <primary_completion_date type="Actual">September 1, 2019</primary_completion_date>
  <phase>N/A</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>Basic Science</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change in labelled (15N-nitrogen) and total nitrogen excretion in urine</measure>
    <time_frame>Baseline to Day 25</time_frame>
    <description>Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-nitrogen) and total nitrogen excretion in urine</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in labelled (15N-urea) and total urea excretion in urine</measure>
    <time_frame>Baseline to Day 25</time_frame>
    <description>Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-urea) and total urea excretion in urine</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in labelled (15N-ammonia) and total ammonia excretion in urine</measure>
    <time_frame>Baseline to Day 25</time_frame>
    <description>Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-ammonia) and total ammonia excretion in urine</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change in Gastrointestinal Tolerability Questionnaire (GITQ) through the collection of daily patient questionnaires</measure>
    <time_frame>Baseline to Day 32</time_frame>
    <description>Evaluate the effect of KB195 on self-report questionnaires including the Gastrointestinal Tolerability Questionnaire, an assessment of the frequency and severity of GI symptoms, e.g., gas, abdominal pain, calculated on a scale from 0 (None/Not applicable) to a maximum score of 60 (Severe/Much more than usual) for all questions</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in Bristol Stool Scale (BSS) through the collection of daily patient questionnaires</measure>
    <time_frame>Baseline to Day 32</time_frame>
    <description>Evaluate the effect of KB195 on self-report questionnaires including the Bristol Stool Scale, an assessment of stool consistency on a scale from 1 (separate hard lumps, like nuts, hard to pass) through 7 (watery, no solid pieces, entirely liquid).</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Incidence of adverse events</measure>
    <time_frame>Baseline to Day 32</time_frame>
  </other_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">4</enrollment>
  <condition>Urea Cycle Disorders</condition>
  <arm_group>
    <arm_group_label>KB195 Arm</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>KB195 (a novel mixture of oligosaccharides)</intervention_name>
    <description>KB195 (a novel mixture of oligosaccharides) for oral intake for 21 days.</description>
    <arm_group_label>KB195 Arm</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Confirmed UCD patient at any age ≥ 14 years&#xD;
&#xD;
          -  Parental/legal guardian permission or patient's written informed consent or assent, as&#xD;
             applicable&#xD;
&#xD;
          -  Be willing and able to comply with study requirements&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Any medical condition unrelated to the sequelae of UCD&#xD;
&#xD;
          -  Patient has N-acetylglutamate synthase (NAGS) deficiency&#xD;
&#xD;
          -  Recent hospitalization or risk for metabolic decompensation&#xD;
&#xD;
          -  Liver transplantation&#xD;
&#xD;
          -  Systemic antibiotics, probiotics or prebiotics that do not align with study guidances&#xD;
&#xD;
          -  Change in dose or frequency of any drug or other compound to modulate GI motility&#xD;
&#xD;
          -  Contraindications or known allergy/sensitivity to the use of the study products&#xD;
&#xD;
          -  Use of an investigational drug, product or device within 30 days prior to Screening&#xD;
             Visit, or current enrolment in another investigational drug, product, or device study&#xD;
&#xD;
          -  Considered to be at risk for noncompliance or unlikely for any reason to be able to&#xD;
             comply with the study procedures&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>14 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Mark Wingertzahn</last_name>
    <role>Study Director</role>
    <affiliation>Kaleido Biosciences</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University Children's Hospital Zurich</name>
      <address>
        <city>Zürich</city>
        <zip>CH-8032</zip>
        <country>Switzerland</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Switzerland</country>
  </location_countries>
  <verification_date>January 2020</verification_date>
  <study_first_submitted>December 7, 2018</study_first_submitted>
  <study_first_submitted_qc>January 4, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 9, 2019</study_first_posted>
  <last_update_submitted>January 8, 2020</last_update_submitted>
  <last_update_submitted_qc>January 8, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">January 10, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>KB195</keyword>
  <keyword>Urea Cycle Disorders</keyword>
  <keyword>UCD</keyword>
  <keyword>microbiome</keyword>
  <keyword>Kaleido</keyword>
  <keyword>ammonia</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Urea Cycle Disorders, Inborn</mesh_term>
  </condition_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/NCT03797131
org study id: K013-118
nct id: NCT03797131
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This clinical food study aims to explore the effect of KB195, a novel mixture of
      oligosaccharides, on the metabolism of nitrogen by the microbiome in patients with urea cycle
      disorders (UCDs). This will be done using a stable isotope to assess nitrogen metabolism in
      the blood, urine, and stool. The study will also assess the safety and tolerability of KB195
      in patients with UCDs.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Basic Science
masking: None (Open Label)
measure: Change in labelled (15N-nitrogen) and total nitrogen excretion in urine
time frame: Baseline to Day 25
description: Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-nitrogen) and total nitrogen excretion in urine
measure: Change in labelled (15N-urea) and total urea excretion in urine
time frame: Baseline to Day 25
description: Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-urea) and total urea excretion in urine
measure: Change in labelled (15N-ammonia) and total ammonia excretion in urine
time frame: Baseline to Day 25
description: Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-ammonia) and total ammonia excretion in urine
measure: Change in Gastrointestinal Tolerability Questionnaire (GITQ) through the collection of daily patient questionnaires
time frame: Baseline to Day 32
description: Evaluate the effect of KB195 on self-report questionnaires including the Gastrointestinal Tolerability Questionnaire, an assessment of the frequency and severity of GI symptoms, e.g., gas, abdominal pain, calculated on a scale from 0 (None/Not applicable) to a maximum score of 60 (Severe/Much more than usual) for all questions
measure: Change in Bristol Stool Scale (BSS) through the collection of daily patient questionnaires
time frame: Baseline to Day 32
description: Evaluate the effect of KB195 on self-report questionnaires including the Bristol Stool Scale, an assessment of stool consistency on a scale from 1 (separate hard lumps, like nuts, hard to pass) through 7 (watery, no solid pieces, entirely liquid).
measure: Incidence of adverse events
time frame: Baseline to Day 32
arm group label: KB195 Arm
arm group type: Experimental
intervention type: Other
intervention name: KB195 (a novel mixture of oligosaccharides)
description: KB195 (a novel mixture of oligosaccharides) for oral intake for 21 days.
arm group label: KB195 Arm
criteria: 
gender: All
minimum age: 14 Years
maximum age: N/A
healthy volunteers: No
last name: Mark Wingertzahn
role: Study Director
affiliation: Kaleido Biosciences
facility: 
country: Switzerland
responsible party type: Sponsor
mesh term: Urea Cycle Disorders, Inborn
sharing ipd: No