<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/NCT03792451</url>
  </required_header>
  <id_info>
    <org_study_id>P00029611</org_study_id>
    <nct_id>NCT03792451</nct_id>
  </id_info>
  <brief_title>Nutrition Status of Adults Treated With Pegvaliase</brief_title>
  <official_title>Nutrition Status of Adults With Phenylketonuria Treated With Pegvaliase</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Boston Children's Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>BioMarin Pharmaceutical</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Boston Children's Hospital</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>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      Conduct a cross-sectional analysis of the nutritional status of individuals with PKU treated&#xD;
      with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more&#xD;
      months while on pegvaliase treatment. We will evaluate laboratory markers of nutritional&#xD;
      status, including markers of protein and micronutrient nutriture. In addition, a three-day&#xD;
      food record and food frequency questionnaire will be obtained to assess protein intake and&#xD;
      overall diet quality.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">January 17, 2019</start_date>
  <completion_date type="Actual">December 31, 2020</completion_date>
  <primary_completion_date type="Actual">December 31, 2020</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>Cross-Sectional</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Mean concentrations of serum marker(s) of protein status</measure>
    <time_frame>through study completion, an average of 1 year</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean concentrations of serum marker(s) of micronutrient status</measure>
    <time_frame>through study completion, an average of 1 year</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean concentrations of serum marker(s) of essential fatty acid status</measure>
    <time_frame>through study completion, an average of 1 year</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean concentrations of serum marker(s) of cardiovascular status</measure>
    <time_frame>through study completion, an average of 1 year</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean protein intake (g/day) as reported on a three-day food record</measure>
    <time_frame>through study completion, an average of 1 year</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean micronutrient intake (%DRI) as reported on a three-day food record</measure>
    <time_frame>through study completion, an average of 1 year</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Mean Healthy Eating Index-2015 scores calculated from the Diet History Questionnaire III</measure>
    <time_frame>through study completion, an average of 1 year</time_frame>
    <description>&quot;The Healthy Eating Index-2015 (HEI-2015) is… used to assess how well a set of foods aligns with the 2015-2020 Dietary Guidelines for Americans. The HEI-2015 includes 13 components that can be summed to a maximum total score of 100 points. The components capture… adequacy components and… moderation components. For the adequacy components, higher scores reflect higher intakes that meet or exceed the standards. For the moderation components, higher scores reflect lower intakes because lower intakes are more desirable. A higher total score indicates a diet that aligns better with the Dietary Guidelines.&quot; - https://www.cnpp.usda.gov/sites/default/files/healthy_eating_index/HEI2015-ScoresForAmericans.pdf&#xD;
Adequacy components include total fruits, whole fruits, total vegetables, greens and beans, whole grains, dairy, total protein foods, seafood and plant proteins, fatty acids. Moderation components include refined grains, sodium, added sugars, and saturated fats.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Correlation between serum micronutrient concentrations and average micronutrient intake</measure>
    <time_frame>through study completion, an average of 1 year</time_frame>
  </secondary_outcome>
  <enrollment type="Actual">18</enrollment>
  <condition>Phenylketonurias</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Pegvaliase</intervention_name>
    <description>Treated with pegvaliase for one or more years or followed an unrestricted diet for 6 or more months while being treated with pegvaliase.</description>
    <other_name>Palynziq</other_name>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        This cross-sectional study will recruit adults with PKU who have been treated with&#xD;
        pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more&#xD;
        months while on pegvaliase treatment. This may include individuals who participated in the&#xD;
        phase III clinical trials for pegvaliase.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Diagnosis of phenylketonuria&#xD;
&#xD;
          2. Aged 18-65 years&#xD;
&#xD;
          3. Currently treated with pegvaliase&#xD;
&#xD;
          4. Have either 1) received treatment with pegvaliase for one or more years or 2) followed&#xD;
             an unrestricted diet for 6 or more months while on pegvaliase&#xD;
&#xD;
          5. Currently following an unrestricted diet that meets or exceeds the RDA for protein&#xD;
             (0.8 g/kg/day)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Unable to consent to study&#xD;
&#xD;
          2. Under age 18 years or over age 65&#xD;
&#xD;
          3. Routine food intake provides less than the RDA for protein (0.8 g/kg/d)&#xD;
&#xD;
          4. Consuming any PKU medical foods&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>65 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Krista Viau, PhD, RD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Boston Children's Hospital</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Boston Children's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>March 2021</verification_date>
  <study_first_submitted>December 31, 2018</study_first_submitted>
  <study_first_submitted_qc>January 2, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 3, 2019</study_first_posted>
  <last_update_submitted>March 2, 2021</last_update_submitted>
  <last_update_submitted_qc>March 2, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">March 4, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Boston Children's Hospital</investigator_affiliation>
    <investigator_full_name>Krista Viau</investigator_full_name>
    <investigator_title>Clinical Nutrition Specialist II</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Phenylketonurias</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/NCT03792451
org study id: P00029611
nct id: NCT03792451
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
is us export: No
Conduct a cross-sectional analysis of the nutritional status of individuals with PKU treated
      with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more
      months while on pegvaliase treatment. We will evaluate laboratory markers of nutritional
      status, including markers of protein and micronutrient nutriture. In addition, a three-day
      food record and food frequency questionnaire will be obtained to assess protein intake and
      overall diet quality.
observational model: Cohort
time perspective: Cross-Sectional
measure: Mean concentrations of serum marker(s) of protein status
time frame: through study completion, an average of 1 year
measure: Mean concentrations of serum marker(s) of micronutrient status
time frame: through study completion, an average of 1 year
measure: Mean concentrations of serum marker(s) of essential fatty acid status
time frame: through study completion, an average of 1 year
measure: Mean concentrations of serum marker(s) of cardiovascular status
time frame: through study completion, an average of 1 year
measure: Mean protein intake (g/day) as reported on a three-day food record
time frame: through study completion, an average of 1 year
measure: Mean micronutrient intake (%DRI) as reported on a three-day food record
time frame: through study completion, an average of 1 year
measure: Mean Healthy Eating Index-2015 scores calculated from the Diet History Questionnaire III
time frame: through study completion, an average of 1 year
description: "The Healthy Eating Index-2015 (HEI-2015) is… used to assess how well a set of foods aligns with the 2015-2020 Dietary Guidelines for Americans. The HEI-2015 includes 13 components that can be summed to a maximum total score of 100 points. The components capture… adequacy components and… moderation components. For the adequacy components, higher scores reflect higher intakes that meet or exceed the standards. For the moderation components, higher scores reflect lower intakes because lower intakes are more desirable. A higher total score indicates a diet that aligns better with the Dietary Guidelines." - https://www.cnpp.usda.gov/sites/default/files/healthy_eating_index/HEI2015-ScoresForAmericans.pdf
Adequacy components include total fruits, whole fruits, total vegetables, greens and beans, whole grains, dairy, total protein foods, seafood and plant proteins, fatty acids. Moderation components include refined grains, sodium, added sugars, and saturated fats.
measure: Correlation between serum micronutrient concentrations and average micronutrient intake
time frame: through study completion, an average of 1 year
intervention type: Drug
intervention name: Pegvaliase
description: Treated with pegvaliase for one or more years or followed an unrestricted diet for 6 or more months while being treated with pegvaliase.
other name: Palynziq
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: 65 Years
healthy volunteers: No
last name: Krista Viau, PhD, RD
role: Principal Investigator
affiliation: Boston Children's Hospital
facility: 
country: United States
responsible party type: Principal Investigator
investigator affiliation: Boston Children's Hospital
investigator full name: Krista Viau
investigator title: Clinical Nutrition Specialist II
mesh term: Phenylketonurias
sharing ipd: No