xml: <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/NCT05995717</url>
    </required_header>
    <id_info>
      <org_study_id>PKU-PAED-LIQ-2022-10-07</org_study_id>
      <secondary_id>327641</secondary_id>
      <secondary_id>23/NW/0185</secondary_id>
      <nct_id>NCT05995717</nct_id>
    </id_info>
    <brief_title>Evaluation of PKU UP</brief_title>
    <acronym>PKU UP</acronym>
    <official_title>An Acceptability Study to Evaluate the Compliance, Gastrointestinal Tolerance, Palatability and Metabolic Control of Children With Phenylketonuria (PKU) When Using PKU UP (a Food for Special Medical Purposes) as Part of Dietary Management.</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Vitaflo International, Ltd</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
      <collaborator>
        <agency>Birmingham Women's and Children's NHS Foundation Trust</agency>
        <agency_class>Other</agency_class>
      </collaborator>
    </sponsors>
    <source>Vitaflo International, Ltd</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>
        PKU UP is a prospective, single-arm, open-label, 26-week acceptability study to evaluate PKU&#xD;
        UP for the dietary management of participants with phenylketonuria (PKU). Up to 15&#xD;
        participants aged 1 - 10 years old will be recruited and it is anticipated the study will&#xD;
        open in at least three sites in the United Kingdom (UK) to recruit the target number of&#xD;
        participants in the required timeframe.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        PKU is a rare inborn error of metabolism with a prevalence of 1 in 10,000 in the UK&#xD;
        population. The mainstay of treatment for most individuals is dietary therapy with a strict&#xD;
        protein-restricted diet. We propose to recruit children with a diagnosis of PKU aged 1-10&#xD;
        years.&#xD;
  &#xD;
        The study product, PKU UP, is a food for special medical purposes (FSMP), as defined by the&#xD;
        Delegated Act EU 2016/128, used for the dietary management of Phenylketonuria (PKU).&#xD;
  &#xD;
        The study will be investigating the acceptability of PKU UP, as defined by the Advisory&#xD;
        Committee on Borderline Substances (ACBS). This includes the following:&#xD;
  &#xD;
          -  participant adherence to recommended intakes&#xD;
  &#xD;
          -  gastrointestinal symptoms&#xD;
  &#xD;
          -  evaluations of palatability.&#xD;
  &#xD;
        Each participant will be on the study for 26 weeks. This includes evaluation part 1 and&#xD;
        evaluation part 2:&#xD;
  &#xD;
          -  Part 1 consists of a 12 week period where participants introduce the study product into&#xD;
             their diet. Gastrointestinal tolerance, metabolic control, compliance, product&#xD;
             acceptability and dietary quality will be evaluated.&#xD;
  &#xD;
          -  Part 2 consists of a 14 week period where participants continue to take the study&#xD;
             product for a longer assessment of growth and nutritional status.&#xD;
  &#xD;
        Data collection will be performed using paper and/or electronic Case Report Forms completed&#xD;
        by the investigators at the baseline, evaluation periods and end of study visits. There will&#xD;
        also be three questionnaires completed by the parents/guardians over the course of the study,&#xD;
        in relation to protein substitute intake, gastrointestinal tolerance, and product&#xD;
        acceptability.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">October 16, 2023</start_date>
    <completion_date type="Anticipated">June 16, 2025</completion_date>
    <primary_completion_date type="Anticipated">April 16, 2025</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>Treatment</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Change in phe level between baseline and visit 3</measure>
      <time_frame>26 weeks</time_frame>
      <description>Change in phe level as measured by dried blood spot testing</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in tyrosine level between baseline and visit 3</measure>
      <time_frame>26 weeks</time_frame>
      <description>Change in tyrosine level as measured by dried blood spot testing</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in PI impression of metabolic control</measure>
      <time_frame>Week 0, week 6, week 12, week 26</time_frame>
      <description>Assessment of the participant's metabolic control in the opinion of the investigator</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in the growth of participants as measured by Height (length in under 2 years of age), Head circumference in under 2 years of age and Weight.</measure>
      <time_frame>Week 0, week 12, week 26</time_frame>
      <description>Assessed via anthropometry measurements. Participants will be weighed and measured for growth monitoring.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in GI tolerance (participant)</measure>
      <time_frame>Week 0, week 6, week 12, week 26</time_frame>
      <description>Gastrointestinal tolerance assessed using the PedsQL Gastrointestinal Symptoms Scale completed by the participants</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in PI impression of GI tolerance</measure>
      <time_frame>Week 0, week 6, week 12, week 26</time_frame>
      <description>Assessment of the participant's GI tolerance in the opinion of the investigator</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in compliance with study product consumption</measure>
      <time_frame>Week 0, week 6, week 12, week 26</time_frame>
      <description>Assessed via participant diary regarding study product intake</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in PI impression of study product intake</measure>
      <time_frame>Week 0, week 6, week 12, week 26</time_frame>
      <description>Assessment of the participant's compliance in the opinion of the investigator</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in product acceptability</measure>
      <time_frame>Week 0, week 12, week 26</time_frame>
      <description>Assessed via a product acceptability questionnaire completed by the participant/parent</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in dietary intake</measure>
      <time_frame>Week 0, week 12, week 26</time_frame>
      <description>Assessed via 24-hour recall and low protein food frequency questionnaire</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in nutritional biochemistry</measure>
      <time_frame>Week 0, week 26</time_frame>
      <description>Assessed via venous blood sample of approximately 7ml</description>
    </primary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">15</enrollment>
    <condition>PKU</condition>
    <arm_group>
      <arm_group_label>PKU UP</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>All participants will be assessed by their dietitian and prescribed an appropriate amount of the study product, PKU UP, to manage their phenylketonuria.</description>
    </arm_group>
    <intervention>
      <intervention_type>Dietary Supplement</intervention_type>
      <intervention_name>PKU UP</intervention_name>
      <description>PKU UP is a food for special medical purposes (FSMP). This product is for use in the dietary management of Phenylketonuria (PKU) in children aged from one (1) to ten (10) years.&#xD;
  PKU UP is a neutral-flavoured, ready-to-drink, low-phenylalanine protein substitute containing a blend of glycomacropeptide (GMP) isolate, essential and non-essential amino acids, fat, carbohydrate, vitamins, minerals and docosahexaenoic acid (DHA).</description>
      <arm_group_label>PKU UP</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Diagnosis of PKU requiring a low-protein diet and protein substitute.&#xD;
  &#xD;
            -  Participants who are adherent with their blood phenylalanine (Phe) testing, in the&#xD;
               opinion of the investigator.&#xD;
  &#xD;
            -  Participants aged 1-10 years.&#xD;
  &#xD;
            -  Participants who are anticipated to be able to take at least half of their daily&#xD;
               protein equivalent requirement from PKU UP.&#xD;
  &#xD;
            -  Willingly given, written, informed consent from the parent(s)/guardian(s).&#xD;
  &#xD;
            -  Willingly given, written assent by the participant (if appropriate).&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Inability to comply with the study protocol, in the opinion of the investigator.&#xD;
  &#xD;
            -  Any co-morbidity, which, in the opinion of the investigator, would preclude&#xD;
               participation in the study.&#xD;
  &#xD;
            -  Diagnosis of persistent hyperphenylalaninemia, or mild PKU not requiring a low protein&#xD;
               diet and protein substitute.&#xD;
  &#xD;
            -  Allergy or intolerance to milk.&#xD;
  &#xD;
            -  Participants who are currently participating in, plan to participate in or have&#xD;
               participated in an interventional investigational drug, food or medical device study&#xD;
               within 30 days prior to the screening visit.&#xD;
  &#xD;
            -  Use of additional micronutrient supplements during the evaluation period, unless&#xD;
               clinically indicated and prescribed by the Investigator (must be recorded in both the&#xD;
               medical records and case report form).&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>1 Year</minimum_age>
      <maximum_age>10 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Anita MacDonald</last_name>
      <role>Principal Investigator</role>
      <affiliation>Birmingham Women's and Children's NHS Foundation Trust</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Binyam Tesfaye</last_name>
      <phone>0151 709 9020</phone>
      <email>Binyam.Tesfaye@Vitaflo.co.uk</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Erika Brennan</last_name>
      <phone>0151 709 9020</phone>
      <email>ClinicalTrialsTeam@Vitaflo.co.uk</email>
    </overall_contact_backup>
    <location>
      <facility>
        <name>Royal Belfast Hospital for Sick Children</name>
        <address>
          <city>Belfast</city>
          <zip>BT12 6BE</zip>
          <country>United Kingdom</country>
        </address>
      </facility>
      <contact>
        <last_name>Anne Grimsley</last_name>
        <phone>07936 877 113</phone>
        <email>Anne.Grimsley@BelfastTrust.HSCNI.net</email>
      </contact>
    </location>
    <location>
      <facility>
        <name>Birmingham Children's Hospital</name>
        <address>
          <city>Birmingham</city>
          <zip>B4 6NH</zip>
          <country>United Kingdom</country>
        </address>
      </facility>
      <contact>
        <last_name>Anita MacDonald</last_name>
        <phone>0121 333 8024</phone>
        <email>Anita.MacDonald@NHS.net</email>
      </contact>
    </location>
    <location>
      <facility>
        <name>University Hospitals Bristol and Weston NHS Foundation Trust</name>
        <address>
          <city>Bristol</city>
          <zip>BS2 8BJ</zip>
          <country>United Kingdom</country>
        </address>
      </facility>
      <contact>
        <last_name>Camille Newby</last_name>
        <phone>0117 342 8011</phone>
        <email>Camille.Newby@UHBW.NHS.UK</email>
      </contact>
    </location>
    <location>
      <facility>
        <name>Great Ormond Street Hospital for Children NHS Foundation Trust</name>
        <address>
          <city>London</city>
          <zip>WC1N 3JH</zip>
          <country>United Kingdom</country>
        </address>
      </facility>
      <contact>
        <last_name>Louise van Dorp</last_name>
        <phone>0207 813 8549</phone>
        <email>Louise.VanDorp@GOSH.NHS.UK</email>
      </contact>
    </location>
    <location_countries>
      <country>United Kingdom</country>
    </location_countries>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 3, 2023</study_first_submitted>
    <study_first_submitted_qc>August 15, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 16, 2023</study_first_posted>
    <last_update_submitted>August 15, 2023</last_update_submitted>
    <last_update_submitted_qc>August 15, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 16, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>Phenylketonuria</keyword>
    <keyword>PKU</keyword>
    <keyword>Glyco-Macro-Peptide</keyword>
    <keyword>GMP</keyword>
    <keyword>amino acid</keyword>
    <keyword>AA</keyword>
    <keyword>protein substitute</keyword>
    <keyword>PS</keyword>
    <keyword>United Kingdom</keyword>
    <keyword>UK</keyword>
    <keyword>children</keyword>
    <keyword>PKU UP</keyword>
    <keyword>Glycomacropeptide</keyword>
    <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>
  
  
text: 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/NCT05995717
  org study id: PKU-PAED-LIQ-2022-10-07
  secondary id: 327641
  secondary id: 23/NW/0185
  nct id: NCT05995717
  lead sponsor: 
  collaborator: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  PKU UP is a prospective, single-arm, open-label, 26-week acceptability study to evaluate PKU
        UP for the dietary management of participants with phenylketonuria (PKU). Up to 15
        participants aged 1 - 10 years old will be recruited and it is anticipated the study will
        open in at least three sites in the United Kingdom (UK) to recruit the target number of
        participants in the required timeframe.
  PKU is a rare inborn error of metabolism with a prevalence of 1 in 10,000 in the UK
        population. The mainstay of treatment for most individuals is dietary therapy with a strict
        protein-restricted diet. We propose to recruit children with a diagnosis of PKU aged 1-10
        years.
  
        The study product, PKU UP, is a food for special medical purposes (FSMP), as defined by the
        Delegated Act EU 2016/128, used for the dietary management of Phenylketonuria (PKU).
  
        The study will be investigating the acceptability of PKU UP, as defined by the Advisory
        Committee on Borderline Substances (ACBS). This includes the following:
  
          -  participant adherence to recommended intakes
  
          -  gastrointestinal symptoms
  
          -  evaluations of palatability.
  
        Each participant will be on the study for 26 weeks. This includes evaluation part 1 and
        evaluation part 2:
  
          -  Part 1 consists of a 12 week period where participants introduce the study product into
             their diet. Gastrointestinal tolerance, metabolic control, compliance, product
             acceptability and dietary quality will be evaluated.
  
          -  Part 2 consists of a 14 week period where participants continue to take the study
             product for a longer assessment of growth and nutritional status.
  
        Data collection will be performed using paper and/or electronic Case Report Forms completed
        by the investigators at the baseline, evaluation periods and end of study visits. There will
        also be three questionnaires completed by the parents/guardians over the course of the study,
        in relation to protein substitute intake, gastrointestinal tolerance, and product
        acceptability.
  allocation: N/A
  intervention model: Single Group Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Change in phe level between baseline and visit 3
  time frame: 26 weeks
  description: Change in phe level as measured by dried blood spot testing
  measure: Change in tyrosine level between baseline and visit 3
  time frame: 26 weeks
  description: Change in tyrosine level as measured by dried blood spot testing
  measure: Change in PI impression of metabolic control
  time frame: Week 0, week 6, week 12, week 26
  description: Assessment of the participant's metabolic control in the opinion of the investigator
  measure: Change in the growth of participants as measured by Height (length in under 2 years of age), Head circumference in under 2 years of age and Weight.
  time frame: Week 0, week 12, week 26
  description: Assessed via anthropometry measurements. Participants will be weighed and measured for growth monitoring.
  measure: Change in GI tolerance (participant)
  time frame: Week 0, week 6, week 12, week 26
  description: Gastrointestinal tolerance assessed using the PedsQL Gastrointestinal Symptoms Scale completed by the participants
  measure: Change in PI impression of GI tolerance
  time frame: Week 0, week 6, week 12, week 26
  description: Assessment of the participant's GI tolerance in the opinion of the investigator
  measure: Change in compliance with study product consumption
  time frame: Week 0, week 6, week 12, week 26
  description: Assessed via participant diary regarding study product intake
  measure: Change in PI impression of study product intake
  time frame: Week 0, week 6, week 12, week 26
  description: Assessment of the participant's compliance in the opinion of the investigator
  measure: Change in product acceptability
  time frame: Week 0, week 12, week 26
  description: Assessed via a product acceptability questionnaire completed by the participant/parent
  measure: Change in dietary intake
  time frame: Week 0, week 12, week 26
  description: Assessed via 24-hour recall and low protein food frequency questionnaire
  measure: Change in nutritional biochemistry
  time frame: Week 0, week 26
  description: Assessed via venous blood sample of approximately 7ml
  arm group label: PKU UP
  arm group type: Experimental
  description: All participants will be assessed by their dietitian and prescribed an appropriate amount of the study product, PKU UP, to manage their phenylketonuria.
  intervention type: Dietary Supplement
  intervention name: PKU UP
  description: PKU UP is a food for special medical purposes (FSMP). This product is for use in the dietary management of Phenylketonuria (PKU) in children aged from one (1) to ten (10) years.
  PKU UP is a neutral-flavoured, ready-to-drink, low-phenylalanine protein substitute containing a blend of glycomacropeptide (GMP) isolate, essential and non-essential amino acids, fat, carbohydrate, vitamins, minerals and docosahexaenoic acid (DHA).
  arm group label: PKU UP
  criteria: 
  gender: All
  minimum age: 1 Year
  maximum age: 10 Years
  healthy volunteers: No
  last name: Anita MacDonald
  role: Principal Investigator
  affiliation: Birmingham Women's and Children's NHS Foundation Trust
  last name: Binyam Tesfaye
  phone: 0151 709 9020
  email: Binyam.Tesfaye@Vitaflo.co.uk
  last name: Erika Brennan
  phone: 0151 709 9020
  email: ClinicalTrialsTeam@Vitaflo.co.uk
  facility: 
  contact: 
  facility: 
  contact: 
  facility: 
  contact: 
  facility: 
  contact: 
  country: United Kingdom
  responsible party type: Sponsor
  mesh term: Phenylketonurias
  sharing ipd: No