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/NCT05975684</url>
    </required_header>
    <id_info>
      <org_study_id>STUDY00002491</org_study_id>
      <nct_id>NCT05975684</nct_id>
    </id_info>
    <brief_title>Baclofen for Children With Rumination Syndrome</brief_title>
    <official_title>A Randomized Controlled Pilot Trial of Baclofen for Children With Rumination Syndrome</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Nationwide Children's Hospital</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Nationwide Children's Hospital</source>
    <oversight_info>
      <has_dmc>No</has_dmc>
      <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
      <is_us_export>Yes</is_us_export>
    </oversight_info>
    <brief_summary>
      <textblock>
        The goal of this clinical trial is to learn about baclofen in pediatric patients with&#xD;
        rumination syndrome. The main question it aims to answer is whether baclofen is effective in&#xD;
        treating children with rumination syndrome. Participants will be asked to take baclofen or&#xD;
        placebo for 4 weeks and fill out surveys regarding symptoms.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        Rumination syndrome is a functional gastrointestinal disorder characterized by effortless,&#xD;
        repetitive regurgitation of recently ingested meal. The objective of this study is to&#xD;
        evaluate the efficacy of baclofen, a GABA-b agonist, in children with rumination syndrome. We&#xD;
        hypothesize that children with rumination syndrome who are treated with baclofen in addition&#xD;
        to standard behavioral treatment will have greater clinical improvement compared to those who&#xD;
        receive placebo.&#xD;
  &#xD;
        After enrollment into the study, participants will be randomized into one of two parallel&#xD;
        groups in a 1:1 ratio: (a) baclofen or (b) placebo control. The research team will be blinded&#xD;
        to randomization. Participants will subsequently meet with investigational pharmacist and&#xD;
        receive four-week supply of either baclofen 0.5 mg/kg/day up to 15 mg divided three times a&#xD;
        day or matching placebo three times a day in liquid formulation. At baseline, we will collect&#xD;
        information on demographics, medical, and surgical history, relevant diagnostic testing, and&#xD;
        relevant past treatments (including working with a psychologist). Patients/families will&#xD;
        complete a baseline questionnaire (rumination severity questions, PedsQL, route of nutrition,&#xD;
        medications) and then the patient will start taking study drug or placebo in addition to&#xD;
        their usual care as determined by the patient's primary gastroenterologist.&#xD;
        Participants/families will complete follow-up questionnaires at week 1, week 2, week 4, and&#xD;
        then week 8. The questionnaires will be filled out by the patient or guardian electronically&#xD;
        via REDCap. We will closely monitor for side effects. In addition to asking about any side&#xD;
        effects at follow-up timepoints, we will also emphasize that families should contact us using&#xD;
        the research team phone number with any instances of suspected side effects even before the&#xD;
        next follow-up timepoint. After the 4-week drug or placebo period, participants are free to&#xD;
        start or continue baclofen, or use other treatment modalities, as determined by their primary&#xD;
        GI team.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Recruiting</overall_status>
    <start_date type="Actual">January 31, 2023</start_date>
    <completion_date type="Anticipated">April 2024</completion_date>
    <primary_completion_date type="Anticipated">December 2023</primary_completion_date>
    <phase>Phase 3</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>Parallel groups of (a) baclofen or (b) placebo control</intervention_model_description>
      <primary_purpose>Treatment</primary_purpose>
      <masking>Triple (Participant, Care Provider, Investigator)</masking>
      <masking_description>Participants will be randomized into one of two parallel groups in 1:1 ratio. The specific details of randomization sequence will be managed by investigational pharmacy. The participant, care provider, and research team are blinded to randomization.</masking_description>
    </study_design_info>
    <primary_outcome>
      <measure>Vomiting once a week or less</measure>
      <time_frame>We will assess this specific metric at the end of four weeks.</time_frame>
      <description>Percentage of patients in each group who are vomiting once a week or less. This will be evaluated using a rumination severity survey.</description>
    </primary_outcome>
    <number_of_arms>2</number_of_arms>
    <enrollment type="Anticipated">50</enrollment>
    <condition>Rumination Syndrome</condition>
    <arm_group>
      <arm_group_label>Baclofen</arm_group_label>
      <arm_group_type>Active Comparator</arm_group_type>
      <description>Baclofen 0.5 mg/kg/day up to 15 mg/day divided three times a day in liquid formulation for 4 weeks.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Placebo</arm_group_label>
      <arm_group_type>Placebo Comparator</arm_group_type>
      <description>Matching placebo three times a day in liquid formulation for 4 weeks.</description>
    </arm_group>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Baclofen</intervention_name>
      <description>0.5 mg/kg/day up to 15 mg/day divided three times a day for 4 weeks</description>
      <arm_group_label>Baclofen</arm_group_label>
    </intervention>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Placebo</intervention_name>
      <description>Placebo three times a day for 4 weeks</description>
      <arm_group_label>Placebo</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Children 4-18 years of age who meets Rome IV diagnostic criteria for rumination&#xD;
               syndrome and are see at NCH GI clinic&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Patients who have contraindications to baclofen due to medical history and/or current&#xD;
               medications&#xD;
  &#xD;
            -  Patients who are non- English speaking&#xD;
  &#xD;
            -  Patients who are pregnant&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>4 Years</minimum_age>
      <maximum_age>18 Years</maximum_age>
      <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
    </eligibility>
    <overall_contact>
      <last_name>Peter Lu, MD, MS</last_name>
      <phone>6147223450</phone>
      <email>peter.lu@nationwidechildrens.org</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Janice Khoo, MD</last_name>
      <phone>6147223450</phone>
      <email>janice.khoo@nationwidechildrens.org</email>
    </overall_contact_backup>
    <location>
      <facility>
        <name>Nationwide Children's Hospital</name>
        <address>
          <city>Columbus</city>
          <state>Ohio</state>
          <zip>73205</zip>
          <country>United States</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Janice Khoo, MD</last_name>
        <phone>614-722-3450</phone>
      </contact>
    </location>
    <location_countries>
      <country>United States</country>
    </location_countries>
    <verification_date>July 2023</verification_date>
    <study_first_submitted>July 27, 2023</study_first_submitted>
    <study_first_submitted_qc>July 27, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 4, 2023</study_first_posted>
    <last_update_submitted>July 27, 2023</last_update_submitted>
    <last_update_submitted_qc>July 27, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 4, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Principal Investigator</responsible_party_type>
      <investigator_affiliation>Nationwide Children's Hospital</investigator_affiliation>
      <investigator_full_name>Peter Lu</investigator_full_name>
      <investigator_title>Associate Professor of Clinical Pediatrics</investigator_title>
    </responsible_party>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Rumination Syndrome</mesh_term>
      <mesh_term>Syndrome</mesh_term>
      <mesh_term>Feeding and Eating Disorders of Childhood</mesh_term>
    </condition_browse>
    <intervention_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Baclofen</mesh_term>
    </intervention_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/NCT05975684
  org study id: STUDY00002491
  nct id: NCT05975684
  lead sponsor: 
  has dmc: No
  is fda regulated drug: Yes
  is fda regulated device: No
  is us export: Yes
  The goal of this clinical trial is to learn about baclofen in pediatric patients with
        rumination syndrome. The main question it aims to answer is whether baclofen is effective in
        treating children with rumination syndrome. Participants will be asked to take baclofen or
        placebo for 4 weeks and fill out surveys regarding symptoms.
  Rumination syndrome is a functional gastrointestinal disorder characterized by effortless,
        repetitive regurgitation of recently ingested meal. The objective of this study is to
        evaluate the efficacy of baclofen, a GABA-b agonist, in children with rumination syndrome. We
        hypothesize that children with rumination syndrome who are treated with baclofen in addition
        to standard behavioral treatment will have greater clinical improvement compared to those who
        receive placebo.
  
        After enrollment into the study, participants will be randomized into one of two parallel
        groups in a 1:1 ratio: (a) baclofen or (b) placebo control. The research team will be blinded
        to randomization. Participants will subsequently meet with investigational pharmacist and
        receive four-week supply of either baclofen 0.5 mg/kg/day up to 15 mg divided three times a
        day or matching placebo three times a day in liquid formulation. At baseline, we will collect
        information on demographics, medical, and surgical history, relevant diagnostic testing, and
        relevant past treatments (including working with a psychologist). Patients/families will
        complete a baseline questionnaire (rumination severity questions, PedsQL, route of nutrition,
        medications) and then the patient will start taking study drug or placebo in addition to
        their usual care as determined by the patient's primary gastroenterologist.
        Participants/families will complete follow-up questionnaires at week 1, week 2, week 4, and
        then week 8. The questionnaires will be filled out by the patient or guardian electronically
        via REDCap. We will closely monitor for side effects. In addition to asking about any side
        effects at follow-up timepoints, we will also emphasize that families should contact us using
        the research team phone number with any instances of suspected side effects even before the
        next follow-up timepoint. After the 4-week drug or placebo period, participants are free to
        start or continue baclofen, or use other treatment modalities, as determined by their primary
        GI team.
  allocation: Randomized
  intervention model: Parallel Assignment
  intervention model description: Parallel groups of (a) baclofen or (b) placebo control
  primary purpose: Treatment
  masking: Triple (Participant, Care Provider, Investigator)
  masking description: Participants will be randomized into one of two parallel groups in 1:1 ratio. The specific details of randomization sequence will be managed by investigational pharmacy. The participant, care provider, and research team are blinded to randomization.
  measure: Vomiting once a week or less
  time frame: We will assess this specific metric at the end of four weeks.
  description: Percentage of patients in each group who are vomiting once a week or less. This will be evaluated using a rumination severity survey.
  arm group label: Baclofen
  arm group type: Active Comparator
  description: Baclofen 0.5 mg/kg/day up to 15 mg/day divided three times a day in liquid formulation for 4 weeks.
  arm group label: Placebo
  arm group type: Placebo Comparator
  description: Matching placebo three times a day in liquid formulation for 4 weeks.
  intervention type: Drug
  intervention name: Baclofen
  description: 0.5 mg/kg/day up to 15 mg/day divided three times a day for 4 weeks
  arm group label: Baclofen
  intervention type: Drug
  intervention name: Placebo
  description: Placebo three times a day for 4 weeks
  arm group label: Placebo
  criteria: 
  gender: All
  minimum age: 4 Years
  maximum age: 18 Years
  healthy volunteers: Accepts Healthy Volunteers
  last name: Peter Lu, MD, MS
  phone: 6147223450
  email: peter.lu@nationwidechildrens.org
  last name: Janice Khoo, MD
  phone: 6147223450
  email: janice.khoo@nationwidechildrens.org
  facility: 
  status: Recruiting
  contact: 
  country: United States
  responsible party type: Principal Investigator
  investigator affiliation: Nationwide Children's Hospital
  investigator full name: Peter Lu
  investigator title: Associate Professor of Clinical Pediatrics
  mesh term: Rumination Syndrome
  mesh term: Syndrome
  mesh term: Feeding and Eating Disorders of Childhood
  mesh term: Baclofen
  sharing ipd: No