<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/NCT03245762</url>
  </required_header>
  <id_info>
    <org_study_id>IRB201700423 -A</org_study_id>
    <secondary_id>OCR16237</secondary_id>
    <nct_id>NCT03245762</nct_id>
  </id_info>
  <brief_title>Intranasal Oxytocin for Infants With Prader-Willi Syndrome</brief_title>
  <official_title>Intranasal Oxytocin for Treatment of Infants and Children With Prader-Willi Syndrome in Nutritional Phase 1a - Phase 2 Study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University of Florida</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Prader-Willi Syndrome Association</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>University of Florida</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to compare the change in suck and swallow competency from&#xD;
      baseline to morning of day 6 with intranasal oxytocin spray vs placebo in infants/children&#xD;
      with Prader-Willi Syndrome who are in nutritional phase 1a. Videofluoroscopic swallow studies&#xD;
      will be performed on treatment day 1 and on the day following treatment morning of day 6.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The overall objective of this Phase 2 trial is to compare the change from baseline to morning&#xD;
      of day 6 of Internasal Oxytocin (IN-OT) on suck and swallow competency in infants/children&#xD;
      with Prader-Willi Syndrome (PWS) who are in nutritional phase 1a.&#xD;
&#xD;
      Study Hypothesis 1: The Study team hypothesize that replacing Oxytocin (OT) in infants and&#xD;
      children who are in nutritional phase 1a will improve their suck and swallow, potentially&#xD;
      even eliminating the need for gastrostomy tubes and nasogastric tubes for feeding, and&#xD;
      decreasing the risk of aspiration with oral feeding.&#xD;
&#xD;
      Study Hypothesis 2: The Study team hypothesize that replacing OT in infants and children with&#xD;
      PWS will result in improved eye contact, daytime alertness, and feelings of bonding between&#xD;
      the parents and the infant.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">August 1, 2017</start_date>
  <completion_date type="Actual">January 4, 2018</completion_date>
  <primary_completion_date type="Actual">January 4, 2018</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>Parallel Assignment</intervention_model>
    <primary_purpose>Basic Science</primary_purpose>
    <masking>Double (Care Provider, Outcomes Assessor)</masking>
    <masking_description>This will be a double-blinded study, as no studies to date have compared this treatment in infants with placebo. Board-certified speech and language pathologist who will be blinded to the treatment arm of each patient.</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a</measure>
    <time_frame>baseline to day 5</time_frame>
    <description>Swallow study Overall improvement</description>
  </primary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">15</enrollment>
  <condition>Prader-Willi Syndrome</condition>
  <arm_group>
    <arm_group_label>Intranasal oxytocin</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>IN-placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Intervention: 4 IU/day of placebo via nasal spray device each morning.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Oxytocin</intervention_name>
    <description>4 IU/day of oxytocin administered via nasal spray device each morning.</description>
    <arm_group_label>Intranasal oxytocin</arm_group_label>
    <other_name>Syntocinon</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>4 IU/day of placebo administered via nasal spray device each morning</description>
    <arm_group_label>IN-placebo</arm_group_label>
    <other_name>Saline</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Individuals with genetically confirmed PWS who are in nutritional phase 1a, as&#xD;
             determined by PI&#xD;
&#xD;
          2. Physical exam and laboratory results that are within the normal range.&#xD;
&#xD;
          3. Presence of a parent/caregiver/guardian that is able to consent for their&#xD;
             participation.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Exposure to any investigational agent in the 30 days prior to randomization.&#xD;
&#xD;
          2. Prior chronic treatment with oxytocin.&#xD;
&#xD;
          3. A medical condition that might interfere with the conduct of the study, confound&#xD;
             interpretation of study results or endanger the subject's well-being.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Week</minimum_age>
    <maximum_age>6 Months</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Jennifer Miller, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Florida</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of Florida</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32610</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>March 2020</verification_date>
  <study_first_submitted>August 7, 2017</study_first_submitted>
  <study_first_submitted_qc>August 7, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 10, 2017</study_first_posted>
  <results_first_submitted>January 2, 2019</results_first_submitted>
  <results_first_submitted_qc>June 6, 2019</results_first_submitted_qc>
  <results_first_posted type="Actual">June 25, 2019</results_first_posted>
  <last_update_submitted>March 24, 2020</last_update_submitted>
  <last_update_submitted_qc>March 24, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">March 26, 2020</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>Prader-Willi Syndrome</mesh_term>
    <mesh_term>Syndrome</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Oxytocin</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol and Statistical Analysis Plan</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>August 17, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/62/NCT03245762/Prot_SAP_000.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Intranasal Oxytocin</title>
          <description>Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning.&#xD;
Oxytocin: 4 IU/day of oxytocin administered via nasal spray device each morning.</description>
        </group>
        <group group_id="P2">
          <title>IN-placebo</title>
          <description>Intervention: 4 IU/day of placebo via nasal spray device each morning.&#xD;
Placebo: 4 IU/day of placebo administered via nasal spray device each morning</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="10"/>
                <participants group_id="P2" count="5"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="10"/>
                <participants group_id="P2" count="5"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Intranasal Oxytocin</title>
          <description>Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning.&#xD;
Oxytocin: 4 IU/day of oxytocin administered via nasal spray device each morning.</description>
        </group>
        <group group_id="B2">
          <title>IN-placebo</title>
          <description>Intervention: 4 IU/day of placebo via nasal spray device each morning.&#xD;
Placebo: 4 IU/day of placebo administered via nasal spray device each morning</description>
        </group>
        <group group_id="B3">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="10"/>
            <count group_id="B2" value="5"/>
            <count group_id="B3" value="15"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="10"/>
                    <measurement group_id="B2" value="5"/>
                    <measurement group_id="B3" value="15"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="7"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="9"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="8"/>
                    <measurement group_id="B2" value="5"/>
                    <measurement group_id="B3" value="13"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="7"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="11"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="9"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="13"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Europe</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a</title>
        <description>Swallow study Overall improvement</description>
        <time_frame>baseline to day 5</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Intranasal Oxytocin</title>
            <description>Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning.&#xD;
Oxytocin: 4 IU/day of oxytocin administered via nasal spray device each morning.</description>
          </group>
          <group group_id="O2">
            <title>IN-placebo</title>
            <description>Intervention: 4 IU/day of placebo via nasal spray device each morning.&#xD;
Placebo: 4 IU/day of placebo administered via nasal spray device each morning</description>
          </group>
        </group_list>
        <measure>
          <title>Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a</title>
          <description>Swallow study Overall improvement</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="10"/>
                <count group_id="O2" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Improved Competency</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="8"/>
                    <measurement group_id="O2" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>No Change in Competency'</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Worsened Competency</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.930</p_value>
            <method>Ordinal Categorical Analysis</method>
            <param_type>Odds Ratio (OR)</param_type>
            <param_value>0.9</param_value>
            <ci_n_sides>2-Sided</ci_n_sides>
          </analysis>
        </analysis_list>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>7 days</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Intranasal Oxytocin</title>
          <description>Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning.&#xD;
Oxytocin: 4 IU/day of oxytocin administered via nasal spray device each morning.</description>
        </group>
        <group group_id="E2">
          <title>IN-placebo</title>
          <description>Intervention: 4 IU/day of placebo via nasal spray device each morning.&#xD;
Placebo: 4 IU/day of placebo administered via nasal spray device each morning</description>
        </group>
      </group_list>
      <serious_events>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="10"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="10"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="10"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <limitations_and_caveats>Short study with small number of participants</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Jennifer Miller, MD</name_or_title>
      <organization>University of Florida</organization>
      <phone>352-265-7337</phone>
      <email>millejl@peds.ufl.edu</email>
    </point_of_contact>
  </clinical_results>
</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/NCT03245762
org study id: IRB201700423 -A
secondary id: OCR16237
nct id: NCT03245762
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this study is to compare the change in suck and swallow competency from
      baseline to morning of day 6 with intranasal oxytocin spray vs placebo in infants/children
      with Prader-Willi Syndrome who are in nutritional phase 1a. Videofluoroscopic swallow studies
      will be performed on treatment day 1 and on the day following treatment morning of day 6.
The overall objective of this Phase 2 trial is to compare the change from baseline to morning
      of day 6 of Internasal Oxytocin (IN-OT) on suck and swallow competency in infants/children
      with Prader-Willi Syndrome (PWS) who are in nutritional phase 1a.

      Study Hypothesis 1: The Study team hypothesize that replacing Oxytocin (OT) in infants and
      children who are in nutritional phase 1a will improve their suck and swallow, potentially
      even eliminating the need for gastrostomy tubes and nasogastric tubes for feeding, and
      decreasing the risk of aspiration with oral feeding.

      Study Hypothesis 2: The Study team hypothesize that replacing OT in infants and children with
      PWS will result in improved eye contact, daytime alertness, and feelings of bonding between
      the parents and the infant.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Basic Science
masking: Double (Care Provider, Outcomes Assessor)
masking description: This will be a double-blinded study, as no studies to date have compared this treatment in infants with placebo. Board-certified speech and language pathologist who will be blinded to the treatment arm of each patient.
measure: Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a
time frame: baseline to day 5
description: Swallow study Overall improvement
arm group label: Intranasal oxytocin
arm group type: Active Comparator
description: Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning.
arm group label: IN-placebo
arm group type: Placebo Comparator
description: Intervention: 4 IU/day of placebo via nasal spray device each morning.
intervention type: Drug
intervention name: Oxytocin
description: 4 IU/day of oxytocin administered via nasal spray device each morning.
arm group label: Intranasal oxytocin
other name: Syntocinon
intervention type: Drug
intervention name: Placebo
description: 4 IU/day of placebo administered via nasal spray device each morning
arm group label: IN-placebo
other name: Saline
criteria: 
gender: All
minimum age: 1 Week
maximum age: 6 Months
healthy volunteers: No
last name: Jennifer Miller, MD
role: Principal Investigator
affiliation: University of Florida
facility: 
country: United States
responsible party type: Sponsor
mesh term: Prader-Willi Syndrome
mesh term: Syndrome
mesh term: Oxytocin
sharing ipd: Undecided
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
limitations and caveats: Short study with small number of participants
point of contact: 