<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/NCT02205450</url>
  </required_header>
  <id_info>
    <org_study_id>CPT_ENDOPED201401</org_study_id>
    <nct_id>NCT02205450</nct_id>
  </id_info>
  <brief_title>Growth Hormone in Children Under 2 Years With Prader-Willi in Hospital of Sabadell</brief_title>
  <official_title>Experience With Growth Hormone (GH) in Children Under 2 Years With Prader-Willi Syndrome (PWS) in the Pediatric Endocrine Department of the Hospital of Sabadell</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Corporacion Parc Tauli</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Corporacion Parc Tauli</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The PWS is a genetic disease with intellectual disabilities associated with multiple&#xD;
      manifestations in other body systems. It is characterized by hypothalamic-pituitary&#xD;
      abnormalities with severe hypotonia during the early years of life, conditioning feeding&#xD;
      difficulties. Hyperphagia appears later, causing severe obesity in pre - school ages. Other&#xD;
      endocrine abnormalities associated produce short stature, GH deficiency and hypogonadotropic&#xD;
      hypogonadism. These patients also have varying cognitive dysfunction associated as well as&#xD;
      learning problems, compounded by the development of psychological-psychiatric and behavioral&#xD;
      problems language. The aetiology of GH decreased secretion of the SPW is controversial, it is&#xD;
      known that IGF -1 levels are reduced in children and adults with PWS. The rational use of GH&#xD;
      is derived from knowledge of comorbidities observed in PWS, which seem to be related to GH&#xD;
      deficiency: hypotonia, altered body composition, decreased growth, even obesity.&#xD;
&#xD;
      • The GH is accepted since 2000 for the treatment of PWS. Following fatal episodes in our&#xD;
      country, it was decided to start treatment at 2 years of age in an arbitrary manner, but not&#xD;
      in the U.S. or France. Subsequent studies have found that GH per se is not a risk factor for&#xD;
      mortality. The currently published data supporting the benefits of GH treatment when started&#xD;
      between 4 and 6 months of life, even some experts advocate starting at 3 months, but due to&#xD;
      the lack of consensus on the age of onset treatment, despite the benefits of your home at an&#xD;
      early age before the onset of obesity often starts around 2 years of life.&#xD;
&#xD;
      HYPOTHESIS The use of GH is safe and effective in patients with PWS children under 2 years&#xD;
      old.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>September 2014</start_date>
  <completion_date type="Actual">July 29, 2019</completion_date>
  <primary_completion_date type="Actual">July 29, 2019</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>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>To assess the safe use of GH in children under 2 year old with Prader Willi Syndrome</measure>
    <time_frame>Two years</time_frame>
    <description>Collect any Serious Adverse Event during the length of study</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Evaluate the impact of treatment with GH in kids under 2 years old on body composition</measure>
    <time_frame>Every 3 months during 2 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluate the impact of treatment with GH in kids under 2 years old on start walking</measure>
    <time_frame>Every 3 months during 2 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluate the impact of treatment with GH in kids under 2 years old on the speech beginning</measure>
    <time_frame>Every 3 months during 2 years</time_frame>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">16</enrollment>
  <condition>Prader-Willi Syndrome</condition>
  <arm_group>
    <arm_group_label>Children under 2 years with Prader-Willi Syndrome</arm_group_label>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Recombinant Somatropin</intervention_name>
    <arm_group_label>Children under 2 years with Prader-Willi Syndrome</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Hospitalary population&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Children under 2 years&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        -&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>3 Months</minimum_age>
    <maximum_age>2 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Raquel Corripio, PI</last_name>
    <role>Principal Investigator</role>
    <affiliation>Corporacio PT</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Corporació Sanitària Parc Taulí</name>
      <address>
        <city>Sabadell</city>
        <state>Barcelona</state>
        <zip>08208</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Spain</country>
  </location_countries>
  <link>
    <url>https://doi.org/10.1515/jpem-2018-0539</url>
    <description>Safety and effectiveness of growth hormone therapy in infants with Prader-Willi syndrome younger than 2 years: a prospective study Raquel Corripio ORCID logo, Carla Tubau, Laura Calvo, Carme Brun, Núria Capdevila, Helena Larramona and Elisabeth Gabau</description>
  </link>
  <verification_date>March 2022</verification_date>
  <study_first_submitted>July 30, 2014</study_first_submitted>
  <study_first_submitted_qc>July 30, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">July 31, 2014</study_first_posted>
  <last_update_submitted>March 15, 2022</last_update_submitted>
  <last_update_submitted_qc>March 15, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">March 31, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Corporacion Parc Tauli</investigator_affiliation>
    <investigator_full_name>Raquel Corripio-Collado</investigator_full_name>
    <investigator_title>MD PhD</investigator_title>
  </responsible_party>
  <keyword>Prader-Willi Syndrome</keyword>
  <keyword>Growth Hormone</keyword>
  <keyword>Children</keyword>
  <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>
  <!-- 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/NCT02205450
org study id: CPT_ENDOPED201401
nct id: NCT02205450
lead sponsor: 
has dmc: No
The PWS is a genetic disease with intellectual disabilities associated with multiple
      manifestations in other body systems. It is characterized by hypothalamic-pituitary
      abnormalities with severe hypotonia during the early years of life, conditioning feeding
      difficulties. Hyperphagia appears later, causing severe obesity in pre - school ages. Other
      endocrine abnormalities associated produce short stature, GH deficiency and hypogonadotropic
      hypogonadism. These patients also have varying cognitive dysfunction associated as well as
      learning problems, compounded by the development of psychological-psychiatric and behavioral
      problems language. The aetiology of GH decreased secretion of the SPW is controversial, it is
      known that IGF -1 levels are reduced in children and adults with PWS. The rational use of GH
      is derived from knowledge of comorbidities observed in PWS, which seem to be related to GH
      deficiency: hypotonia, altered body composition, decreased growth, even obesity.

      • The GH is accepted since 2000 for the treatment of PWS. Following fatal episodes in our
      country, it was decided to start treatment at 2 years of age in an arbitrary manner, but not
      in the U.S. or France. Subsequent studies have found that GH per se is not a risk factor for
      mortality. The currently published data supporting the benefits of GH treatment when started
      between 4 and 6 months of life, even some experts advocate starting at 3 months, but due to
      the lack of consensus on the age of onset treatment, despite the benefits of your home at an
      early age before the onset of obesity often starts around 2 years of life.

      HYPOTHESIS The use of GH is safe and effective in patients with PWS children under 2 years
      old.
observational model: Cohort
time perspective: Prospective
measure: To assess the safe use of GH in children under 2 year old with Prader Willi Syndrome
time frame: Two years
description: Collect any Serious Adverse Event during the length of study
measure: Evaluate the impact of treatment with GH in kids under 2 years old on body composition
time frame: Every 3 months during 2 years
measure: Evaluate the impact of treatment with GH in kids under 2 years old on start walking
time frame: Every 3 months during 2 years
measure: Evaluate the impact of treatment with GH in kids under 2 years old on the speech beginning
time frame: Every 3 months during 2 years
arm group label: Children under 2 years with Prader-Willi Syndrome
intervention type: Drug
intervention name: Recombinant Somatropin
arm group label: Children under 2 years with Prader-Willi Syndrome
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 3 Months
maximum age: 2 Years
healthy volunteers: No
last name: Raquel Corripio, PI
role: Principal Investigator
affiliation: Corporacio PT
facility: 
country: Spain
url: https://doi.org/10.1515/jpem-2018-0539
description: Safety and effectiveness of growth hormone therapy in infants with Prader-Willi syndrome younger than 2 years: a prospective study Raquel Corripio ORCID logo, Carla Tubau, Laura Calvo, Carme Brun, Núria Capdevila, Helena Larramona and Elisabeth Gabau
responsible party type: Principal Investigator
investigator affiliation: Corporacion Parc Tauli
investigator full name: Raquel Corripio-Collado
investigator title: MD PhD
mesh term: Prader-Willi Syndrome
mesh term: Syndrome