<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/NCT04419363</url>
  </required_header>
  <id_info>
    <org_study_id>20200528104746</org_study_id>
    <nct_id>NCT04419363</nct_id>
  </id_info>
  <brief_title>Burosumab in Children and Adolescents With X-linked Hypophosphatemia</brief_title>
  <official_title>12-months of Treatment With Burosumab in Children and Adolescents With X-linked Hypophosphatemia: a Prospective Longitudinal Cohort Study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Bicetre Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Bicetre Hospital</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      In this prospective longitudinal cohort study we studied the efficacy and safety of burosumab&#xD;
      in real-clinical practice for &lt;13- and &gt;13-years old children affected with X-linked&#xD;
      hypophosphatemia.&#xD;
&#xD;
      57 children with XLH were switched from conventional treatment to burosumab. After 12 months&#xD;
      we assessed the efficacy and safety of treatment with burosumab on the whole cohort and&#xD;
      separately on the cohort of &gt;13-years old adolescents.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date type="Actual">March 18, 2018</start_date>
  <completion_date type="Anticipated">September 16, 2022</completion_date>
  <primary_completion_date type="Actual">March 20, 2019</primary_completion_date>
  <phase>Phase 4</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>Radiological changes in rachitic lesions evaluated with knee MRI</measure>
    <time_frame>12 months</time_frame>
    <description>maximum width of the physis and transverse extent of widening</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>serum phosphate</measure>
    <time_frame>12 months</time_frame>
    <description>mmol/l</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>renal phosphate reabsorption</measure>
    <time_frame>12 months</time_frame>
    <description>mmol/l</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>alkaline phosphatase</measure>
    <time_frame>12 months</time_frame>
    <description>U/l</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>1,25(OH)vitaminD</measure>
    <time_frame>12 months</time_frame>
    <description>pg/ml</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>parathyroid hormone</measure>
    <time_frame>12 months</time_frame>
    <description>ng/l</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>height</measure>
    <time_frame>12 months</time_frame>
    <description>standard deviation score</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>functional capacity</measure>
    <time_frame>12 months</time_frame>
    <description>6-minute walk test, standard deviation score</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>incidence of dental abcesses</measure>
    <time_frame>12 months</time_frame>
    <description>dental examination</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>incidence of hearing problems</measure>
    <time_frame>12 months</time_frame>
    <description>ORL examination, audiogramm</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>incidence of neurological problems (craniosynostosis, Chiari I malformation)</measure>
    <time_frame>12 months</time_frame>
    <description>neurosurgical examination and brain MRI</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>incidence of nephrocalcinosis</measure>
    <time_frame>12 months</time_frame>
    <description>renal ultrasound</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>incidence of hyperparathyroidism</measure>
    <time_frame>12 months</time_frame>
    <description>blood levels of parathyroid hormone</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>incidence of any side effects</measure>
    <time_frame>12 months</time_frame>
    <description>registration of any side effects during the treatment by telephone call</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">57</enrollment>
  <condition>Rare Diseases</condition>
  <condition>X-linked Hypophosphatemia</condition>
  <arm_group>
    <arm_group_label>The whole cohort</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Children affected with X-linked hypophosphatemia of average age of 9.8 years were switch from conventional therapy to burosumab</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Burosumab Injection</intervention_name>
    <description>Children affected with X-linked hypophosphatemia were switched from conventional therapy to burosumab</description>
    <arm_group_label>The whole cohort</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  insufficient response or refractory to conventional therapy;&#xD;
&#xD;
          -  complications of conventional therapy: hypercalciuria and/or nephrocalcinosis, and/or&#xD;
             persistent secondary hyperparathyroidism;&#xD;
&#xD;
          -  need for rapid restoration of phosphate metabolism, e.g., late diagnosis (aged &gt;8&#xD;
             years) and/or preparation for planned orthopaedic surgery.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Year</minimum_age>
    <maximum_age>20 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Hospital Bicetre</name>
      <address>
        <city>Le Kremlin-Bicêtre</city>
        <zip>94270</zip>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Volha Zhukouskaya, MD, PhD</last_name>
      <phone>+33766681018</phone>
      <email>vzhukuskaya@genethon.fr</email>
    </contact>
    <investigator>
      <last_name>Agnès Linglart, MD, PhD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>June 2020</verification_date>
  <study_first_submitted>May 29, 2020</study_first_submitted>
  <study_first_submitted_qc>June 3, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 5, 2020</study_first_posted>
  <last_update_submitted>June 3, 2020</last_update_submitted>
  <last_update_submitted_qc>June 3, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">June 5, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Bicetre Hospital</investigator_affiliation>
    <investigator_full_name>Volha Zhukouskaya</investigator_full_name>
    <investigator_title>MD, PhD</investigator_title>
  </responsible_party>
  <keyword>X-linked hypophosphataemia</keyword>
  <keyword>fibroblast growth factor 23</keyword>
  <keyword>burosumab</keyword>
  <keyword>hyperparathyroidism</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Familial Hypophosphatemic Rickets</mesh_term>
    <mesh_term>Hypophosphatemia</mesh_term>
    <mesh_term>Rare Diseases</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/NCT04419363
org study id: 20200528104746
nct id: NCT04419363
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
In this prospective longitudinal cohort study we studied the efficacy and safety of burosumab
      in real-clinical practice for <13- and >13-years old children affected with X-linked
      hypophosphatemia.

      57 children with XLH were switched from conventional treatment to burosumab. After 12 months
      we assessed the efficacy and safety of treatment with burosumab on the whole cohort and
      separately on the cohort of >13-years old adolescents.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Radiological changes in rachitic lesions evaluated with knee MRI
time frame: 12 months
description: maximum width of the physis and transverse extent of widening
measure: serum phosphate
time frame: 12 months
description: mmol/l
measure: renal phosphate reabsorption
time frame: 12 months
description: mmol/l
measure: alkaline phosphatase
time frame: 12 months
description: U/l
measure: 1,25(OH)vitaminD
time frame: 12 months
description: pg/ml
measure: parathyroid hormone
time frame: 12 months
description: ng/l
measure: height
time frame: 12 months
description: standard deviation score
measure: functional capacity
time frame: 12 months
description: 6-minute walk test, standard deviation score
measure: incidence of dental abcesses
time frame: 12 months
description: dental examination
measure: incidence of hearing problems
time frame: 12 months
description: ORL examination, audiogramm
measure: incidence of neurological problems (craniosynostosis, Chiari I malformation)
time frame: 12 months
description: neurosurgical examination and brain MRI
measure: incidence of nephrocalcinosis
time frame: 12 months
description: renal ultrasound
measure: incidence of hyperparathyroidism
time frame: 12 months
description: blood levels of parathyroid hormone
measure: incidence of any side effects
time frame: 12 months
description: registration of any side effects during the treatment by telephone call
arm group label: The whole cohort
arm group type: Experimental
description: Children affected with X-linked hypophosphatemia of average age of 9.8 years were switch from conventional therapy to burosumab
intervention type: Drug
intervention name: Burosumab Injection
description: Children affected with X-linked hypophosphatemia were switched from conventional therapy to burosumab
arm group label: The whole cohort
criteria: 
gender: All
minimum age: 1 Year
maximum age: 20 Years
healthy volunteers: No
facility: 
status: Recruiting
contact: 
investigator: 
country: France
responsible party type: Principal Investigator
investigator affiliation: Bicetre Hospital
investigator full name: Volha Zhukouskaya
investigator title: MD, PhD
mesh term: Familial Hypophosphatemic Rickets
mesh term: Hypophosphatemia
mesh term: Rare Diseases
sharing ipd: No