<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/NCT04408300</url>
  </required_header>
  <id_info>
    <org_study_id>APS_2020_5</org_study_id>
    <nct_id>NCT04408300</nct_id>
  </id_info>
  <brief_title>Study of Retinal Vascular Parameters in Patients With Wilson's Disease</brief_title>
  <acronym>WILLY</acronym>
  <official_title>Study of Retinal Vascular Parameters in Patients With Wilson's Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Fondation Ophtalmologique Adolphe de Rothschild</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Fondation Ophtalmologique Adolphe de Rothschild</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>
      The rare disease reference center &quot; Wilson disease and other rare copper-related diseases&quot; of&#xD;
      the Rothschild Foundation follows a large number of patients with Wilson's with varying&#xD;
      degrees of impairment and located at different times of their care.&#xD;
&#xD;
      Many people with Wilson's disease have a characteristic greenish-brown ring, known as&#xD;
      Kayser-Fleischer, appearing at the periphery of the cornea due to a deposit of copper at the&#xD;
      Descemet membrane. As a general rule, if the patient is compliant with his treatment, the&#xD;
      ring usually disappears within a few years, although it may persist in some patients.&#xD;
      However, apart from the stage of diagnosis, and the evolution of the ring, ophthalmological&#xD;
      examinations are little used for the follow-up of these patients.&#xD;
&#xD;
      The objective of this study is to describe the retinal parameters, in particular vascular&#xD;
      with two new retinal imaging technologies (OCT-A :Optical Coherence Tomography-Angiography ,&#xD;
      Adaptive optics) in patients with Wilson's disease and to correlate them with the parameters&#xD;
      of the usual follow-up of these patients (hepatic assessment, exchangeable plasma copper,&#xD;
      neurological scores, compliance, etc.).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">June 4, 2020</start_date>
  <completion_date type="Actual">February 15, 2022</completion_date>
  <primary_completion_date type="Actual">January 6, 2022</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>Other</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Measuring the width of the Kayser-Fleischer ring</measure>
    <time_frame>Enrollment</time_frame>
    <description>OCT-B scan</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Corrected visual acuity measurement</measure>
    <time_frame>Enrollment</time_frame>
    <description>ETDRS</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Intraocular pressure measurement (mmHg)</measure>
    <time_frame>Enrollment</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Measurement of the thickness of the retinal nerve fibers layer (RNFL)</measure>
    <time_frame>Enrollment</time_frame>
    <description>OCT-B</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Measurement of the central avascular zone (in µm2)</measure>
    <time_frame>Inclusion</time_frame>
    <description>OCT-A</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Measurement of the capillary density of the superficial plexus (in%)</measure>
    <time_frame>Enrollment</time_frame>
    <description>OCT-A</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Measurement of the capillary density of the deep plexus (in%)</measure>
    <time_frame>Enrollment</time_frame>
    <description>OCT-A</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Measurement of photoreceptor density (per mm2)</measure>
    <time_frame>Enrollment</time_frame>
    <description>Adaptive optics</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Measurement of wall to lumen ratio (in %)</measure>
    <time_frame>Enrollment</time_frame>
    <description>Adaptive optics</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Measurement of the arterial wall section area (µm²)</measure>
    <time_frame>Enrollment</time_frame>
    <description>Adaptive optics</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Measurement of the diameter of the arterial lumen (µm)</measure>
    <time_frame>Enrollment</time_frame>
    <description>Adaptive optics</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">124</enrollment>
  <condition>Wilson Disease</condition>
  <arm_group>
    <arm_group_label>Ophthalmological exam</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Procedure</intervention_type>
    <intervention_name>Ophthalmological exam</intervention_name>
    <description>Retinophotography, OCT-A (Optical Coherence Tomography- Angiography), OCT-B scan (Optical Coherence Tomography B Scan), Measurement of corrected visual acuity in ETDRS (Early Treatment Diabetic Retinopathy Study), Measurement of intraocular pressure, adaptive optics</description>
    <arm_group_label>Ophthalmological exam</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Wilson's disease with Ferenci score ≥4&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Neurological or behavioral handicap that does not allow for ophthalmological&#xD;
             examinations&#xD;
&#xD;
          -  Hepatic decompensation that does not allow for ophthalmological examinations&#xD;
&#xD;
          -  Pregnant or breastfeeding woman&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Aurélia POUJOIS, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Fondation A. de Rothschild</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Hôpital Fondation A. de Rothschild</name>
      <address>
        <city>Paris</city>
        <zip>75019</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>March 2022</verification_date>
  <study_first_submitted>May 26, 2020</study_first_submitted>
  <study_first_submitted_qc>May 26, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">May 29, 2020</study_first_posted>
  <last_update_submitted>March 2, 2022</last_update_submitted>
  <last_update_submitted_qc>March 2, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">March 3, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Optical Coherence Tomography</keyword>
  <keyword>Adaptive optics</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hepatolenticular Degeneration</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/NCT04408300
org study id: APS_2020_5
nct id: NCT04408300
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
The rare disease reference center " Wilson disease and other rare copper-related diseases" of
      the Rothschild Foundation follows a large number of patients with Wilson's with varying
      degrees of impairment and located at different times of their care.

      Many people with Wilson's disease have a characteristic greenish-brown ring, known as
      Kayser-Fleischer, appearing at the periphery of the cornea due to a deposit of copper at the
      Descemet membrane. As a general rule, if the patient is compliant with his treatment, the
      ring usually disappears within a few years, although it may persist in some patients.
      However, apart from the stage of diagnosis, and the evolution of the ring, ophthalmological
      examinations are little used for the follow-up of these patients.

      The objective of this study is to describe the retinal parameters, in particular vascular
      with two new retinal imaging technologies (OCT-A :Optical Coherence Tomography-Angiography ,
      Adaptive optics) in patients with Wilson's disease and to correlate them with the parameters
      of the usual follow-up of these patients (hepatic assessment, exchangeable plasma copper,
      neurological scores, compliance, etc.).
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Other
masking: None (Open Label)
measure: Measuring the width of the Kayser-Fleischer ring
time frame: Enrollment
description: OCT-B scan
measure: Corrected visual acuity measurement
time frame: Enrollment
description: ETDRS
measure: Intraocular pressure measurement (mmHg)
time frame: Enrollment
measure: Measurement of the thickness of the retinal nerve fibers layer (RNFL)
time frame: Enrollment
description: OCT-B
measure: Measurement of the central avascular zone (in µm2)
time frame: Inclusion
description: OCT-A
measure: Measurement of the capillary density of the superficial plexus (in%)
time frame: Enrollment
description: OCT-A
measure: Measurement of the capillary density of the deep plexus (in%)
time frame: Enrollment
description: OCT-A
measure: Measurement of photoreceptor density (per mm2)
time frame: Enrollment
description: Adaptive optics
measure: Measurement of wall to lumen ratio (in %)
time frame: Enrollment
description: Adaptive optics
measure: Measurement of the arterial wall section area (µm²)
time frame: Enrollment
description: Adaptive optics
measure: Measurement of the diameter of the arterial lumen (µm)
time frame: Enrollment
description: Adaptive optics
arm group label: Ophthalmological exam
arm group type: Experimental
intervention type: Procedure
intervention name: Ophthalmological exam
description: Retinophotography, OCT-A (Optical Coherence Tomography- Angiography), OCT-B scan (Optical Coherence Tomography B Scan), Measurement of corrected visual acuity in ETDRS (Early Treatment Diabetic Retinopathy Study), Measurement of intraocular pressure, adaptive optics
arm group label: Ophthalmological exam
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Aurélia POUJOIS, MD
role: Principal Investigator
affiliation: Fondation A. de Rothschild
facility: 
country: France
responsible party type: Sponsor
mesh term: Hepatolenticular Degeneration