<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/NCT04465188</url>
  </required_header>
  <id_info>
    <org_study_id>P160948J</org_study_id>
    <secondary_id>2018-A01081-54</secondary_id>
    <nct_id>NCT04465188</nct_id>
  </id_info>
  <brief_title>Scleral Buckling for Retinal Detachment Prevention in Genetically Confirmed Stickler Syndrome</brief_title>
  <acronym>STL-DR-PREV</acronym>
  <official_title>Scleral Buckling for Retinal Detachment Prevention in Genetically Confirmed Stickler Syndrome : a Randomized Controlled Trial</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Assistance Publique - Hôpitaux de Paris</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Assistance Publique - Hôpitaux de Paris</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</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 determine whether a scleral buckling surgical procedure&#xD;
      performed on fellow eyes of patients with genetically confirmed Stickler syndrome can prevent&#xD;
      the occurrence retinal detachment and/or severe vision loss of the study eye.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Stickler syndrome (STL) is a genetic disease affecting several organs. However the major risk&#xD;
      is represented by the development of retinal detachment (RD). Up to 80% of patients present a&#xD;
      RD and 25% to 80% have sequential bilateral RD. The surgical outcome of RD in this population&#xD;
      is worse than that of the general RD population mainly because of the severity at diagnosis&#xD;
      and the high frequency and severity of postoperative complications. Prevention has therefore&#xD;
      emerged as a therapeutic option for this identifiable high-risk group of patients. Prevention&#xD;
      has been proposed especially for the fellow (contralateral) eye of patients having presented&#xD;
      a RD in their first eye and often lost vision as a consequence of retinal detachment in the&#xD;
      first eye.&#xD;
&#xD;
      The investigators hypothesize that a scleral buckling surgical procedure performed for the&#xD;
      fellow eye of STL patients having recently presented a RD of the first eye could prevent the&#xD;
      occurrence of bilateral RD and/or vision loss.&#xD;
&#xD;
      Stickler patients are regularly diagnosed and followed-up in referral centers mainly in the&#xD;
      context of a recent retinal detachment. They are proposed genetic testing as part of the&#xD;
      standard of care. Genetically confirmed Stickler patients having recently presented (&lt;24&#xD;
      months) a retinal detachment and treated as part of the standard care are eligible for this&#xD;
      study. During the usual follow-up visits performed for their recent first RD operation they&#xD;
      will be informed and offered to participate in the &quot;STL-DR-PREV study&quot; for the prevention of&#xD;
      RD of their fellow eye.&#xD;
&#xD;
      Patients in the intervention arm will undergo an encircling scleral buckle surgical&#xD;
      procedure, which is a routine surgical procedure used for decades and still in use to treat&#xD;
      RD that will be performed in the present study to prevent rather than to treat retinal&#xD;
      detachment from a healthy eye of a patient having a genetically confirmed Stickler Syndrome.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Not yet recruiting</overall_status>
  <start_date type="Anticipated">June 2023</start_date>
  <completion_date type="Anticipated">January 2030</completion_date>
  <primary_completion_date type="Anticipated">January 2030</primary_completion_date>
  <phase>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>Prevention</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Best corrected visual acuity and retinal detachment rate</measure>
    <time_frame>5 years</time_frame>
    <description>Early Treatment Diabetic Retinopathy Study (ETRDS) best corrected visual acuity (BCVA) and dilated funduscopic examinations at scheduled follow-up (every 6 months)</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Rate of retinal breaks</measure>
    <time_frame>5 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Rate of intravitreal hemorrhage</measure>
    <time_frame>5 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Outcome in prevention-failed study eyes</measure>
    <time_frame>5 years</time_frame>
    <description>outcome of surgery for RD in the study eye of patients for whom prevention failed and had to be operated on for RD repair of the study eye</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Effect of add-on cryotherapy</measure>
    <time_frame>5 years</time_frame>
    <description>To assess within the experimental arm whether patients having had cryotherapy as part (allowed by the protocol at the surgeon's discretion in addition to the standard procedure) of the scleral buckling procedure present a decrease in the rate of RD as compared to buckled patients without cryotherapy</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">40</enrollment>
  <condition>Stickler Syndrome</condition>
  <arm_group>
    <arm_group_label>Experimental arm</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Surgical procedure to prevent retinal detachment in the unaffected eye</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Control arm</arm_group_label>
    <arm_group_type>No Intervention</arm_group_type>
    <description>Standard procedure of clinical practice without any surgical procedure for the unaffected fellow eye.</description>
  </arm_group>
  <intervention>
    <intervention_type>Procedure</intervention_type>
    <intervention_name>Encircling scleral buckle surgical procedure</intervention_name>
    <description>Encircling scleral buckle surgical procedure under general anesthesia</description>
    <arm_group_label>Experimental arm</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Genetically confirmed Stickler Syndrome&#xD;
&#xD;
          -  Aged 5 to 35&#xD;
&#xD;
          -  Recent retinal detachment in the non-study eye (&lt;24 months)&#xD;
&#xD;
          -  Signed informed consent to participate in the study&#xD;
&#xD;
          -  Sufficient patient cooperation to perform a complete ophthalmologic examination&#xD;
             including a dilated fundus examination&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Any type of previous prevention (laser, cryotherapy, scleral buckle) for the study eye&#xD;
&#xD;
          -  Subclinical retinal rhegmatogenous detachment in the study eye.&#xD;
&#xD;
          -  Any other ocular disease unrelated to Stickler syndrome significantly affecting visual&#xD;
             acuity (ETDRS BCVA&lt;20/400).&#xD;
&#xD;
          -  Contraindications to general anesthesia&#xD;
&#xD;
          -  Posterior vitreoretinal traction on optical coherence tomography&#xD;
&#xD;
          -  Intraoperative detection of one or more of the above-mentioned non-inclusion criteria&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>5 Years</minimum_age>
    <maximum_age>35 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Antoine BREZIN, MD,Phd</last_name>
    <role>Study Director</role>
    <affiliation>Assistance Publique - Hôpitaux de Paris</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Pierre-Raphael ROTHSCHILD, MD, Phd</last_name>
    <phone>+33 1 58 41 24 16</phone>
    <email>pierreraphael.rothschild@aphp.fr</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Laetitia PEAUDECERF</last_name>
    <phone>+331 58 41 12 13</phone>
    <email>laetitia.peaudecerf@aphp.fr</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Hôpital Cochin</name>
      <address>
        <city>Paris</city>
        <zip>75014</zip>
        <country>France</country>
      </address>
    </facility>
    <contact>
      <last_name>Pierre-Raphael ROTHSCHILD, MD, Phd</last_name>
      <phone>+331 58 41 24 16</phone>
      <email>pierreraphael.rothschild@aphp.fr</email>
    </contact>
    <contact_backup>
      <last_name>Guillaume MASSON, MS</last_name>
      <phone>+331 58 41 34 78</phone>
      <email>guillaume.masson@aphp.fr</email>
    </contact_backup>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <reference>
    <citation>Monin C, Van Effenterre G, Andre-Sereys P, Haut J. [Prevention of retinal detachment in Wagner-Stickler disease. Comparative study of different methods. Apropos of 22 cases]. J Fr Ophtalmol. 1994;17(3):167-74. French.</citation>
    <PMID>8182253</PMID>
  </reference>
  <reference>
    <citation>Abeysiri P, Bunce C, da Cruz L. Outcomes of surgery for retinal detachment in patients with Stickler syndrome: a comparison of two sequential 20-year cohorts. Graefes Arch Clin Exp Ophthalmol. 2007 Nov;245(11):1633-8. doi: 10.1007/s00417-007-0609-2. Epub 2007 Jun 20.</citation>
    <PMID>17579881</PMID>
  </reference>
  <reference>
    <citation>Carroll C, Papaioannou D, Rees A, Kaltenthaler E. The clinical effectiveness and safety of prophylactic retinal interventions to reduce the risk of retinal detachment and subsequent vision loss in adults and children with Stickler syndrome: a systematic review. Health Technol Assess. 2011 Apr;15(16):iii-xiv, 1-62. doi: 10.3310/hta15160.</citation>
    <PMID>21466760</PMID>
  </reference>
  <reference>
    <citation>Ang A, Poulson AV, Goodburn SF, Richards AJ, Scott JD, Snead MP. Retinal detachment and prophylaxis in type 1 Stickler syndrome. Ophthalmology. 2008 Jan;115(1):164-8. doi: 10.1016/j.ophtha.2007.03.059. Epub 2007 Aug 2.</citation>
    <PMID>17675240</PMID>
  </reference>
  <reference>
    <citation>Fincham GS, Pasea L, Carroll C, McNinch AM, Poulson AV, Richards AJ, Scott JD, Snead MP. Prevention of retinal detachment in Stickler syndrome: the Cambridge prophylactic cryotherapy protocol. Ophthalmology. 2014 Aug;121(8):1588-97. doi: 10.1016/j.ophtha.2014.02.022. Epub 2014 May 1.</citation>
    <PMID>24793526</PMID>
  </reference>
  <verification_date>February 2023</verification_date>
  <study_first_submitted>July 8, 2020</study_first_submitted>
  <study_first_submitted_qc>July 8, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 9, 2020</study_first_posted>
  <last_update_submitted>February 6, 2023</last_update_submitted>
  <last_update_submitted_qc>February 6, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">February 8, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Stickler syndrome</keyword>
  <keyword>Retinal detachment</keyword>
  <keyword>Prophylaxis</keyword>
  <keyword>Collagen</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Craniofacial Abnormalities</mesh_term>
    <mesh_term>Osteochondrodysplasias</mesh_term>
    <mesh_term>Arthritis</mesh_term>
    <mesh_term>Hearing Loss, Sensorineural</mesh_term>
    <mesh_term>Retinal Detachment</mesh_term>
    <mesh_term>Eye Diseases, Hereditary</mesh_term>
    <mesh_term>Connective Tissue Diseases</mesh_term>
    <mesh_term>Syndrome</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/NCT04465188
org study id: P160948J
secondary id: 2018-A01081-54
nct id: NCT04465188
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The purpose of this study is to determine whether a scleral buckling surgical procedure
      performed on fellow eyes of patients with genetically confirmed Stickler syndrome can prevent
      the occurrence retinal detachment and/or severe vision loss of the study eye.
Stickler syndrome (STL) is a genetic disease affecting several organs. However the major risk
      is represented by the development of retinal detachment (RD). Up to 80% of patients present a
      RD and 25% to 80% have sequential bilateral RD. The surgical outcome of RD in this population
      is worse than that of the general RD population mainly because of the severity at diagnosis
      and the high frequency and severity of postoperative complications. Prevention has therefore
      emerged as a therapeutic option for this identifiable high-risk group of patients. Prevention
      has been proposed especially for the fellow (contralateral) eye of patients having presented
      a RD in their first eye and often lost vision as a consequence of retinal detachment in the
      first eye.

      The investigators hypothesize that a scleral buckling surgical procedure performed for the
      fellow eye of STL patients having recently presented a RD of the first eye could prevent the
      occurrence of bilateral RD and/or vision loss.

      Stickler patients are regularly diagnosed and followed-up in referral centers mainly in the
      context of a recent retinal detachment. They are proposed genetic testing as part of the
      standard of care. Genetically confirmed Stickler patients having recently presented (<24
      months) a retinal detachment and treated as part of the standard care are eligible for this
      study. During the usual follow-up visits performed for their recent first RD operation they
      will be informed and offered to participate in the "STL-DR-PREV study" for the prevention of
      RD of their fellow eye.

      Patients in the intervention arm will undergo an encircling scleral buckle surgical
      procedure, which is a routine surgical procedure used for decades and still in use to treat
      RD that will be performed in the present study to prevent rather than to treat retinal
      detachment from a healthy eye of a patient having a genetically confirmed Stickler Syndrome.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Prevention
masking: None (Open Label)
measure: Best corrected visual acuity and retinal detachment rate
time frame: 5 years
description: Early Treatment Diabetic Retinopathy Study (ETRDS) best corrected visual acuity (BCVA) and dilated funduscopic examinations at scheduled follow-up (every 6 months)
measure: Rate of retinal breaks
time frame: 5 years
measure: Rate of intravitreal hemorrhage
time frame: 5 years
measure: Outcome in prevention-failed study eyes
time frame: 5 years
description: outcome of surgery for RD in the study eye of patients for whom prevention failed and had to be operated on for RD repair of the study eye
measure: Effect of add-on cryotherapy
time frame: 5 years
description: To assess within the experimental arm whether patients having had cryotherapy as part (allowed by the protocol at the surgeon's discretion in addition to the standard procedure) of the scleral buckling procedure present a decrease in the rate of RD as compared to buckled patients without cryotherapy
arm group label: Experimental arm
arm group type: Experimental
description: Surgical procedure to prevent retinal detachment in the unaffected eye
arm group label: Control arm
arm group type: No Intervention
description: Standard procedure of clinical practice without any surgical procedure for the unaffected fellow eye.
intervention type: Procedure
intervention name: Encircling scleral buckle surgical procedure
description: Encircling scleral buckle surgical procedure under general anesthesia
arm group label: Experimental arm
criteria: 
gender: All
minimum age: 5 Years
maximum age: 35 Years
healthy volunteers: No
last name: Antoine BREZIN, MD,Phd
role: Study Director
affiliation: Assistance Publique - Hôpitaux de Paris
last name: Pierre-Raphael ROTHSCHILD, MD, Phd
phone: +33 1 58 41 24 16
email: pierreraphael.rothschild@aphp.fr
last name: Laetitia PEAUDECERF
phone: +331 58 41 12 13
email: laetitia.peaudecerf@aphp.fr
facility: 
contact: 
contact backup: 
country: France
citation: Monin C, Van Effenterre G, Andre-Sereys P, Haut J. [Prevention of retinal detachment in Wagner-Stickler disease. Comparative study of different methods. Apropos of 22 cases]. J Fr Ophtalmol. 1994;17(3):167-74. French.
PMID: 8182253
citation: Abeysiri P, Bunce C, da Cruz L. Outcomes of surgery for retinal detachment in patients with Stickler syndrome: a comparison of two sequential 20-year cohorts. Graefes Arch Clin Exp Ophthalmol. 2007 Nov;245(11):1633-8. doi: 10.1007/s00417-007-0609-2. Epub 2007 Jun 20.
PMID: 17579881
citation: Carroll C, Papaioannou D, Rees A, Kaltenthaler E. The clinical effectiveness and safety of prophylactic retinal interventions to reduce the risk of retinal detachment and subsequent vision loss in adults and children with Stickler syndrome: a systematic review. Health Technol Assess. 2011 Apr;15(16):iii-xiv, 1-62. doi: 10.3310/hta15160.
PMID: 21466760
citation: Ang A, Poulson AV, Goodburn SF, Richards AJ, Scott JD, Snead MP. Retinal detachment and prophylaxis in type 1 Stickler syndrome. Ophthalmology. 2008 Jan;115(1):164-8. doi: 10.1016/j.ophtha.2007.03.059. Epub 2007 Aug 2.
PMID: 17675240
citation: Fincham GS, Pasea L, Carroll C, McNinch AM, Poulson AV, Richards AJ, Scott JD, Snead MP. Prevention of retinal detachment in Stickler syndrome: the Cambridge prophylactic cryotherapy protocol. Ophthalmology. 2014 Aug;121(8):1588-97. doi: 10.1016/j.ophtha.2014.02.022. Epub 2014 May 1.
PMID: 24793526
responsible party type: Sponsor
mesh term: Craniofacial Abnormalities
mesh term: Osteochondrodysplasias
mesh term: Arthritis
mesh term: Hearing Loss, Sensorineural
mesh term: Retinal Detachment
mesh term: Eye Diseases, Hereditary
mesh term: Connective Tissue Diseases
mesh term: Syndrome
sharing ipd: No