<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/NCT01691261</url>
  </required_header>
  <id_info>
    <org_study_id>B4711001</org_study_id>
    <secondary_id>2011-005493-37</secondary_id>
    <nct_id>NCT01691261</nct_id>
  </id_info>
  <brief_title>A Study Of Implantation Of Retinal Pigment Epithelium In Subjects With Acute Wet Age Related Macular Degeneration</brief_title>
  <official_title>Phase 1, Open-label, Safety and Feasibility Study of Implantation of Pf-05206388 (Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (Rpe) Living Tissue Equivalent) in Subjects With Acute Wet Age Related Macular Degeneration and Recent Rapid Vision Decline</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Moorfields Eye Hospital NHS Foundation Trust</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>University College, London</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Moorfields Eye Hospital NHS Foundation Trust</source>
  <oversight_info>
    <has_dmc>Yes</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>
      Phase 1 trial of retinal pigment epithelium replacement in subjects with wet age-related&#xD;
      macular degeneration in whom there is rapidly progressing vision loss&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Phase 1, open-label, safety and feasibility study of implantation of PF-05206388 (human&#xD;
      embryonic stem cell derived retinal pigment epithelium) in subjects with wet age related&#xD;
      macular degeneration and rapid vision loss&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">October 14, 2021</start_date>
  <completion_date type="Anticipated">March 14, 2024</completion_date>
  <primary_completion_date type="Anticipated">October 10, 2023</primary_completion_date>
  <phase>Phase 1</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>Incidence and severity of adverse events.</measure>
    <time_frame>52 weeks</time_frame>
    <description>The number of Adverse Events (AE) and Serious Adverse Events (SAE) noted during the study and an assessment of whether they are trial product related</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more at Week 24.</measure>
    <time_frame>24 weeks</time_frame>
    <description>The number of patients with a difference between the baseline BCVA and BCVA at 24 weeks in ETDRS letters, where the differences is 15 letters or more, as a percentage of the total number of cases.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more</measure>
    <time_frame>Weeks 1,2,4,8, 12,16, 36, 52</time_frame>
    <description>The number of patients with a difference between the baseline BCVA and BCVA at weeks 1,2,4,8, 12,16, 36, 52 in ETDRS letters, where the differences is 15 letters or more, as a percentage of the total number of cases.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Mean change of best corrected visual acuity (BCVA) from baseline by study visit.</measure>
    <time_frame>52 weeks</time_frame>
    <description>The mean difference between the baseline BCVA and final BCVA in ETDRS letters for all cases</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Position of PF-05206388 by serial biomicroscopic evaluation.</measure>
    <time_frame>Day 2 and Weeks 1, 2, 4, 8, 10, 12, 16, 24, 36, 52</time_frame>
    <description>Measurement of movement in millimetre and rotation in degrees measure relative to baseline, at day 2 and weeks 1, 2, 4, 8, 10, 12, 16, 24, 36 and 52</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Position and presence of pigmented RPE cells by serial fundus photography</measure>
    <time_frame>Weeks 2, 4, 8, 10, 12, 16, 24, 36, 52</time_frame>
    <description>The subjective reporting of area of pigmentation as a % with cross reference to the OCT at weeks 2, 4, 8, 10, 12, 16, 24, 36 and 52</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Mean change from baseline in contrast sensitivity by Pelli Robson test</measure>
    <time_frame>Weeks 24, 52</time_frame>
    <description>The mean difference between the baseline BCVA and final BCVA in Pelli Robson letters read, across all subjects</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in liver and renal function by blood tests and liver ultrasound .</measure>
    <time_frame>Weeks 24 and 52</time_frame>
    <description>Record of any abnormalities in liver and renal function on blood testing and any abnormalities detected on the liver ultrasound.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in leakage or perfusion in normal fundal vasculature and presence of abnormal vasculature by fundus fluorescein angiography.</measure>
    <time_frame>Weeks 4, 8, 12, 24 and 52</time_frame>
    <description>Assessment and noting of abnormalities on Funded fluorescine angiography at weeks 4, 8, 12, 24 and 52.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in central 30 degree of visual function by Humphrey Field test.</measure>
    <time_frame>Weeks 4, 8, 12, 24 and 52</time_frame>
    <description>Recording and reporting of any changes on the central 30 degree field on the automated Humphrey Field test at weeks 4, 8, 12, 24 and 52</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in thickness of RPE layer by B-mode orbital ultrasound.</measure>
    <time_frame>Weeks 4, 8, 16, 24, 36, 52</time_frame>
    <description>Recording of any changes in thickness of RPE layer by B-mode orbital ultrasound carried out by the ocular oncologist or medical physicist at weeks 4, 8, 16, 24, 36, 52.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">10</enrollment>
  <condition>Age Related Macular Degeneration</condition>
  <arm_group>
    <arm_group_label>Treatment</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>PF-05206388 Retinal Pigment Epithelium living tissue equivalent for intraocular use in the form of a monolayer of Retinal Pigmented Epithelial (RPE) cells immobilized on a polyester membrane</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>PF-05206388</intervention_name>
    <description>PF-05206388 will be provided as a Retinal Pigment Epithelium living tissue equivalent for intraocular use in the form of a monolayer of Retinal Pigmented Epithelial (RPE) cells immobilized on a polyester membrane. The membrane is approximately 6 mm x 3 mm and will contain a confluent layer of RPE cells, at a nominal dose of 17 mm2. The implant is intended to be life-long.</description>
    <arm_group_label>Treatment</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Male and /or post-menopausal female subjects aged 60 years or above.&#xD;
&#xD;
          -  Diagnosis of wet Age-related Macular Degeneration (AMD) plus rapid recent vision&#xD;
             decline&#xD;
&#xD;
          -  An informed consent document signed and dated by the subject or a legal&#xD;
             representative.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Pregnant females; breastfeeding females; and females of childbearing potential.&#xD;
&#xD;
          -  Treatment with an investigational drug within 30 days (or as determined by the local&#xD;
             requirement, whichever is longer) or 5 half-lives preceding the first dose of study&#xD;
             medication.&#xD;
&#xD;
          -  Current or previous significant other ocular disease in the study eye, as determined&#xD;
             by the investigator.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>60 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Moorfields</last_name>
    <role>Study Director</role>
    <affiliation>Moorfields Eye Hospital NHS Foundation Trust</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Tania West</last_name>
    <phone>02072533411</phone>
    <phone_ext>2036</phone_ext>
    <email>moorfields.resadmin@nhs.net</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Daniela Narvaez</last_name>
    <phone>02072533411</phone>
    <phone_ext>2036</phone_ext>
    <email>moorfields.resadmin@nhs.net</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Moorfields Eye Hospital NHS Foundation Trust</name>
      <address>
        <city>London</city>
        <zip>EC1V 2PD</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
    <status>Recruiting</status>
  </location>
  <location>
    <facility>
      <name>Moorfields Eye Hospital NHS Foundation Trust</name>
      <address>
        <city>London</city>
        <zip>EC1V 2PD</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Moorfields R&amp;D</last_name>
      <phone>020 7253 3411</phone>
      <phone_ext>2937</phone_ext>
      <email>moorfields.resadmin@nhs.net</email>
    </contact>
  </location>
  <location_countries>
    <country>United Kingdom</country>
  </location_countries>
  <link>
    <url>https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=B4711001&amp;StudyName=A%20Study%20Of%20Implantation%20Of%20Human%20Embryonic%20Stem%20Cell%20Derived%20Retinal%20Pigment%20Epithelium%20In%20Subjects%20With%20Acute%20Wet%20Age%20Related%20Macular</url>
    <description>To obtain contact information for a study center near you, click here.</description>
  </link>
  <verification_date>August 2022</verification_date>
  <study_first_submitted>September 19, 2012</study_first_submitted>
  <study_first_submitted_qc>September 19, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 24, 2012</study_first_posted>
  <last_update_submitted>August 18, 2022</last_update_submitted>
  <last_update_submitted_qc>August 18, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">August 22, 2022</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>Macular Degeneration</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
    <ipd_description>Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.</ipd_description>
  </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/NCT01691261
org study id: B4711001
secondary id: 2011-005493-37
nct id: NCT01691261
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
Phase 1 trial of retinal pigment epithelium replacement in subjects with wet age-related
      macular degeneration in whom there is rapidly progressing vision loss
Phase 1, open-label, safety and feasibility study of implantation of PF-05206388 (human
      embryonic stem cell derived retinal pigment epithelium) in subjects with wet age related
      macular degeneration and rapid vision loss
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Incidence and severity of adverse events.
time frame: 52 weeks
description: The number of Adverse Events (AE) and Serious Adverse Events (SAE) noted during the study and an assessment of whether they are trial product related
measure: Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more at Week 24.
time frame: 24 weeks
description: The number of patients with a difference between the baseline BCVA and BCVA at 24 weeks in ETDRS letters, where the differences is 15 letters or more, as a percentage of the total number of cases.
measure: Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more
time frame: Weeks 1,2,4,8, 12,16, 36, 52
description: The number of patients with a difference between the baseline BCVA and BCVA at weeks 1,2,4,8, 12,16, 36, 52 in ETDRS letters, where the differences is 15 letters or more, as a percentage of the total number of cases.
measure: Mean change of best corrected visual acuity (BCVA) from baseline by study visit.
time frame: 52 weeks
description: The mean difference between the baseline BCVA and final BCVA in ETDRS letters for all cases
measure: Position of PF-05206388 by serial biomicroscopic evaluation.
time frame: Day 2 and Weeks 1, 2, 4, 8, 10, 12, 16, 24, 36, 52
description: Measurement of movement in millimetre and rotation in degrees measure relative to baseline, at day 2 and weeks 1, 2, 4, 8, 10, 12, 16, 24, 36 and 52
measure: Position and presence of pigmented RPE cells by serial fundus photography
time frame: Weeks 2, 4, 8, 10, 12, 16, 24, 36, 52
description: The subjective reporting of area of pigmentation as a % with cross reference to the OCT at weeks 2, 4, 8, 10, 12, 16, 24, 36 and 52
measure: Mean change from baseline in contrast sensitivity by Pelli Robson test
time frame: Weeks 24, 52
description: The mean difference between the baseline BCVA and final BCVA in Pelli Robson letters read, across all subjects
measure: Change in liver and renal function by blood tests and liver ultrasound .
time frame: Weeks 24 and 52
description: Record of any abnormalities in liver and renal function on blood testing and any abnormalities detected on the liver ultrasound.
measure: Change in leakage or perfusion in normal fundal vasculature and presence of abnormal vasculature by fundus fluorescein angiography.
time frame: Weeks 4, 8, 12, 24 and 52
description: Assessment and noting of abnormalities on Funded fluorescine angiography at weeks 4, 8, 12, 24 and 52.
measure: Change in central 30 degree of visual function by Humphrey Field test.
time frame: Weeks 4, 8, 12, 24 and 52
description: Recording and reporting of any changes on the central 30 degree field on the automated Humphrey Field test at weeks 4, 8, 12, 24 and 52
measure: Change in thickness of RPE layer by B-mode orbital ultrasound.
time frame: Weeks 4, 8, 16, 24, 36, 52
description: Recording of any changes in thickness of RPE layer by B-mode orbital ultrasound carried out by the ocular oncologist or medical physicist at weeks 4, 8, 16, 24, 36, 52.
arm group label: Treatment
arm group type: Experimental
description: PF-05206388 Retinal Pigment Epithelium living tissue equivalent for intraocular use in the form of a monolayer of Retinal Pigmented Epithelial (RPE) cells immobilized on a polyester membrane
intervention type: Biological
intervention name: PF-05206388
description: PF-05206388 will be provided as a Retinal Pigment Epithelium living tissue equivalent for intraocular use in the form of a monolayer of Retinal Pigmented Epithelial (RPE) cells immobilized on a polyester membrane. The membrane is approximately 6 mm x 3 mm and will contain a confluent layer of RPE cells, at a nominal dose of 17 mm2. The implant is intended to be life-long.
arm group label: Treatment
criteria: 
gender: All
minimum age: 60 Years
maximum age: N/A
healthy volunteers: No
last name: Moorfields
role: Study Director
affiliation: Moorfields Eye Hospital NHS Foundation Trust
last name: Tania West
phone: 02072533411
phone ext: 2036
email: moorfields.resadmin@nhs.net
last name: Daniela Narvaez
phone: 02072533411
phone ext: 2036
email: moorfields.resadmin@nhs.net
facility: 
status: Recruiting
facility: 
status: Recruiting
contact: 
country: United Kingdom
url: https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=B4711001&StudyName=A%20Study%20Of%20Implantation%20Of%20Human%20Embryonic%20Stem%20Cell%20Derived%20Retinal%20Pigment%20Epithelium%20In%20Subjects%20With%20Acute%20Wet%20Age%20Related%20Macular
description: To obtain contact information for a study center near you, click here.
responsible party type: Sponsor
mesh term: Macular Degeneration
sharing ipd: No
ipd description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.