<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/NCT03305029</url>
  </required_header>
  <id_info>
    <org_study_id>CHA2015-08-141</org_study_id>
    <nct_id>NCT03305029</nct_id>
  </id_info>
  <brief_title>The Safety and Tolerability of Sub-retinal Transplantation of SCNT-hES-RPE Cells in Patients With Advanced Dry AMD</brief_title>
  <official_title>Open-Label, Single-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of SCNT-hES-RPE Cells in Patients With Advanced Dry AMD</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>CHA University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>CHA University</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      To evaluate the safety and tolerability of human somatic cell nuclear transfer embryonic stem&#xD;
      cell derived retinal pigmented epithelial(SCNT-hES-RPE) cellular therapy in patients with&#xD;
      advanced dry AMD&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      To evaluate the safety and tolerability of the SCNT-hES-RPEs, to assess the number of&#xD;
      SCNT-hES-RPE cells to be transplanted in future studies and to evaluate on an exploratory&#xD;
      basis potential efficacy endpoints to be used in future studies of SCNT-hES-RPE cellular&#xD;
      therapy.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Enrolling by invitation</last_known_status>
  <start_date>May 2016</start_date>
  <completion_date type="Anticipated">April 2019</completion_date>
  <primary_completion_date type="Anticipated">April 2019</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>Safety of SCNT-hES-RPE cells</measure>
    <time_frame>60 months</time_frame>
    <description>The transplantation of SCNT-hES-RPE cells will be considered safe in the absence of:&#xD;
Any grade 2 (NCI CTCAE V4.03) or greater adverse event related to the cell product&#xD;
Any evidence that the cells are contaminated with an infectious agent&#xD;
Any evidence that the cells show tumorigenic potential</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>● Change in the mean of BCVA</measure>
    <time_frame>60 months</time_frame>
    <description>Best corrected ETDRS visual acuity scores</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>● Autofluorescence photography</measure>
    <time_frame>60 months</time_frame>
    <description>Autofluorescence photography of GA</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>● Reading speed</measure>
    <time_frame>60 months</time_frame>
    <description>MNReading speed measurements</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>● Structural evidence (OCT imaging, fluorescein angiography, autofluorescense photography, slitlamp examination with fundus phot ography) that cells have been implanted in the correct location</measure>
    <time_frame>60 months</time_frame>
    <description>Evidence of structural changes</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">3</enrollment>
  <condition>Dry Age Related Macular Degeneration</condition>
  <arm_group>
    <arm_group_label>SCNT-hES-RPE Cells</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Pars plana vitrectomy and Sub-retinal Transplantation of Human Somatic cell nuclear transfer Embryonic Stem Cell Derived Retinal Pigmented Epithelial Cells (SCNT-hES-RPE Cells) in Patients with Advanced Dry Age-related Macular Degeneration(AMD)</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>SCNT-hES-RPE Cells</intervention_name>
    <description>Pars Plana Vitrectomy and Sub-retinal Transplantation of Human Somatic cell nuclear transfer Embryonic Stem Cell Derived Retinal Pigmented Epithelial(SCNT-hES-RPE) Cells in Patients with Advanced Dry Age-related Macular Degeneration</description>
    <arm_group_label>SCNT-hES-RPE Cells</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Adult male or female over 50 years of age.&#xD;
&#xD;
          2. Patient should be in sufficiently good health to reasonably expect survival for at&#xD;
             least four years after treatment&#xD;
&#xD;
          3. Clinical findings consistent with advanced dry AMD with evidence of one or more areas&#xD;
             of &gt;250 microns of geographic atrophy (as defined in the Age-Related eye Disease Study&#xD;
             [AREDS] study) involving the central fovea.&#xD;
&#xD;
          4. GA defined as attenuation or loss of RPE as observed by biomicroscopy, OCT, and FA.&#xD;
&#xD;
          5. The visual acuity (BCVA) of the eye to receive the transplant will be no better than&#xD;
             20/200 in Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity.&#xD;
&#xD;
          6. The visual acuity (BCVA) of the eye that is NOT to receive the transplant will be same&#xD;
             or better than the eye to receive the transplant&#xD;
&#xD;
          7. Medically suitable to undergo vitrectomy and subretinal injection.&#xD;
&#xD;
          8. Medically suitable for general anesthesia or waking sedation, if needed.&#xD;
&#xD;
          9. If female and of childbearing potential, willing to medically acceptable methods of&#xD;
             contraception during the study.&#xD;
&#xD;
         10. If male, willing to use barrier and spermicidal contraception during the study.&#xD;
&#xD;
         11. Willing to defer all future blood, blood component or tissue donation.&#xD;
&#xD;
         12. Able to understand and willing to sign the informed consent.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Presence of active or inactive CNV in the eye to be treated.&#xD;
&#xD;
          2. Presence or history of retinal dystrophy, retinitis pigmentosa, chorioretinitis,&#xD;
             central serious choroidopathy, diabetic retinopathy, other retinal vascular or&#xD;
             degenerative disease other than ARMD, optic neuropathy, uveitis, intraocular&#xD;
             inflammatory disease, retinal detachment repair or any other sight-threatening ocular&#xD;
             disease.&#xD;
&#xD;
          3. Presence of glaucomatous optic neuropathy in the study eye, uncontrolled IOP, or use&#xD;
             of two or more agents to control IOP (acetazolamide, beta blocker, alpha-1-agonist,&#xD;
             prostaglandins, anhydrous carbonic inhibitors)&#xD;
&#xD;
          4. Cataract of sufficient severity likely to necessitate surgical extraction within 1&#xD;
             year.&#xD;
&#xD;
          5. Axial myopia of greater than -8 diopters.&#xD;
&#xD;
          6. Axial length greater than 28 mm.&#xD;
&#xD;
          7. Presence of significant lens opacities or other media opacity.&#xD;
&#xD;
          8. History of ocular lens removal within previous 3 months in the study eye.&#xD;
&#xD;
          9. History of ocular surgery in the study eye in the previous 3 months in the study eye.&#xD;
&#xD;
         10. History of malignancy or evidence of malignancy in screening test.&#xD;
&#xD;
         11. Medically not suitable for transplantation of an embryonic stem cell line: Any&#xD;
             laboratory value which falls slightly outside of the normal range will be reviewed by&#xD;
             the Medical Monitor and Investigators to determine its clinical significance. If it is&#xD;
             determined not to be clinically significant, the patient may be enrolled into the&#xD;
             study.&#xD;
&#xD;
               -  History of drug abuse, identified in medical history taking.&#xD;
&#xD;
               -  Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.&#xD;
&#xD;
               -  Any immunodeficiency.&#xD;
&#xD;
               -  Negative cancer screening within previous 12 months: complete history &amp; physical&#xD;
                  examination; negative chest roentgenogram (CXR); negative blood test(including&#xD;
                  CBC &amp; manual differential); negative urinalysis (U/A); normal thyroid exam(T3,&#xD;
                  T4, TSH); if male, negative for prostate specific antigen (PSA); negative for&#xD;
                  upper gastrointestinal series or esophagogastroduodenoscopy; negative for&#xD;
                  α-fetoprotein(AFP); negative fecal occult blood test &amp; negative colonoscopy; if&#xD;
                  female, normal clinical breast exam and, negative mammogram, negative breast&#xD;
                  ultrasonography; if female, normal pelvic examination with Papanicolaou smear;&#xD;
&#xD;
               -  Any of the below criteria of diabetes : Fasting blood glucose≥126mg/dl; random&#xD;
                  plasma glucose with typical symptoms of diabetes≥200mg/dl; HbA1c ≥ 7%&#xD;
&#xD;
         12. History of myocardial infarction or cerebrovascular accident in previous 12 months.&#xD;
&#xD;
         13. History of cognitive impairments or dementia which may impact the patients ability&#xD;
             participate in the informed consent process and to appropriately complete evaluations.&#xD;
&#xD;
         14. Current participation in any other clinical trial.&#xD;
&#xD;
         15. Participation within previous 6 months in any clinical trial of a drug by ocular or&#xD;
             systemic administration.&#xD;
&#xD;
         16. If female, pregnancy or lactation.&#xD;
&#xD;
         17. Any other medical condition, which, in the Investigator's judgment, will interfere&#xD;
             with the patient's ability to comply with the protocol, compromises patient safety, or&#xD;
             interferes with the interpretation of the study results.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>50 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>CHA Bundang Medical Center</name>
      <address>
        <city>Seongnam-si</city>
        <state>Gyeonggi-do</state>
        <zip>463-712</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Korea, Republic of</country>
  </location_countries>
  <verification_date>October 2017</verification_date>
  <study_first_submitted>July 6, 2016</study_first_submitted>
  <study_first_submitted_qc>October 2, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">October 9, 2017</study_first_posted>
  <last_update_submitted>October 2, 2017</last_update_submitted>
  <last_update_submitted_qc>October 2, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">October 9, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>CHA University</investigator_affiliation>
    <investigator_full_name>Won Kyung, Song</investigator_full_name>
    <investigator_title>Principal Investigator</investigator_title>
  </responsible_party>
  <keyword>Age related macular degeneration</keyword>
  <keyword>embryonic stem cells</keyword>
  <keyword>somatic cell nuclear transfer</keyword>
  <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>
  </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/NCT03305029
org study id: CHA2015-08-141
nct id: NCT03305029
lead sponsor: 
has dmc: No
To evaluate the safety and tolerability of human somatic cell nuclear transfer embryonic stem
      cell derived retinal pigmented epithelial(SCNT-hES-RPE) cellular therapy in patients with
      advanced dry AMD
To evaluate the safety and tolerability of the SCNT-hES-RPEs, to assess the number of
      SCNT-hES-RPE cells to be transplanted in future studies and to evaluate on an exploratory
      basis potential efficacy endpoints to be used in future studies of SCNT-hES-RPE cellular
      therapy.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Safety of SCNT-hES-RPE cells
time frame: 60 months
description: The transplantation of SCNT-hES-RPE cells will be considered safe in the absence of:
Any grade 2 (NCI CTCAE V4.03) or greater adverse event related to the cell product
Any evidence that the cells are contaminated with an infectious agent
Any evidence that the cells show tumorigenic potential
measure: ● Change in the mean of BCVA
time frame: 60 months
description: Best corrected ETDRS visual acuity scores
measure: ● Autofluorescence photography
time frame: 60 months
description: Autofluorescence photography of GA
measure: ● Reading speed
time frame: 60 months
description: MNReading speed measurements
measure: ● Structural evidence (OCT imaging, fluorescein angiography, autofluorescense photography, slitlamp examination with fundus phot ography) that cells have been implanted in the correct location
time frame: 60 months
description: Evidence of structural changes
arm group label: SCNT-hES-RPE Cells
arm group type: Experimental
description: Pars plana vitrectomy and Sub-retinal Transplantation of Human Somatic cell nuclear transfer Embryonic Stem Cell Derived Retinal Pigmented Epithelial Cells (SCNT-hES-RPE Cells) in Patients with Advanced Dry Age-related Macular Degeneration(AMD)
intervention type: Drug
intervention name: SCNT-hES-RPE Cells
description: Pars Plana Vitrectomy and Sub-retinal Transplantation of Human Somatic cell nuclear transfer Embryonic Stem Cell Derived Retinal Pigmented Epithelial(SCNT-hES-RPE) Cells in Patients with Advanced Dry Age-related Macular Degeneration
arm group label: SCNT-hES-RPE Cells
criteria: 
gender: All
minimum age: 50 Years
maximum age: N/A
healthy volunteers: No
facility: 
country: Korea, Republic of
responsible party type: Principal Investigator
investigator affiliation: CHA University
investigator full name: Won Kyung, Song
investigator title: Principal Investigator
mesh term: Macular Degeneration
sharing ipd: No