<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/NCT01674829</url>
  </required_header>
  <id_info>
    <org_study_id>CHA CTP 1101</org_study_id>
    <nct_id>NCT01674829</nct_id>
  </id_info>
  <brief_title>A Phase I/IIa, Open-Label, Single-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial(MA09-hRPE) Cells in Patients With Advanced Dry Age-related Macular Degeneration(AMD)</brief_title>
  <official_title>A Phase I/IIa, Open-Label, Single-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial(MA09-hRPE) Cells in Patients With Advanced Dry Age-related Macular Degeneration(AMD)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>CHABiotech CO., Ltd</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>CHABiotech CO., Ltd</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      To evaluate the safety and tolerability of MA09-hRPE cellular therapy in patients with&#xD;
      advanced dry AMD To evaluate the safety of the surgical procedures when used to implant&#xD;
      MA09-hRPE cells To assess the number of hRPE cells to be transplanted in future studies To&#xD;
      evaluate on an exploratory basis potential efficacy endpoints to be used in future studies of&#xD;
      MA09-hRPE cellular therapy.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Active, not recruiting</last_known_status>
  <start_date>September 2012</start_date>
  <completion_date type="Anticipated">June 2020</completion_date>
  <primary_completion_date type="Anticipated">June 2020</primary_completion_date>
  <phase>Phase 1/Phase 2</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 hESC derived RPE cells</measure>
    <time_frame>12 months</time_frame>
    <description>The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe in the absence of:&#xD;
Any grade 2 (NCI grading system) 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>exploratory evaluations for potential efficacy endpoints</measure>
    <time_frame>12months</time_frame>
    <description>Secondary endpoints will be evaluated as exploratory evaluations for potential efficacy endpoints.&#xD;
Change in the mean of BCVA&#xD;
Autofluorescense photography&#xD;
Reading speed&#xD;
Evidence of successful engraftment will consist of:&#xD;
Structural evidence (OCT imaging, fluorescein angiography, slitlamp examination with fundus photography) that cells have been implanted in the correct location&#xD;
Electroretinographic evidence (mfERG) showing enhanced activity in the implant location</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">12</enrollment>
  <condition>Dry Age Related Macular Degeneration</condition>
  <arm_group>
    <arm_group_label>Biological: MA09-hRPE Cellular therapy</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Biological: MA09-hRPE Cellular therapy&#xD;
Cohort 1 50,000 cells&#xD;
Cohort 2 100,000 cells&#xD;
Cohort 3 150,000 cells&#xD;
Cohort 4 200,000 cells</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>MA09-hRPE</intervention_name>
    <arm_group_label>Biological: MA09-hRPE Cellular therapy</arm_group_label>
    <other_name>Cohort 1- 50,000 MA09-hRPE cells transplanted</other_name>
    <other_name>Cohort 2- 100,000 MA09-hRPE cells transplanted</other_name>
    <other_name>Cohort 3- 150,000 MA09-hRPE cells transplanted</other_name>
    <other_name>Cohort 4- 200,000 MA09-hRPE cells transplanted</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Adult male or female over 55 years of age. Patient should be in sufficiently good&#xD;
             health to reasonably expect survival for at least four years after treatment&#xD;
&#xD;
          -  Clinical findings consistent with advanced dry AMD with evidence of one or more areas&#xD;
             of &gt;250microns of geographic atrophy (as defined in the Age-Related eye Disease Study&#xD;
             [AREDS] study) involving the central fovea.&#xD;
&#xD;
          -  GA defined as attenuation or loss of RPE as observed by biomicroscopy, OCT, and FA.&#xD;
&#xD;
          -  No evidence of current or prior choroidal neovascularization in the treated eye&#xD;
&#xD;
          -  The visual acuity (BCVA) of the eye to receive the transplant will be no better than&#xD;
             20/400.&#xD;
&#xD;
          -  The visual acuity (BCVA) of the eye that is NOT to receive the transplant will be no&#xD;
             worse than 20/400.&#xD;
&#xD;
          -  Electrophysiological findings consistent with advanced dry AMD.&#xD;
&#xD;
          -  Medically suitable to undergo vitrectomy and subretinal injection.&#xD;
&#xD;
          -  Medically suitable for general anesthesia or waking sedation, if needed.&#xD;
&#xD;
          -  Medically suitable for transplantation of an embryonic stem cell line:&#xD;
&#xD;
        Any 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 study.&#xD;
&#xD;
        Normal serum chemistry complete blood count [CBC], prothrombin time [PT], and activated&#xD;
        partial thromboplastin time [aPTT] Negative urine screen for drugs of abuse. Negative human&#xD;
        immunodeficiency virus (HIV), hepatitis B (HBV), hepatitis C (HCV) serologies.&#xD;
&#xD;
        No history of malignancy&#xD;
&#xD;
        Negative cancer screening within previous 6 months:&#xD;
&#xD;
        complete history &amp; physical examination; dermatological screening exam for malignant&#xD;
        lesions; negative fecal occult blood test &amp; negative colonoscopy within previous 7 years;&#xD;
        negative chest roentgenogram (CXR); normal CBC &amp; manual differential; negative urinalysis&#xD;
        (U/A); normal thyroid exam; if male, normal testicular examination; digital rectal&#xD;
        examination (DRE) and prostate specific antigen (PSA); if female, normal pelvic examination&#xD;
        with Papanicolaou smear; and If female, normal clinical breast exam and, negative&#xD;
        mammogram. If female and of childbearing potential, willing to use two effective forms of&#xD;
        birth control during the study.&#xD;
&#xD;
        If male, willing to use barrier and spermicidal contraception during the study. Willing to&#xD;
        defer all future blood, blood component or tissue donation. Able to understand and willing&#xD;
        to sign the informed consent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Presence of active or inactive CNV in the eye to be treated.&#xD;
&#xD;
          -  Presence or history of retinal dystrophy, retinitis pigmentosa, chorioretinitis,&#xD;
             central serious choroidopathy, diabetic retinopathy or other retinal vascular or&#xD;
             degenerative disease other than ARMD.&#xD;
&#xD;
          -  History of optic neuropathy.&#xD;
&#xD;
          -  Macular atrophy due to causes other than AMD.&#xD;
&#xD;
          -  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;
          -  Cataract of sufficient severity likely to necessitate surgical extraction within 1&#xD;
             year.&#xD;
&#xD;
          -  History of retinal detachment repair in the study eye.&#xD;
&#xD;
          -  Axial myopia of greater than -8 diopters&#xD;
&#xD;
          -  Axial length greater than 28 mm.&#xD;
&#xD;
          -  History of myocardial infarction in previous 12 months.&#xD;
&#xD;
          -  History of diabetes mellitus.&#xD;
&#xD;
          -  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;
          -  Any immunodeficiency.&#xD;
&#xD;
          -  Any current immunosuppressive therapy other than intermittent or low dose&#xD;
             corticosteroids.&#xD;
&#xD;
          -  Alanine transaminase/aspartate aminotransferase (ALT/AST) &gt;1.5 times the upper limit&#xD;
             of normal or any known liver disease.&#xD;
&#xD;
          -  Renal insufficiency, as defined by creatine level &gt;1.3 mg/dL.&#xD;
&#xD;
          -  A hemoglobin concentration of less than 10 gm/dL, a platelet count of less than&#xD;
             100k/mm3 or an absolute neutrophil count of less than 1000/mm3 at study entry.&#xD;
&#xD;
          -  Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.&#xD;
&#xD;
          -  Current participation in any other clinical trial.&#xD;
&#xD;
          -  Participation within previous 6 months in any clinical trial of a drug by ocular or&#xD;
             systemic administration.&#xD;
&#xD;
          -  Any other sight-threatening ocular disease.&#xD;
&#xD;
          -  Any history of retinal vascular disease (compromised blood-retinal barrier. Glaucoma.&#xD;
&#xD;
          -  Uveitis or other intraocular inflammatory disease.&#xD;
&#xD;
          -  Significant lens opacities or other media opacity.&#xD;
&#xD;
          -  Ocular lens removal within previous 3 months.&#xD;
&#xD;
          -  Ocular surgery in the study eye in the previous 3 months&#xD;
&#xD;
          -  If female, pregnancy or lactation.&#xD;
&#xD;
          -  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>55 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Wonkyung Song, MD. PhD.</last_name>
    <role>Principal Investigator</role>
    <affiliation>CHA Bundang Medical Center</affiliation>
  </overall_official>
  <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>November 2019</verification_date>
  <study_first_submitted>August 22, 2012</study_first_submitted>
  <study_first_submitted_qc>August 27, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 29, 2012</study_first_posted>
  <last_update_submitted>November 14, 2019</last_update_submitted>
  <last_update_submitted_qc>November 14, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">November 18, 2019</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>
  <!-- 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/NCT01674829
org study id: CHA CTP 1101
nct id: NCT01674829
lead sponsor: 
has dmc: Yes
To evaluate the safety and tolerability of MA09-hRPE cellular therapy in patients with
      advanced dry AMD To evaluate the safety of the surgical procedures when used to implant
      MA09-hRPE cells To assess the number of hRPE cells to be transplanted in future studies To
      evaluate on an exploratory basis potential efficacy endpoints to be used in future studies of
      MA09-hRPE cellular therapy.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Safety of hESC derived RPE cells
time frame: 12 months
description: The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe in the absence of:
Any grade 2 (NCI grading system) 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: exploratory evaluations for potential efficacy endpoints
time frame: 12months
description: Secondary endpoints will be evaluated as exploratory evaluations for potential efficacy endpoints.
Change in the mean of BCVA
Autofluorescense photography
Reading speed
Evidence of successful engraftment will consist of:
Structural evidence (OCT imaging, fluorescein angiography, slitlamp examination with fundus photography) that cells have been implanted in the correct location
Electroretinographic evidence (mfERG) showing enhanced activity in the implant location
arm group label: Biological: MA09-hRPE Cellular therapy
arm group type: Experimental
description: Biological: MA09-hRPE Cellular therapy
Cohort 1 50,000 cells
Cohort 2 100,000 cells
Cohort 3 150,000 cells
Cohort 4 200,000 cells
intervention type: Biological
intervention name: MA09-hRPE
arm group label: Biological: MA09-hRPE Cellular therapy
other name: Cohort 1- 50,000 MA09-hRPE cells transplanted
other name: Cohort 2- 100,000 MA09-hRPE cells transplanted
other name: Cohort 3- 150,000 MA09-hRPE cells transplanted
other name: Cohort 4- 200,000 MA09-hRPE cells transplanted
criteria: 
gender: All
minimum age: 55 Years
maximum age: N/A
healthy volunteers: No
last name: Wonkyung Song, MD. PhD.
role: Principal Investigator
affiliation: CHA Bundang Medical Center
facility: 
country: Korea, Republic of
responsible party type: Sponsor
mesh term: Macular Degeneration