<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/NCT01678963</url>
  </required_header>
  <id_info>
    <org_study_id>OHR-002</org_study_id>
    <nct_id>NCT01678963</nct_id>
  </id_info>
  <brief_title>Efficacy and Safety of Squalamine Lactate Eye Drops in Subjects With Neovascular (Wet) Age-related Macular Degeneration (AMD)</brief_title>
  <official_title>Phase II Study of the Efficacy and Safety of Squalamine Lactate Ophthalmic Formulation 0.2% BID in Subjects With Neovascular AMD.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Ohr Pharmaceutical Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Ohr Pharmaceutical Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to evaluate the safety and efficacy of topical ophthalmic&#xD;
      squalamine lactate eye drops in treating patients with neovascular age-related macular&#xD;
      degeneration (wet AMD), a degenerative retinal eye disease that causes a progressive,&#xD;
      irreversible, severe loss of central vision.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Age-related macular degeneration (AMD) is a degenerative retinal eye disease that causes a&#xD;
      progressive loss of central vision. AMD is the leading cause of legal blindness among adults&#xD;
      age 50 or older in the Western world and affects 25-30 million people globally. This number&#xD;
      is expected to triple over the next 25 years. Central vision loss from AMD is caused by the&#xD;
      degeneration of light-sensing cells in the macula called photoreceptors. The macula, the&#xD;
      central portion of the retina, is responsible for perceiving fine visual detail. As&#xD;
      photoreceptors begin to degenerate, so does the individual's central vision. The extent of&#xD;
      vision loss varies widely and is related to the type of AMD, its severity and other&#xD;
      individual characteristics.&#xD;
&#xD;
      AMD presents itself in two different forms - a &quot;dry&quot; form and the more severe &quot;wet&quot; form. Dry&#xD;
      AMD, the more common and milder form of AMD, accounts for 85% to 90% of all cases. It results&#xD;
      in varying forms of sight loss and may or may not eventually develop into the wet form.&#xD;
      Although the wet form of AMD accounts for only 10% to 15% of all AMD, the chance for severe&#xD;
      sight loss is much greater. Wet AMD is responsible for 90% of severe vision loss associated&#xD;
      with AMD. Approximately 500,000 new cases of wet AMD are diagnosed annually worldwide. In&#xD;
      North America alone, approximately 200,000 new cases of wet AMD are diagnosed each year.&#xD;
&#xD;
      Squalamine lactate has been found to be an inhibitor of new blood vessel formation&#xD;
      (angiogenesis) induced by VEGF, PDGF or bFGF. Since angiogenesis is implicated in the growth&#xD;
      and maintenance of choroidal neovascularization, squalamine lactate is potentially an&#xD;
      attractive development candidate in the treatment of age-related macular degeneration (AMD),&#xD;
      in which blood vessel proliferation has a role.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>November 2012</start_date>
  <completion_date type="Actual">March 2015</completion_date>
  <primary_completion_date type="Actual">March 2015</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>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Need for continued concomitant therapy</measure>
    <time_frame>9 months</time_frame>
    <description>Lucentis (ranibizumab) is the current standard of care for the treatment of wet AMD. All patients will receive an initial injection of Lucentis prior to randomization and then be evaluated monthly for their need for further Lucentis injections using protocol defined retreatment criteria.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Best Corrected Visual Acuity (BCVA)</measure>
    <time_frame>9 months</time_frame>
    <description>Evaluation of the effect of treatment on visual function (BCVA) as measured using the EDTRS chart measured at an initial distance of 4 meters.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of subjects with adverse events as a measure of safety and tolerability</measure>
    <time_frame>9 months</time_frame>
    <description>The frequency, severity, seriousness of all adverse events including their relationship to study drug and effect on discontinuation from the study will be monitored, recorded and analysed.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">142</enrollment>
  <condition>Neovascular Age Related Macular Degeneration</condition>
  <arm_group>
    <arm_group_label>Squalamine</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Squalamine eye drop 0.2%</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Vehicle Control</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Eye drop vehicle control</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Squalamine lactate</intervention_name>
    <description>Ophthalmic solution 0.2%</description>
    <arm_group_label>Squalamine</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Vehicle control</intervention_name>
    <description>Ophthalmic solution vehicle control</description>
    <arm_group_label>Vehicle Control</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  ≥50 years of age, male or female&#xD;
&#xD;
          -  Have the following criteria in the study eye:&#xD;
&#xD;
               -  A diagnosis of choroidal neovascularization secondary to AMD with total lesion&#xD;
                  area ≤ 12 disc areas with CNV affecting at least 50% of the total lesion area, in&#xD;
                  at least one eye confirmed by fluorescein angiography (via the reading center)&#xD;
&#xD;
               -  Central Retinal Thickness (SD- OCT central 1 mm) of ≥ 300 um&#xD;
&#xD;
               -  Presence of sub-retinal fluid or cystoid edema on OCT. Pigment epithelial&#xD;
                  detachments without subretinal fluid or cystoid edema will be excluded&#xD;
&#xD;
               -  BCVA 20/40 to 20/230 (25 to 70 letters ETDRS)&#xD;
&#xD;
               -  If both eyes qualify the eye with the greater CRT will be the study eye. If both&#xD;
                  equal the right eye will be selected as the study eye.&#xD;
&#xD;
          -  Female subjects must be 1-year postmenopausal or surgically sterilized, Women of&#xD;
             childbearing potential must have a negative urine pregnancy test and must use an&#xD;
             acceptable method of contraception throughout the study.&#xD;
&#xD;
          -  Be willing and able to provide signed informed consent prior to participation in any&#xD;
             study-related procedures.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Neovascularization secondary to any condition other than AMD in the study eye.&#xD;
&#xD;
          -  Blood occupying greater than 50% of the AMD lesion. Blood underlying the fovea.&#xD;
&#xD;
          -  Prior treatment in the study eye with bevacizumab, ranibizumab, aflibercept, PDT,&#xD;
             submacular surgery, any antiangiogenic drug.&#xD;
&#xD;
          -  Confounding ocular conditions in the study eye which will affect interpretation of&#xD;
             OCT, VA or assessment of macular appearance eg: cataract.&#xD;
&#xD;
          -  Subjects with VA worse than 20/200 (less than 34 letters) in the fellow (non-study)&#xD;
             eye.&#xD;
&#xD;
          -  Fibrosis or atrophy, retinal epithelial tear in the center of the fovea in the study&#xD;
             eye or any condition preventing VA improvement.&#xD;
&#xD;
          -  Prior ocular surgery in the study eye (Vitrectomy, scleral buckle, or glaucoma&#xD;
             filter/shunt). Cataract surgery more than 3 months prior to enrollment is allowed so&#xD;
             long as a posterior chamber intraocular lens is in place.&#xD;
&#xD;
          -  Wearing contact lenses.&#xD;
&#xD;
          -  Concomitant therapy with any drug that may affect VA, meds that may be toxic to the&#xD;
             lens/retina or optic nerve.&#xD;
&#xD;
          -  Current ocular or periocular infection in the study eye.&#xD;
&#xD;
          -  Hypersensitivity to Lucentis.&#xD;
&#xD;
          -  Hypersensitivity to squalamine or any component of the ophthalmic formulation&#xD;
&#xD;
          -  Presence of a life threatening disease or currently on treatment for a malignancy.&#xD;
&#xD;
          -  Currently on chemotherapy.&#xD;
&#xD;
          -  Currently on systemic steroids.&#xD;
&#xD;
          -  Pregnant or lactating.&#xD;
&#xD;
          -  Investigational product use of any kind in the previous 30 days.&#xD;
&#xD;
          -  Subjects for whom attendance for monthly examinations may be unreliable eg: dependent&#xD;
             on an elderly caregiver.&#xD;
&#xD;
          -  Glaucoma in the study eye (glaucomatous visual field defect and receiving treatment).&#xD;
&#xD;
          -  Myocardial infarction or cerebrovascular accident or transient ischemic attacks (TIA)&#xD;
             within the past 6 months.&#xD;
&#xD;
          -  Clinical evidence of diabetic retinopathy or diabetic macular edema in the study eye.&#xD;
&#xD;
          -  Uncontrolled hypertension (Diastolic BP &gt;105 mmHg) in spite of antihypertensive&#xD;
             medications.&#xD;
&#xD;
          -  Subjects known to have HIV.&#xD;
&#xD;
          -  A history of drug or alcohol abuse.&#xD;
&#xD;
          -  Subjects unable to administer eye drops reliably.&#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>Retina Associates SW</name>
      <address>
        <city>Tucson</city>
        <state>Arizona</state>
        <zip>85710</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>California Retinal Consultants</name>
      <address>
        <city>Bakersfield</city>
        <state>California</state>
        <zip>93309</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Retina-Vitreous Associates</name>
      <address>
        <city>Beverly Hilss</city>
        <state>California</state>
        <zip>90211</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Colorado Retina</name>
      <address>
        <city>Golden</city>
        <state>Colorado</state>
        <zip>80401</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Florida Eye Microsurgical Institute Inc.</name>
      <address>
        <city>Boynton Beach</city>
        <state>Florida</state>
        <zip>33426</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Retina Health Center</name>
      <address>
        <city>Fort Myers</city>
        <state>Florida</state>
        <zip>33907</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Midwest Eye Institute</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46290</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Elman Retina</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21237</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cumberland Valley Retina Consultants</name>
      <address>
        <city>Hagerstown</city>
        <state>Maryland</state>
        <zip>21740</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ophthalmic Consultants of Boston</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vision Research Foundation</name>
      <address>
        <city>Grand Rapids</city>
        <state>Michigan</state>
        <zip>49546</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vision Research Foundation</name>
      <address>
        <city>Royal Oak</city>
        <state>Michigan</state>
        <zip>48073</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vision Research Foundation</name>
      <address>
        <city>Traverse City</city>
        <state>Michigan</state>
        <zip>49686</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Total Practice Management</name>
      <address>
        <city>New Brunswick</city>
        <state>New Jersey</state>
        <zip>08901</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Macula Care</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10021</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Retina Associates of Cleveland</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44122</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>PA Retina</name>
      <address>
        <city>Camp Hill</city>
        <state>Pennsylvania</state>
        <zip>17011</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>TN Retina</name>
      <address>
        <city>Nashville</city>
        <state>Tennessee</state>
        <zip>37203</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Medical Center Ophthalmology Associates</name>
      <address>
        <city>San Antonio</city>
        <state>Texas</state>
        <zip>78240</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Wisconsin</name>
      <address>
        <city>Madison</city>
        <state>Wisconsin</state>
        <zip>53705</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>June 2015</verification_date>
  <study_first_submitted>August 27, 2012</study_first_submitted>
  <study_first_submitted_qc>August 30, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 5, 2012</study_first_posted>
  <last_update_submitted>June 11, 2015</last_update_submitted>
  <last_update_submitted_qc>June 11, 2015</last_update_submitted_qc>
  <last_update_posted type="Estimate">June 12, 2015</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Neovascular AMD</keyword>
  <keyword>Wet AMD</keyword>
  <keyword>AMD</keyword>
  <keyword>Age related macular degeneration</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Macular Degeneration</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Squalamine</mesh_term>
  </intervention_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/NCT01678963
org study id: OHR-002
nct id: NCT01678963
lead sponsor: 
has dmc: No
The purpose of this study is to evaluate the safety and efficacy of topical ophthalmic
      squalamine lactate eye drops in treating patients with neovascular age-related macular
      degeneration (wet AMD), a degenerative retinal eye disease that causes a progressive,
      irreversible, severe loss of central vision.
Age-related macular degeneration (AMD) is a degenerative retinal eye disease that causes a
      progressive loss of central vision. AMD is the leading cause of legal blindness among adults
      age 50 or older in the Western world and affects 25-30 million people globally. This number
      is expected to triple over the next 25 years. Central vision loss from AMD is caused by the
      degeneration of light-sensing cells in the macula called photoreceptors. The macula, the
      central portion of the retina, is responsible for perceiving fine visual detail. As
      photoreceptors begin to degenerate, so does the individual's central vision. The extent of
      vision loss varies widely and is related to the type of AMD, its severity and other
      individual characteristics.

      AMD presents itself in two different forms - a "dry" form and the more severe "wet" form. Dry
      AMD, the more common and milder form of AMD, accounts for 85% to 90% of all cases. It results
      in varying forms of sight loss and may or may not eventually develop into the wet form.
      Although the wet form of AMD accounts for only 10% to 15% of all AMD, the chance for severe
      sight loss is much greater. Wet AMD is responsible for 90% of severe vision loss associated
      with AMD. Approximately 500,000 new cases of wet AMD are diagnosed annually worldwide. In
      North America alone, approximately 200,000 new cases of wet AMD are diagnosed each year.

      Squalamine lactate has been found to be an inhibitor of new blood vessel formation
      (angiogenesis) induced by VEGF, PDGF or bFGF. Since angiogenesis is implicated in the growth
      and maintenance of choroidal neovascularization, squalamine lactate is potentially an
      attractive development candidate in the treatment of age-related macular degeneration (AMD),
      in which blood vessel proliferation has a role.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Need for continued concomitant therapy
time frame: 9 months
description: Lucentis (ranibizumab) is the current standard of care for the treatment of wet AMD. All patients will receive an initial injection of Lucentis prior to randomization and then be evaluated monthly for their need for further Lucentis injections using protocol defined retreatment criteria.
measure: Best Corrected Visual Acuity (BCVA)
time frame: 9 months
description: Evaluation of the effect of treatment on visual function (BCVA) as measured using the EDTRS chart measured at an initial distance of 4 meters.
measure: Number of subjects with adverse events as a measure of safety and tolerability
time frame: 9 months
description: The frequency, severity, seriousness of all adverse events including their relationship to study drug and effect on discontinuation from the study will be monitored, recorded and analysed.
arm group label: Squalamine
arm group type: Experimental
description: Squalamine eye drop 0.2%
arm group label: Vehicle Control
arm group type: Placebo Comparator
description: Eye drop vehicle control
intervention type: Drug
intervention name: Squalamine lactate
description: Ophthalmic solution 0.2%
arm group label: Squalamine
intervention type: Drug
intervention name: Vehicle control
description: Ophthalmic solution vehicle control
arm group label: Vehicle Control
criteria: 
gender: All
minimum age: 50 Years
maximum age: N/A
healthy volunteers: No
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country: United States
responsible party type: Sponsor
mesh term: Macular Degeneration
mesh term: Squalamine