<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/NCT01676831</url>
  </required_header>
  <id_info>
    <org_study_id>813320(UPCC03411)</org_study_id>
    <secondary_id>1R01FD004092-01A1</secondary_id>
    <nct_id>NCT01676831</nct_id>
    <nct_alias>NCT01497795</nct_alias>
  </id_info>
  <brief_title>Topical Resiquimod for the Treatment of Early Stage Cutaneous T Cell Lymphoma (CTCL)</brief_title>
  <official_title>A Phase I/IIa, Dose-Ranging Safety and Efficacy Study of Topical Resiquimod for the Treatment of Early Stage Cutaneous T Cell Lymphoma</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Abramson Cancer Center at Penn Medicine</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Abramson Cancer Center at Penn Medicine</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The objective of this study is to explore the safety and the preliminary efficacy of two&#xD;
      concentrations (0.06% and 0.03%)gel that is applied to lesions of early stage (IA, IB,IIA)&#xD;
      Cutaneous T Cell Lymphoma patients.&#xD;
&#xD;
      This study is supported by grant 1R01FD004092-01A1 from the Office of Orphan Products&#xD;
      Development, FDA.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is an open-label, dose-ranging study in subjects with early stage (IA, IB, 2A) CTCL.&#xD;
      Patients with early stage CTCL will be screened for eligibility. Eligible subjects will be&#xD;
      enrolled in up to 2 treatment groups of up to 8 subjects each.&#xD;
&#xD;
      Treatment groups will be:&#xD;
&#xD;
        1. Resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted&#xD;
           according to tolerability. Subjects will begin dosing at 3 times per week (3x/wk), and&#xD;
           will be evaluated at the clinic every two weeks. The dosing frequency (1, 2, 3, 5, or&#xD;
           7x/wk) may be adjusted in a stepwise manner after each two week interval based on the&#xD;
           physician assessment of tolerability (maintained, increased, decreased with or without a&#xD;
           dosing interruption [rest period]). Resiquimod will be applied for 8 weeks (COT1)&#xD;
           followed by a 4 week no-treatment period. If the subject has not required permanent&#xD;
           discontinuation from treatment, the subject will repeat a second course of treatment of&#xD;
           8 weeks (COT2) followed by a 4 week no-treatment period. Subjects will apply up to 500&#xD;
           mg of study drug per day based upon the total surface area that is treated (~250 mg of&#xD;
           product / 50 cm2 of lesion surface area).&#xD;
&#xD;
        2. Resiquimod 0.03% applied as described for Treatment Group 1. However, initial&#xD;
           applications will be 5 times per week with the dosing frequency adjusted upward as&#xD;
           tolerated every two weeks.&#xD;
&#xD;
      The initial cohort will be assigned to Treatment Group 1. After 4 subjects have completed at&#xD;
      least 4 weeks of dosing, a safety review meeting will be conducted by a committee consisting&#xD;
      of the P.I., a biostatistician, and at least one other physician familiar with CTCL&#xD;
      responses. The Safety Review Committee (SRC) will determine, based on the review of the&#xD;
      tolerability data, the starting concentration/frequency of the next group of 4 subjects.&#xD;
&#xD;
      For a given subject the concentration assignment (0.06% or 0.03%) will remain the same (only&#xD;
      frequency may vary). It is planned that approximately 8 subjects will be enrolled in each&#xD;
      group.&#xD;
&#xD;
      A treatment regimen will be considered inadequately tolerated if 2 or more subjects within&#xD;
      the treatment cohort require protocol mandated permanent discontinuation. Treatment regimens&#xD;
      for newly enrolled subjects and/or of current subjects on treatment will be adjusted&#xD;
      accordingly per protocol. The Safety Review Committee (SRC) will determine the occurrence of&#xD;
      any dose-limiting toxicities (DLTs), defined below in Section 6.4.6. A subsequent phase II&#xD;
      study will further explore efficacy of the MTD in an expanded study.&#xD;
&#xD;
      Up to 2 subjects per Treatment Group who discontinue from the study due to reasons unrelated&#xD;
      to safety reasons (e.g. personal, lost to follow-up, etc) may be replaced.&#xD;
&#xD;
      The investigator will determine the target CTCL lesions and treatment area. During each COT,&#xD;
      subjects will treat at least 1 but no more than 4 target lesions in the 0.06% dosing group&#xD;
      and no ,more than 5 target lesions in the 0.03% treatment group with a total combined&#xD;
      treatment area that is ≥25 cm2 but ≤100 cm2. Unless a lesion is considered to have completely&#xD;
      resolved by clinical assessment at 4 weeks post COT1 (PCOT1), subjects will treat the same&#xD;
      baseline target lesions throughout the COT1 and COT2. The amount of drug applied per dose&#xD;
      during each COT may vary depending on the total size of the target lesions but may not exceed&#xD;
      500 mg per day.&#xD;
&#xD;
      For COT1, subjects will be evaluated at baseline, Week 2, 4, 6, 8 and 12 (PCOT1). For COT2,&#xD;
      subjects will be evaluated at Week 12 (PCOT1/COT2 baseline) 14, 16, 18, 20 and 24 (4 weeks&#xD;
      post COT2, PCOT2). The End of study (EOS) will be at PCOT2, or if a subject permanently&#xD;
      discontinues study drug prematurely, at 4 weeks after the last dose.&#xD;
&#xD;
      Rest periods from treatment may be instituted by the investigator as needed to manage&#xD;
      tolerance, with resumption of treatment upon adequate resolution per investigator discretion.&#xD;
&#xD;
      Subjects in both treatment groups who experience a partial response but not a complete&#xD;
      response at week 24 can continue treatment for up to another 12 weeks with the same&#xD;
      concentration.&#xD;
&#xD;
      During this time they will be evaluated every 4 weeks up to 12 weeks. At that time they will&#xD;
      have a 4 week rest period and then a final evaluation will be performed.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>February 2012</start_date>
  <completion_date type="Actual">September 28, 2015</completion_date>
  <primary_completion_date type="Actual">January 2015</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>Non-Randomized</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>The Number of Participants That Tolerated the Maximum Drug Dose</measure>
    <time_frame>after 4 subjects have completed 4 weeks of study drug</time_frame>
    <description>After four subjects have completed at least four weeks of study drug dosing a safety review meeting will be conducted by a safety review committee. No subjects will be enrolled in the next concentration (0.03%)group until all eight have been evaluated in the 0.06% group.&#xD;
The safety review committee reviews all patient data including adverse events to indicate whether the patient can escalate to the highest dose.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Secondary End Points: Efficacy- CAILDS SCORE</measure>
    <time_frame>Up to 24 weeks or At the conclusion of patient therapy</time_frame>
    <description>The secondary endpoint is:&#xD;
• The Response Rate (Complete or Partial Response) based on Composite Assessment of Index Lesions Disease Severity (CAILDS) score at EOS for the baseline target lesions. Complete Response is defined as a score of '0' on the CAILDS scale. Partial response is defined as a reduction of at least 50% in the CAILDS. The outcome measure will indicate how many received complete response (CR), partial response (PR), and stable disease (SD).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Secondary End Points: Efficacy- SWAT SCORE</measure>
    <time_frame>Up to 24 weeks or At the conclusion of patient therapy</time_frame>
    <description>The secondary endpoint is:&#xD;
The total surface area of involvement will also be assessed (SWAT score). A partial response will represent improvement in 50% or greater of the skin surface area with regression of lesions. A complete response will represent complete clear clearing of skin lesions and in the case of stage IIA patients, resolution of lymphadenopathy. The outcome measure will indicate how many received complete response (CR), partial response (PR), and stable disease (SD).</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">13</enrollment>
  <condition>Cutaneous T Cell Lymphoma</condition>
  <arm_group>
    <arm_group_label>topical resiquimod 0.06%</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>topical resiquimod 0.03%</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Topical resiquimod 0.06%</intervention_name>
    <description>topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.</description>
    <arm_group_label>topical resiquimod 0.06%</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>topical resiquimod 0.03%</intervention_name>
    <description>topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.</description>
    <arm_group_label>topical resiquimod 0.03%</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Males or female ≥18 years of age at the time of study enrollment&#xD;
&#xD;
          2. Have a clinical diagnosis of cutaneous T cell lymphoma CTCL, including documentation&#xD;
             of a skin biopsy with histological findings consistent with CTCL (atypical&#xD;
             epidermotrophic or folliculocentric T-cells). Unconfirmed diagnosis of CTCL must have&#xD;
             a biopsy to confirm at screening&#xD;
&#xD;
          3. Have Stage IA, IB or IIA: T1 or T2 (patches or plaques) with measurable lesions.&#xD;
&#xD;
          4. Previous treatment with at least one standard therapy used to treat Stage IA, IB or&#xD;
             IIA CTCL including but not limited to oral corticosteroids, high-potency topical&#xD;
             corticosteroids, topical mechlorethamine, topical bexarotene, PUVA, UVB, total body&#xD;
             electron beam radiation, biological response or oral methotrexate.&#xD;
&#xD;
          5. Have measurable skin disease with at least 1 to 4 eligible baseline target lesions&#xD;
             with a total area &gt;25 cm2 but &lt;100 cm2. Eligible lesions must be below the neck and&#xD;
             may not involve the genitalia, intertriginous areas, internally, or to frankly&#xD;
             ulcerated or infected skin.&#xD;
&#xD;
          6. Generally healthy other than for CTCL, or with other stable diseases/conditions that&#xD;
             are adequately controlled.&#xD;
&#xD;
          7. Willing and able to provide written informed consent.&#xD;
&#xD;
          8. Willing and able to adhere to the protocol requirements, including but not limited to&#xD;
             study drug dosing, study drug visits, medication and treatment restrictions, and&#xD;
             laboratory tests.&#xD;
&#xD;
          9. Willing and able to discontinue concomitant medications or treatments for CTCL during&#xD;
             the study.&#xD;
&#xD;
         10. If a female of child bearing potential, willing to use adequate contraception (defined&#xD;
             as double-method contraception, e.g. oral contraceptive usage by subject and condom by&#xD;
             partner). Non-child bearing potential is defined as being at least 2 years&#xD;
             post-menopausal or being surgically sterile.&#xD;
&#xD;
         11. Willing to abstain from therapeutic sunbathing, tanning beds, etc. for the duration of&#xD;
             the study.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Have a known allergy to resiquimod or any of the excipients in the study drug.&#xD;
&#xD;
          2. Stage IIB or greater CTCL.&#xD;
&#xD;
          3. Require immediate treatment for progressive CTCL.&#xD;
&#xD;
          4. Are unable to discontinue current treatment for CTCL due to risk of progression.&#xD;
&#xD;
          5. Within 8 weeks of treatment initiation (Day 0), have received treatment with:&#xD;
&#xD;
               -  Total body electron beam radiation&#xD;
&#xD;
               -  Investigational drugs or treatments&#xD;
&#xD;
          6. Within 4 weeks of treatment initiation (Day 0), have received treatment with:&#xD;
&#xD;
               -  Local radiation therapy&#xD;
&#xD;
               -  UVB therapy&#xD;
&#xD;
               -  PUVA&#xD;
&#xD;
               -  Any topical chemotherapy&#xD;
&#xD;
               -  Photopheresis&#xD;
&#xD;
               -  Systemic retinoids, corticosteroids, immune response modifiers including&#xD;
                  imiquimod, interferon inducers, chemotherapeutic agents, biologic agents&#xD;
                  including interferon&#xD;
&#xD;
               -  Topical corticosteroids or retinoids&#xD;
&#xD;
          7. Within 2 weeks of treatment initiation (Day 0), have received at or adjacent to the&#xD;
             target treatment lesions.&#xD;
&#xD;
               -  Any surgical procedures other than biopsies related to CTCL diagnosis or&#xD;
                  follow-up&#xD;
&#xD;
               -  Any topical treatment other than bland moisturizers (creams, lotions, emollients,&#xD;
                  etc).&#xD;
&#xD;
          8. Have other concurrent cutaneous conditions in the treatment area or immediately&#xD;
             adjacent to the treatment area that would be exacerbated by resiquimod or interfere&#xD;
             with assessments.&#xD;
&#xD;
          9. Have a grade 2 or greater laboratory abnormalities (CTCAE v4) at baseline for any of&#xD;
             the following:&#xD;
&#xD;
               -  Hemoglobin&#xD;
&#xD;
               -  White blood cell count&#xD;
&#xD;
               -  Platelet count&#xD;
&#xD;
               -  Alanine transferase&#xD;
&#xD;
               -  Aspartate transferase&#xD;
&#xD;
               -  Creatinine&#xD;
&#xD;
         10. Have a known history of or a positive serologic test for infection with human&#xD;
             immunodeficiency virus or human T lymphotrophic virus.&#xD;
&#xD;
         11. Are pregnant or nursing, or intending to become pregnant within the duration of the&#xD;
             study.&#xD;
&#xD;
         12. Have any clinically significant medical conditions that are unstable, progressive, or&#xD;
             inadequately controlled in the opinion of the investigator, that would pose a&#xD;
             potential risk for the subject, result in poor compliance with the study requirements,&#xD;
             or require treatment with an excluded medication or treatment during the study.&#xD;
&#xD;
         13. Have an active chemical or alcohol dependency as assessed by the investigator.&#xD;
&#xD;
         14. Have systemic collagen vascular disorder, systemic autoimmune disease, an organ&#xD;
             transplant or diagnosis of cancer within 5 years other than CTCL (not including basal&#xD;
             cell carcinoma, non-invasive squamous cell cancer of the skin, malignant melanoma in&#xD;
             situ, or cervical carcinoma in situ).&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Alain H Rook, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Pennsylvania</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of Pennsylvania</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2019</verification_date>
  <study_first_submitted>July 15, 2012</study_first_submitted>
  <study_first_submitted_qc>August 30, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 31, 2012</study_first_posted>
  <results_first_submitted>April 12, 2019</results_first_submitted>
  <results_first_submitted_qc>July 23, 2019</results_first_submitted_qc>
  <results_first_posted type="Actual">August 13, 2019</results_first_posted>
  <last_update_submitted>July 23, 2019</last_update_submitted>
  <last_update_submitted_qc>July 23, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">August 13, 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>Lymphoma</mesh_term>
    <mesh_term>Lymphoma, T-Cell</mesh_term>
    <mesh_term>Lymphoma, T-Cell, Peripheral</mesh_term>
    <mesh_term>Lymphoma, T-Cell, Cutaneous</mesh_term>
  </condition_browse>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Topical Resiquimod 0.06%</title>
          <description>Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.</description>
        </group>
        <group group_id="P2">
          <title>Topical Resiquimod 0.03%</title>
          <description>Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="8"/>
                <participants group_id="P2" count="5"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="8"/>
                <participants group_id="P2" count="4"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Topical Resiquimod 0.06%</title>
          <description>Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.</description>
        </group>
        <group group_id="B2">
          <title>Topical Resiquimod 0.03%</title>
          <description>Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.</description>
        </group>
        <group group_id="B3">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="8"/>
            <count group_id="B2" value="5"/>
            <count group_id="B3" value="13"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="8"/>
                    <measurement group_id="B2" value="5"/>
                    <measurement group_id="B3" value="13"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="4"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="7"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="8"/>
                    <measurement group_id="B2" value="5"/>
                    <measurement group_id="B3" value="13"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="3"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="9"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="8"/>
                    <measurement group_id="B2" value="5"/>
                    <measurement group_id="B3" value="13"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>The Number of Participants That Tolerated the Maximum Drug Dose</title>
        <description>After four subjects have completed at least four weeks of study drug dosing a safety review meeting will be conducted by a safety review committee. No subjects will be enrolled in the next concentration (0.03%)group until all eight have been evaluated in the 0.06% group.&#xD;
The safety review committee reviews all patient data including adverse events to indicate whether the patient can escalate to the highest dose.</description>
        <time_frame>after 4 subjects have completed 4 weeks of study drug</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Topical Resiquimod 0.06%</title>
            <description>Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.</description>
          </group>
          <group group_id="O2">
            <title>Topical Resiquimod 0.03%</title>
            <description>Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.</description>
          </group>
        </group_list>
        <measure>
          <title>The Number of Participants That Tolerated the Maximum Drug Dose</title>
          <description>After four subjects have completed at least four weeks of study drug dosing a safety review meeting will be conducted by a safety review committee. No subjects will be enrolled in the next concentration (0.03%)group until all eight have been evaluated in the 0.06% group.&#xD;
The safety review committee reviews all patient data including adverse events to indicate whether the patient can escalate to the highest dose.</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="8"/>
                <count group_id="O2" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="8"/>
                    <measurement group_id="O2" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Secondary End Points: Efficacy- CAILDS SCORE</title>
        <description>The secondary endpoint is:&#xD;
• The Response Rate (Complete or Partial Response) based on Composite Assessment of Index Lesions Disease Severity (CAILDS) score at EOS for the baseline target lesions. Complete Response is defined as a score of '0' on the CAILDS scale. Partial response is defined as a reduction of at least 50% in the CAILDS. The outcome measure will indicate how many received complete response (CR), partial response (PR), and stable disease (SD).</description>
        <time_frame>Up to 24 weeks or At the conclusion of patient therapy</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Topical Resiquimod 0.06%</title>
            <description>Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.</description>
          </group>
          <group group_id="O2">
            <title>Topical Resiquimod 0.03%</title>
            <description>Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.</description>
          </group>
        </group_list>
        <measure>
          <title>Secondary End Points: Efficacy- CAILDS SCORE</title>
          <description>The secondary endpoint is:&#xD;
• The Response Rate (Complete or Partial Response) based on Composite Assessment of Index Lesions Disease Severity (CAILDS) score at EOS for the baseline target lesions. Complete Response is defined as a score of '0' on the CAILDS scale. Partial response is defined as a reduction of at least 50% in the CAILDS. The outcome measure will indicate how many received complete response (CR), partial response (PR), and stable disease (SD).</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="8"/>
                <count group_id="O2" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Complete Response (CR)</title>
                  <measurement_list>
                    <measurement group_id="O1" value="4"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Partial Response (PR)</title>
                  <measurement_list>
                    <measurement group_id="O1" value="3"/>
                    <measurement group_id="O2" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Stable Disease (SD)</title>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Secondary End Points: Efficacy- SWAT SCORE</title>
        <description>The secondary endpoint is:&#xD;
The total surface area of involvement will also be assessed (SWAT score). A partial response will represent improvement in 50% or greater of the skin surface area with regression of lesions. A complete response will represent complete clear clearing of skin lesions and in the case of stage IIA patients, resolution of lymphadenopathy. The outcome measure will indicate how many received complete response (CR), partial response (PR), and stable disease (SD).</description>
        <time_frame>Up to 24 weeks or At the conclusion of patient therapy</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Topical Resiquimod 0.06%</title>
            <description>Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.</description>
          </group>
          <group group_id="O2">
            <title>Topical Resiquimod 0.03%</title>
            <description>Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.</description>
          </group>
        </group_list>
        <measure>
          <title>Secondary End Points: Efficacy- SWAT SCORE</title>
          <description>The secondary endpoint is:&#xD;
The total surface area of involvement will also be assessed (SWAT score). A partial response will represent improvement in 50% or greater of the skin surface area with regression of lesions. A complete response will represent complete clear clearing of skin lesions and in the case of stage IIA patients, resolution of lymphadenopathy. The outcome measure will indicate how many received complete response (CR), partial response (PR), and stable disease (SD).</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="8"/>
                <count group_id="O2" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Complete Response (CR)</title>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Partial Response (PR)</title>
                  <measurement_list>
                    <measurement group_id="O1" value="5"/>
                    <measurement group_id="O2" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Stable Disease (SD)</title>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>through study completion (3years)</time_frame>
      <desc>Patient was assessed at each study visit.</desc>
      <group_list>
        <group group_id="E1">
          <title>Topical Resiquimod 0.06%</title>
          <description>Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.</description>
        </group>
        <group group_id="E2">
          <title>Topical Resiquimod 0.03%</title>
          <description>Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.&#xD;
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.&#xD;
13 patients were consented and enrolled. 1 patient dropped out of the clinical study this patient was in the 0.03% arm of the study. As such 5 patients were measured for adverse events</description>
        </group>
      </group_list>
      <serious_events>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="8"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="8"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="6" subjects_at_risk="8"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>headache</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="8"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>myalgia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="8"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Immune system disorders</title>
            <event_list>
              <event>
                <sub_title>fever</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="8"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>skin erosion</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="8"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>skin irritation</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="8"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>All Principal Investigators ARE employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Study Coordinator</name_or_title>
      <organization>Hospital of the University of Pennsylvania</organization>
      <phone>215-662-6722</phone>
      <email>deborah.leahy@uphs.upenn.edu</email>
    </point_of_contact>
  </clinical_results>
</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/NCT01676831
org study id: 813320(UPCC03411)
secondary id: 1R01FD004092-01A1
nct id: NCT01676831
nct alias: NCT01497795
lead sponsor: 
has dmc: Yes
The objective of this study is to explore the safety and the preliminary efficacy of two
      concentrations (0.06% and 0.03%)gel that is applied to lesions of early stage (IA, IB,IIA)
      Cutaneous T Cell Lymphoma patients.

      This study is supported by grant 1R01FD004092-01A1 from the Office of Orphan Products
      Development, FDA.
This is an open-label, dose-ranging study in subjects with early stage (IA, IB, 2A) CTCL.
      Patients with early stage CTCL will be screened for eligibility. Eligible subjects will be
      enrolled in up to 2 treatment groups of up to 8 subjects each.

      Treatment groups will be:

        1. Resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted
           according to tolerability. Subjects will begin dosing at 3 times per week (3x/wk), and
           will be evaluated at the clinic every two weeks. The dosing frequency (1, 2, 3, 5, or
           7x/wk) may be adjusted in a stepwise manner after each two week interval based on the
           physician assessment of tolerability (maintained, increased, decreased with or without a
           dosing interruption [rest period]). Resiquimod will be applied for 8 weeks (COT1)
           followed by a 4 week no-treatment period. If the subject has not required permanent
           discontinuation from treatment, the subject will repeat a second course of treatment of
           8 weeks (COT2) followed by a 4 week no-treatment period. Subjects will apply up to 500
           mg of study drug per day based upon the total surface area that is treated (~250 mg of
           product / 50 cm2 of lesion surface area).

        2. Resiquimod 0.03% applied as described for Treatment Group 1. However, initial
           applications will be 5 times per week with the dosing frequency adjusted upward as
           tolerated every two weeks.

      The initial cohort will be assigned to Treatment Group 1. After 4 subjects have completed at
      least 4 weeks of dosing, a safety review meeting will be conducted by a committee consisting
      of the P.I., a biostatistician, and at least one other physician familiar with CTCL
      responses. The Safety Review Committee (SRC) will determine, based on the review of the
      tolerability data, the starting concentration/frequency of the next group of 4 subjects.

      For a given subject the concentration assignment (0.06% or 0.03%) will remain the same (only
      frequency may vary). It is planned that approximately 8 subjects will be enrolled in each
      group.

      A treatment regimen will be considered inadequately tolerated if 2 or more subjects within
      the treatment cohort require protocol mandated permanent discontinuation. Treatment regimens
      for newly enrolled subjects and/or of current subjects on treatment will be adjusted
      accordingly per protocol. The Safety Review Committee (SRC) will determine the occurrence of
      any dose-limiting toxicities (DLTs), defined below in Section 6.4.6. A subsequent phase II
      study will further explore efficacy of the MTD in an expanded study.

      Up to 2 subjects per Treatment Group who discontinue from the study due to reasons unrelated
      to safety reasons (e.g. personal, lost to follow-up, etc) may be replaced.

      The investigator will determine the target CTCL lesions and treatment area. During each COT,
      subjects will treat at least 1 but no more than 4 target lesions in the 0.06% dosing group
      and no ,more than 5 target lesions in the 0.03% treatment group with a total combined
      treatment area that is ≥25 cm2 but ≤100 cm2. Unless a lesion is considered to have completely
      resolved by clinical assessment at 4 weeks post COT1 (PCOT1), subjects will treat the same
      baseline target lesions throughout the COT1 and COT2. The amount of drug applied per dose
      during each COT may vary depending on the total size of the target lesions but may not exceed
      500 mg per day.

      For COT1, subjects will be evaluated at baseline, Week 2, 4, 6, 8 and 12 (PCOT1). For COT2,
      subjects will be evaluated at Week 12 (PCOT1/COT2 baseline) 14, 16, 18, 20 and 24 (4 weeks
      post COT2, PCOT2). The End of study (EOS) will be at PCOT2, or if a subject permanently
      discontinues study drug prematurely, at 4 weeks after the last dose.

      Rest periods from treatment may be instituted by the investigator as needed to manage
      tolerance, with resumption of treatment upon adequate resolution per investigator discretion.

      Subjects in both treatment groups who experience a partial response but not a complete
      response at week 24 can continue treatment for up to another 12 weeks with the same
      concentration.

      During this time they will be evaluated every 4 weeks up to 12 weeks. At that time they will
      have a 4 week rest period and then a final evaluation will be performed.
allocation: Non-Randomized
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: The Number of Participants That Tolerated the Maximum Drug Dose
time frame: after 4 subjects have completed 4 weeks of study drug
description: After four subjects have completed at least four weeks of study drug dosing a safety review meeting will be conducted by a safety review committee. No subjects will be enrolled in the next concentration (0.03%)group until all eight have been evaluated in the 0.06% group.
The safety review committee reviews all patient data including adverse events to indicate whether the patient can escalate to the highest dose.
measure: Secondary End Points: Efficacy- CAILDS SCORE
time frame: Up to 24 weeks or At the conclusion of patient therapy
description: The secondary endpoint is:
• The Response Rate (Complete or Partial Response) based on Composite Assessment of Index Lesions Disease Severity (CAILDS) score at EOS for the baseline target lesions. Complete Response is defined as a score of '0' on the CAILDS scale. Partial response is defined as a reduction of at least 50% in the CAILDS. The outcome measure will indicate how many received complete response (CR), partial response (PR), and stable disease (SD).
measure: Secondary End Points: Efficacy- SWAT SCORE
time frame: Up to 24 weeks or At the conclusion of patient therapy
description: The secondary endpoint is:
The total surface area of involvement will also be assessed (SWAT score). A partial response will represent improvement in 50% or greater of the skin surface area with regression of lesions. A complete response will represent complete clear clearing of skin lesions and in the case of stage IIA patients, resolution of lymphadenopathy. The outcome measure will indicate how many received complete response (CR), partial response (PR), and stable disease (SD).
arm group label: topical resiquimod 0.06%
arm group type: Experimental
description: Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
arm group label: topical resiquimod 0.03%
arm group type: Experimental
description: Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
intervention type: Drug
intervention name: Topical resiquimod 0.06%
description: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.
arm group label: topical resiquimod 0.06%
intervention type: Drug
intervention name: topical resiquimod 0.03%
description: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.
arm group label: topical resiquimod 0.03%
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Alain H Rook, M.D.
role: Principal Investigator
affiliation: University of Pennsylvania
facility: 
country: United States
responsible party type: Sponsor
mesh term: Lymphoma
mesh term: Lymphoma, T-Cell
mesh term: Lymphoma, T-Cell, Peripheral
mesh term: Lymphoma, T-Cell, Cutaneous
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 