Topical Resiquimod for the Treatment of Early Stage Cutaneous T Cell Lymphoma (CTCL)

This study is currently recruiting participants.
Verified August 2013 by Abramson Cancer Center of the University of Pennsylvania
Sponsor:
Information provided by (Responsible Party):
Abramson Cancer Center of the University of Pennsylvania
ClinicalTrials.gov Identifier:
NCT01676831
First received: July 15, 2012
Last updated: August 20, 2013
Last verified: August 2013
  Purpose

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.


Condition Intervention Phase
Cutaneous T Cell Lymphoma
Drug: Topical resiquimod 0.06%
Drug: topical resiquimod 0.03%
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Abramson Cancer Center of the University of Pennsylvania:

Primary Outcome Measures:
  • Drug tolerability data [ Time Frame: after 4 subjects have completed 4 weeks of study drug ] [ Designated as safety issue: Yes ]
    After four subjects have completed at least four weeks of study drug dosing a safety review meeting will be conducted by a committee. The committee will determine based on the review of the tolerability data the starting concentration/frequency of the next group. No subjects will be enrolled in the next concentration (0.03%)group until all eight have been evaluated in the 0.06% group.


Secondary Outcome Measures:
  • Secondary end points: Efficacy [ Time Frame: Up to 24 weeks or At the conclusion of patient therapy ] [ Designated as safety issue: No ]

    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 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.



Estimated Enrollment: 20
Study Start Date: February 2012
Estimated Study Completion Date: April 2015
Estimated Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: topical resiquimod 0.06%
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.
Drug: 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.
Experimental: topical resiquimod 0.03%
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.
Drug: 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.

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  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Males or female ≥18 years of age at the time of study enrollment
  2. Have a clinical diagnosis of cutaneous T cell lymphoma CTCL, including documentation of a skin biopsy with histological findings consistent with CTCL (atypical epidermotrophic or folliculocentric T-cells). Unconfirmed diagnosis of CTCL must have a biopsy to confirm at screening
  3. Have Stage IA, IB or IIA: T1 or T2 (patches or plaques) with measurable lesions.
  4. Previous treatment with at least one standard therapy used to treat Stage IA, IB or IIA CTCL including but not limited to oral corticosteroids, high-potency topical corticosteroids, topical mechlorethamine, topical bexarotene, PUVA, UVB, total body electron beam radiation, biological response or oral methotrexate.
  5. Have measurable skin disease with at least 1 to 4 eligible baseline target lesions with a total area >25 cm2 but <100 cm2. Eligible lesions must be below the neck and may not involve the genitalia, intertriginous areas, internally, or to frankly ulcerated or infected skin.
  6. Generally healthy other than for CTCL, or with other stable diseases/conditions that are adequately controlled.
  7. Willing and able to provide written informed consent.
  8. Willing and able to adhere to the protocol requirements, including but not limited to study drug dosing, study drug visits, medication and treatment restrictions, and laboratory tests.
  9. Willing and able to discontinue concomitant medications or treatments for CTCL during the study.
  10. If a female of child bearing potential, willing to use adequate contraception (defined as double-method contraception, e.g. oral contraceptive usage by subject and condom by partner). Non-child bearing potential is defined as being at least 2 years post-menopausal or being surgically sterile.
  11. Willing to abstain from therapeutic sunbathing, tanning beds, etc. for the duration of the study.

Exclusion Criteria:

  1. Have a known allergy to resiquimod or any of the excipients in the study drug.
  2. Stage IIB or greater CTCL.
  3. Require immediate treatment for progressive CTCL.
  4. Are unable to discontinue current treatment for CTCL due to risk of progression.
  5. Within 8 weeks of treatment initiation (Day 0), have received treatment with:

    • Total body electron beam radiation
    • Investigational drugs or treatments
  6. Within 4 weeks of treatment initiation (Day 0), have received treatment with:

    • Local radiation therapy
    • UVB therapy
    • PUVA
    • Any topical chemotherapy
    • Photopheresis
    • Systemic retinoids, corticosteroids, immune response modifiers including imiquimod, interferon inducers, chemotherapeutic agents, biologic agents including interferon
    • Topical corticosteroids or retinoids
  7. Within 2 weeks of treatment initiation (Day 0), have received at or adjacent to the target treatment lesions.

    • Any surgical procedures other than biopsies related to CTCL diagnosis or follow-up
    • Any topical treatment other than bland moisturizers (creams, lotions, emollients, etc).
  8. Have other concurrent cutaneous conditions in the treatment area or immediately adjacent to the treatment area that would be exacerbated by resiquimod or interfere with assessments.
  9. Have a grade 2 or greater laboratory abnormalities (CTCAE v4) at baseline for any of the following:

    • Hemoglobin
    • White blood cell count
    • Platelet count
    • Alanine transferase
    • Aspartate transferase
    • Creatinine
  10. Have a known history of or a positive serologic test for infection with human immunodeficiency virus or human T lymphotrophic virus.
  11. Are pregnant or nursing, or intending to become pregnant within the duration of the study.
  12. Have any clinically significant medical conditions that are unstable, progressive, or inadequately controlled in the opinion of the investigator, that would pose a potential risk for the subject, result in poor compliance with the study requirements, or require treatment with an excluded medication or treatment during the study.
  13. Have an active chemical or alcohol dependency as assessed by the investigator.
  14. Have systemic collagen vascular disorder, systemic autoimmune disease, an organ transplant or diagnosis of cancer within 5 years other than CTCL (not including basal cell carcinoma, non-invasive squamous cell cancer of the skin, malignant melanoma in situ, or cervical carcinoma in situ).
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01676831

Contacts
Contact: Marie A. Buchanan, RN 215 349-5699 marie.buchanan@uphs.upenn.edu

Locations
United States, Pennsylvania
University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Alain Rook, MD    215-662-7610    arook@mail.med.upenn.edu   
Sponsors and Collaborators
Abramson Cancer Center of the University of Pennsylvania
Investigators
Principal Investigator: Alain H Rook, M.D. University of Pennsylvania
  More Information

No publications provided

Responsible Party: Abramson Cancer Center of the University of Pennsylvania
ClinicalTrials.gov Identifier: NCT01676831     History of Changes
Obsolete Identifiers: NCT01497795
Other Study ID Numbers: 813320(UPCC03411), 1R01FD004092-01A1
Study First Received: July 15, 2012
Last Updated: August 20, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Lymphoma
Lymphoma, T-Cell
Lymphoma, T-Cell, Cutaneous
Neoplasms by Histologic Type
Neoplasms
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Lymphoma, Non-Hodgkin

ClinicalTrials.gov processed this record on April 17, 2014