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

NCT01676831 | Completed Phase 1 Results DMC

• 2 other identifiers: 813320(UPCC03411)1R01FD004092-01A1
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Cutaneous T Cell Lymphoma

Outcome Measures

Primary Outcomes (1)

• The Number of Participants That Tolerated the Maximum Drug Dose

  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.

  after 4 subjects have completed 4 weeks of study drug

Secondary Outcomes (2)

• Secondary End Points: Efficacy- CAILDS SCORE

  Up to 24 weeks or At the conclusion of patient therapy

• Secondary End Points: Efficacy- SWAT SCORE

  Up to 24 weeks or At the conclusion of patient therapy

Study Arms (2)

topical resiquimod 0.06%

EXPERIMENTAL

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 0.03%

EXPERIMENTAL

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%

Interventions

Topical resiquimod 0.06% DRUG

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.

topical resiquimod 0.06%

topical resiquimod 0.03% DRUG

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.

topical resiquimod 0.03%

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or female ≥18 years of age at the time of study enrollment
• 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
• Have Stage IA, IB or IIA: T1 or T2 (patches or plaques) with measurable lesions.
• 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.
• 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.
• Generally healthy other than for CTCL, or with other stable diseases/conditions that are adequately controlled.
• Willing and able to provide written informed consent.
• 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.
• Willing and able to discontinue concomitant medications or treatments for CTCL during the study.
• 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.
• Willing to abstain from therapeutic sunbathing, tanning beds, etc. for the duration of the study.

You may not qualify if:

• Have a known allergy to resiquimod or any of the excipients in the study drug.
• Stage IIB or greater CTCL.
• Require immediate treatment for progressive CTCL.
• Are unable to discontinue current treatment for CTCL due to risk of progression.
• Within 8 weeks of treatment initiation (Day 0), have received treatment with:
• Total body electron beam radiation
• Investigational drugs or treatments
• 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

Sponsors & Collaborators

• Abramson Cancer Center at Penn Medicine: lead

Study Sites (1)

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (1)

• Rook AH, Gelfand JM, Wysocka M, Troxel AB, Benoit B, Surber C, Elenitsas R, Buchanan MA, Leahy DS, Watanabe R, Kirsch IR, Kim EJ, Clark RA. Topical resiquimod can induce disease regression and enhance T-cell effector functions in cutaneous T-cell lymphoma. Blood. 2015 Sep 17;126(12):1452-61. doi: 10.1182/blood-2015-02-630335. Epub 2015 Jul 30.

  PMID: 26228486 DERIVED

MeSH Terms

Conditions

Lymphoma, T-Cell, Cutaneous

Condition Hierarchy (Ancestors)

Lymphoma, T-Cell; Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Results Point of Contact

• Title: Study Coordinator
• Organization: Hospital of the University of Pennsylvania

deborah.leahy@uphs.upenn.edu

215-662-6722

Study Officials

• Alain H Rook, M.D.

  University of Pennsylvania

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 15, 2012
• First Posted: August 31, 2012
• Study Start: February 1, 2012
• Primary Completion: January 1, 2015
• Study Completion: September 28, 2015
• Last Updated: August 13, 2019
• Results First Posted: August 13, 2019

Record last verified: 2019-07

Locations

US (1)