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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

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.

Detailed Summary:

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 tolerabili
Sponsor: Abramson Cancer Center of the University of Pennsylvania

Current Primary Outcome: Drug tolerability data [ Time Frame: after 4 subjects have completed 4 weeks of study drug ]

Original Primary Outcome:

• Drug tolerability data [ Time Frame: after 4 subjects have completed 4 weeks of study drug ]
  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 higher concentration (0.2%). group until all eight have been evaluated in the lower dose (0.06%) group.
• Efficacy Endpoints [ Time Frame: up to 24 weeks ]

  The primary 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.

Current Secondary Outcome: Secondary end points: Efficacy [ Time Frame: Up to 24 weeks or At the conclusion of patient therapy ]

Original Secondary Outcome:

Information By: Abramson Cancer Center of the University of Pennsylvania

Dates:
Date Received: July 15, 2012
Date Started: February 2012
Date Completion:
Last Updated: September 18, 2016
Last Verified: July 2016