Therapeutic Efficacy of Topical Sirolimus in Early Stage Cutaneous T-cell Lymphoma (CTCL)
This will be a prospective, non-randomized, open label study of topical sirolimus for the
treatment of CTCL recurrent or refractory to at least one previous skin directed treatment.
The purpose will be evaluation of safety and anti-tumor response as evaluated by serial skin
For subjects with at least partial remission, treatment will be continued for a maximum of 6
months. All subjects will be followed for 6 months from the time of discontinuation of the
study drug or until progression of disease or until a new treatment for CTCL will be
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Overall Response Rate
Determine the efficacy of topical sirolimus in the treatment of early stage CTCL as overall response rate (ORR) Response criteria: Complete response (CR) will be defined as no evidence of clinical skin disease. Partial response (PR) will be defined as a marked improvement in skin disease of > 50% from baseline without new lesions. Stable disease (SD) will be defined as < 25% increase and < 50% clearance in skin disease from baseline without new lesions. Progressive disease (PD) will be defined as > 25% increase in skin disease from baseline or new tumors or loss of response in those with CR or PR (increase in skin score of greater than the sum of nadir plus 50% baseline score). The assessment will be based on Composite Assessment of Index Lesion Severity (CAILS) and, in case of more extensive disease, Modified Severity-Weighed Assessment Tool (mSWAT) scores.
Stefan Schieke, MD
Medical College of Wisconsin
United States: Food and Drug Administration
|Froedtert Hospital and the Medical College of Wisconsin||Milwaukee, Wisconsin 53226|