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Therapeutic Efficacy of Topical Sirolimus in Early Stage Cutaneous T-cell Lymphoma (CTCL)

Phase 1
18 Years
Not Enrolling
Cutaneous T-cell Lymphoma (CTCL)

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Trial Information

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

Study duration:

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

Inclusion Criteria:

- Clinically and histologically confirmed diagnosis of CTCL (early stage disease with
patches and/or thin plaques covering up to 10%, stage IA)

- Relapsed or refractory disease after at least one standard skin directed treatment
including corticosteroids, topical bexarotene, phototherapy

- All subjects must be 18 years of age or older

- Life expectancy ≥ 6 months, determined by the treating physician

- Signed informed consent

Exclusion Criteria:

- Patients currently receiving anticancer therapies or who have received anticancer
therapies within 4 weeks of the start of study drug (including topical or systemic
glucocorticosteroids, chemotherapy, radiation therapy, antibody based therapy, etc.)

- Prior treatment with any investigational drug within the preceding 4 weeks

- Patients receiving chronic, systemic treatment with corticosteroids or another
immunosuppressive agent.

- Female patients who are pregnant or breast feeding, or adults of reproductive
potential who are not using effective birth control methods. Adequate contraception
(oral contraceptives ("the pill"), intrauterine devices (IUDs), contraceptive
implants under the skin, or contraceptive injections, diaphragms with spermicide and
condoms with foam) must be used throughout the trial and for 8 weeks after the last
dose of study drug (women of childbearing potential must have a negative urine within
7 days prior to administration of sirolimus).

- Patients who have received prior treatment with an mTOR inhibitor (e.g., sirolimus,
temsirolimus, everolimus).

- Patients with a known hypersensitivity to sirolimus or other rapamycin (e.g.,
everolimus, temsirolimus) or to its excipient

- History of noncompliance to medical regimens

- Patients unwilling to or unable to comply with the protocol

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Overall Response Rate

Outcome Description:

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.

Outcome Time Frame:

6 months

Safety Issue:


Principal Investigator

Stefan Schieke, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Medical College of Wisconsin


United States: Food and Drug Administration

Study ID:




Start Date:

June 2013

Completion Date:

December 2015

Related Keywords:

  • Cutaneous T-cell Lymphoma (CTCL)
  • Early stage
  • Stage 1A
  • Cutaneous T-cell Lymphoma
  • CTCL
  • Lymphoma
  • Lymphoma, T-Cell
  • Lymphoma, T-Cell, Cutaneous



Froedtert Hospital and the Medical College of WisconsinMilwaukee, Wisconsin  53226