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Sirolimus in Kidney Transplant Patients With Squamous Cell Skin Carcinoma

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by University of Florida
Information provided by (Responsible Party):
University of Florida Identifier:
First received: December 18, 2012
Last updated: April 7, 2014
Last verified: April 2014

Solid organ transplant recipients (SOTR) have a 3-5x increased occurrence of cancer in contrast to the general population with basal and squamous cell skin cancer. The use of immunosuppressant or anti-rejection drugs that are needed after SOTR is known to increase the risk of developing certain kinds of cancer. The purpose of this study is to find out how well Sirolimus (also known as Rapamune) works at treating squamous cell carcinoma in renal transplant patients.

Condition Intervention Phase
Squamous Cell Skin Carcinoma
Drug: Sirolimus
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II Study of Sirolimus in Renal Transplant Patients Diagnosed With New or Recurrent Squamous Cell Skin Carcinoma Currently on Calcineurin-based Immunosuppression.

Resource links provided by NLM:

Further study details as provided by University of Florida:

Primary Outcome Measures:
  • Measure of squamous cell skin carcinoma in patients on sirolimus [ Time Frame: Baseline, time of surgical removal (5 weeks) and 1 year. ] [ Designated as safety issue: No ]
    Baseline: Measuring of squamous cell skin carcinoma, Week 5: Measuring and surgical removal of squamous cell skin cancer with microscopic evaluation, and in 1 year.

Secondary Outcome Measures:
  • Evaluation of skin tumor for squamous cell skin carcinoma after sirolimus therapy. [ Time Frame: At baseline and time of surgical removal (5 weeks). ] [ Designated as safety issue: No ]
    Tumor will be analyzed at baseline and time of surgical removal by both laboratory and microscopic testing.

  • Tumor recurrence. [ Time Frame: From surgical removal to 1 year. ] [ Designated as safety issue: No ]
    Full skin exam done by dermatology.

  • Other cancer development. [ Time Frame: Baseline,5 weeks and 1 year. ] [ Designated as safety issue: No ]
    Full skin exam done by dermatology.

  • Tumor evaluation by western blot analysis. [ Time Frame: At baseline and time of surgical removal(Week 5). ] [ Designated as safety issue: No ]
    Baseline results will be recorded and and then results at time of surgical removal will be reported as increased or decreased expression in comparison to the baseline results.

  • Acute and/or chronic transplant rejection. [ Time Frame: At baseline and 1 year. ] [ Designated as safety issue: Yes ]
    Evaluating for safety reasons of sirolimus.

  • Pre-treatment biopsy immunohistochemistry and PCR testing for oncogenic viruses. [ Time Frame: Baseline. ] [ Designated as safety issue: No ]
    p16 will be assessed for HPV integration and analysis for presence of other oncogenic viruses, EBV, HTLV-1, HHV-8, and the Merkel Cell Virus will be done.

Estimated Enrollment: 43
Study Start Date: April 2013
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: November 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Sirolimus treatment
Patients will receive sirolimus 5 weeks prior to removal of squamous cell skin carcinoma. After the 5 weeks of treatment, nephrology will determine/manage each patient's immunosuppressant therapy.
Drug: Sirolimus
Patients randomized to this arm of the study will receive sirolimus from the time of randomization at least until 5 weeks or the removal of the skin tumor. Nephrology will determine/manage the immunosuppressant therapy.
Other Name: Rapamune

Detailed Description:

This is a Phase II randomized study to evaluate the effectiveness of Sirolimus in treating and preventing squamous cell skin cancer carcinoma using a Simon's 2-stage design. As part of the study, after the biopsy results of the skin cancer show squamous cell carcinoma and consent had been obtain, the participant will begin taking Sirolimus. The calcineurin inhibitor (tacrolimus or cyclosporine) will be discontinued once the transplant doctors find that the participants Sirolimus is therapeutic by checking blood tests once a week x 3 weeks. Approximately 5 weeks after starting Sirolimus, the squamous cell skin cancer will be removed by a surgeon. Participant next follow up visit for the study is scheduled one year post-operatively. .The study will look at your squamous cell skin cancer under the microscope to see if sirolimus had any effect at treating your squamous cell skin cancer.


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

Inclusion Criteria:

  • Histologically or cytologically proven squamous cell skin carcinoma
  • Recipient of a renal organ transplant at least one year prior to study enrollment
  • Receiving a CNI for at least 6 months prior to diagnosis of skin cancer
  • No current evidence of graft rejection, except low-grade, chronic graft rejection
  • Measurable disease by caliper measurement
  • Life expectancy > 6 months
  • Age of at least 18 years
  • Adequate organ and marrow function as determined by ANC, HGB, PLT, Total Bili, AST, and creatinine clearance
  • Ability to understand/willingness to sign a written informed consent form

Exclusion Criteria:

  • Inability to give informed consent
  • Major surgery within 4 week prior to starting study drug
  • Chronic or non-healing open wounds
  • Pregnant and nursing women
  • Women and men of child-bearing potential must agree to use adequate contraception prior to study entry and for the study duration
  • Prior use of an mTOR inhibitor
  • Pre-existing clinically significant cardiac, hepatic, pulmonary, or renal dysfunction
  • HIV-positive patients
  • Proteinuria (> 1 gram)
  • Prior or current history of uncontrolled hyperlipidemia (cholesterol > 302 mg/dl or triglycerides 354 mg/dl
  • Currently receiving any investigational agents
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to sirolimus (mTOR inhibitors)
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrythmia, or psychiatric illness/social situations that would limit compliance with study requirements
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01764607

Contact: Marlene S Sarmiento, RN 352-273-8517
Contact: Priya Gopalan, MD 352-265-0725

United States, Florida
Shands at the University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Marlene S Sarmiento, RN    352-273-8517   
Contact: Priya Gopalan, MD    352-265-0725   
Principal Investigator: Priya Gopalan, MD         
Sponsors and Collaborators
University of Florida
Principal Investigator: Priya Gopalan, MD University of Florida
  More Information

No publications provided

Responsible Party: University of Florida Identifier: NCT01764607     History of Changes
Other Study ID Numbers: 514-2012, 00086505
Study First Received: December 18, 2012
Last Updated: April 7, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Florida:

Additional relevant MeSH terms:
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents
Antineoplastic Agents
Immunologic Factors
Immunosuppressive Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses processed this record on March 01, 2015