Rapamycin Therapy in Head and Neck Squamous Cell Carcinoma
- Advanced-stage head and neck cancer (head and neck squamous cell carcinoma [HNSCC]) has moderately successful treatment outcomes, usually involving surgery as part of the standard treatment. Researchers are investigating the use of the drug rapamycin to prevent tumor growth in HNSCC, and are interested in using it to treat individuals with HNSCC that has not been treated previously with other drugs, radiation, or surgery.
- To evaluate the usefulness of rapamycin in decreasing tumor size prior to surgery for head and neck squamous cell carcinoma.
- Individuals at least 18 years of age who have been diagnosed with advanced head and neck squamous cell carcinoma that has not yet been treated.
- Participants will be screened with a physical examination, medical history, blood tests, and imaging studies.
- Approximately 1 month before scheduled surgery, participants will begin to receive rapamycin. Participants will take rapamycin once daily for 21 days, followed by a 7-day period without the drug.
- During the 21-day rapamycin treatment, participants will have weekly study visits to provide blood and urine samples and have possible tumor biopsies and imaging studies such as x-rays or tumor photographs. Participants will have additional study visits for tests 1 day and 1 week after the end of rapamycin treatment, followed by HNSCC surgery.
- Participants will have a final visit to provide blood samples 30 days after surgery.
- Participants medical records will be reviewed 1 year after surgery; however, participants will not need to have further study visits at this time.
Head and Neck Neoplasms
Carcinoma, Squamous Cell
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Pilot Trial Targeting mTOR as a Novel Mechanism-Based Neoadjuvant Therapy for Head and Neck Cancer|
- Change in levels of pS6, pAKt473, and Ki-67 [ Time Frame: 21 days post treatment with rapamycin ] [ Designated as safety issue: No ]
- Changes in tumor size, blood flow, and standardized uptake value [ Time Frame: 21 days post treatment with rapamycin ] [ Designated as safety issue: No ]
- Changes in circulating cytokines [ Time Frame: 21 days post treatment with rapamycin ] [ Designated as safety issue: No ]
- Apoptotic activity and microvessel density [ Time Frame: 21 days post treatment with rapamycin ] [ Designated as safety issue: No ]
- Clinical and laboratory evaluations for safety [ Time Frame: Pre, during and 30 days post treatment ] [ Designated as safety issue: Yes ]
- Safety evaluation of rapamycin therapy [ Time Frame: Up to one year post treatment ] [ Designated as safety issue: Yes ]
- Exploratory studies of possible effects of rapamycin on tumor size [ Time Frame: 21 days post treatment ] [ Designated as safety issue: No ]
- dynamic CT perfusion, FDG-PET, tumor proliferation, apoptosis, microvessel density and modular changes associated with these effects. [ Time Frame: 21 days post treatment ] [ Designated as safety issue: No ]
- Survival status, recurrence of disease, metastases, and adverse events/serious adverse events, including complications of wound healing, which are related to rapamycin therapy [ Time Frame: Up to one year post treatment ] [ Designated as safety issue: Yes ]
|Study Start Date:||August 2010|
|Estimated Study Completion Date:||December 2015|
|Estimated Primary Completion Date:||June 2015 (Final data collection date for primary outcome measure)|
Active Comparator: 1
Subjects will be treated with sirolimus 21 days
21 evaluable subjects will take rapamycin (sirolimus) orally once per day for 21 days. Before and after dosing tumor assessments to include: photographs, CT & amp; PET scans will be done for tumor measurement.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01195922
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||J. Silvio Gutkind, Ph.D.||National Institute of Dental and Craniofacial Research (NIDCR)|