Everolimus in Treating Cutaneous T-cell Lymphoma (CTCL)
Cutaneous T-cell lymphoma is a rare form of lymphoma of the skin. While early stages are usually confined to the skin, later stages may spread to blood, lymph nodes and other organs. At this point, patients usually require systemic chemotherapy. This study will investigate the effect of everolimus as a treatment for patients diagnosed with CTCL that has either not responded to previous treatments or has recurred despite previous treatments. Everolimus is the common name for the commercial drug Afinitor® (Novartis). It is approved by the U.S. Food and Drug Administration (FDA) for use in kidney and brain cancer. In several different forms of lymphomas, everolimus is used as an investigational drug, which means it has not been approved by the FDA for this group of diseases.
Everolimus blocks a protein (mTOR) that helps cells and tumors to grow. Earlier studies have indicated that the drug everolimus may work against lymphomas including cutaneous T-cell lymphomas. Participation in this study will last as long as the study doctor believes disease has not gotten worse, and patients continue to tolerate the study medication for a maximum of 1 year. Once off the treatment, patients will be followed for two years.
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||PHASE II TRIAL OF THE mTOR INHIBITOR EVEROLIMUS IN RELAPSED OR REFRACTORY CUTANEOUS T-CELL LYMPHOMA (CTCL)|
- Efficacy of treatment [ Time Frame: 12 months after beginning treatment ] [ Designated as safety issue: No ]Determine the efficacy of everolimus in the treatment of CTCL as overall response rate (ORR)
- Time to response [ Time Frame: three months ] [ Designated as safety issue: No ]Determine time to response (TTR)/duration of objective response (DOR)
- progression-free survival [ Time Frame: two years after discontinuing study treatment ] [ Designated as safety issue: No ]Determine progression-free survival of CTCL patients treated with everolimus
- Number of participants with adverse events as a measure of safety and tolerability [ Time Frame: Up to one year ] [ Designated as safety issue: Yes ]Determine the adverse event profile and tolerability of everolimus in patients with CTCL
- effect of mTOR on tumors [ Time Frame: one year ] [ Designated as safety issue: No ]Determine mTOR pathway activation and number of regulatory T cells (Tregs) in pre-treated tumor tissue and evaluate changes following treatment
|Study Start Date:||June 2012|
|Estimated Study Completion Date:||April 2016|
|Estimated Primary Completion Date:||April 2014 (Final data collection date for primary outcome measure)|
Experimental: all patients on study
This will be a prospective, phase II non-randomized, open label study of single agent everolimus for the treatment of CTCL recurrent or refractory to at least one previous treatment other than topical medication. The purpose will be evaluation of safety and anti-tumor response as evaluated by serial skin examinations and assessment of tumor burden in tissue and blood.
This study will be conducted in 2 stages. During stage 1 we will enroll a maximum of 11 subjects to evaluate response rate and will only continue to stage 2, if we observe two or more responses. During stage 2, we will expand the study to an overall N of 28 patients.
The study drug everolimus will be self-administered (by the patients themselves). The investigator will instruct the patient to take the study drug exactly as specified in the protocol. Everolimus should be administered orally once daily, preferably in the morning, at the same time every day with our without food. Everolimus tablets should be swallowed whole with a glass of water. The tablets must not be chewed or crushed.
Everolimus will be administered orally as once daily dose of 10 mg continuously from study day 1 until progression of disease or unacceptable toxicity.
If vomiting occurs, no attempt should be made to replace the vomited dose. All dosages prescribed and dispensed to the patient and all dose changes during the study must be recorded.
|Contact: Stefan Schieke, MD||508-580-1020||Stefan.Schieke@bmc.org|
|United States, Massachusetts|
|Boston Medical Center||Recruiting|
|Boston, Massachusetts, United States, 02118|
|Contact: Sally Fennessey 617-638-8261 firstname.lastname@example.org|
|Principal Investigator:||Adam Lerner, MD||Boston University|