Date Updated
EB Type
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Name of Company
Thomas Jefferson University
Recruitment Status
Recruiting
About the Study

The Department of Dermatology & Cutaneous Biology at Thomas Jefferson University is conducting a phase I/II, open-label clinical trial to evaluate the efficacy and safety of Rigosertib in treating Recessive dystrophic epidermolysis bullosa (RDEB) in patients with advanced cutaneous Squamous Cell Carcinoma (SCC).

Rigosertib is a polo-like kinase (PLK)-1 inhibitor that targets critical signals involved in the growth and survival of cancer cells. We previously demonstrated that RDEB SCC keratinocytes are sensitive to PLK1 inhibition while normal primary keratinocytes are unaffected at comparable doses. Based on these findings, Rigosertib has a mechanism of action that could benefit RDEB SCC patients.

Those enrolled will receive Rigosertib either orally or intravenously throughout the 52-week study period. Subjects will be seen every week for the first 5 weeks of the study, and thereafter every 4 weeks until week 52 of the study

Trial Entry Criteria

RDEB patients aged 18-79 diagnosed with unresectable, locally advanced or metastatic cutaneous SCC that has failed to respond to SCC standard of care are eligible for an initial screening visit. At screening, patients must have measurable disease and must not be receiving any other cancer therapy.

Study enrollment is now open at Thomas Jefferson University in Philadelphia, PA. If you qualify, or if you have questions regarding this study, please communicate with our study staff via email:

Primary Investigator: Dr. Neda Nikbakht: neda.nikbakht@jefferson.edu
Study coordinator: Linda Hosler, email: Linda.Hosler@jefferson.edu