Rigosertib for RDEB-SCC
Research type
Research Study
Full title
A Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients with Recessive Dystrophic Epidermolysis bullosa associated Locally Advanced/Metastatic Squamous Cell Carcinoma
IRAS ID
252118
Contact name
Elisabeth Mayr
Contact email
Sponsor organisation
Gem. Salzburger Landeskliniken Betriebs GesmbH, University Hospital of Dermatology, EB-House Austria
Eudract number
2016-003832-19
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 11 months, 31 days
Research summary
Epidermolysis bullosa (EB) is a heritable skin disease characterized by marked fragility of epithelialized tissue with blistering in skin and mucous membranes following the slightest mechanical trauma. Eighty percent of all patients suffering from recessive dystrophic EB (RDEB), a subtype originating from mutations in the COL7A1 gene, develop squamous cell carcinoma – SCC. In RDEB SCC presents early (most patients are in their 20s or 30s) and shows a highly aggressive metastatic course which often leads to premature death at this young age.
In light of scarce data on the efficacy and safety of systemic treatment regimens for advanced SCC, we propose to perform a small, "first in EB " trial of an experimental drug called rigosertib for the treatment of EB cancer. The trial will be conducted in two study centres, in London and Salzburg, and will last approximately 2.5 years with each patient recruited being in the study for 1 year. The drug is an inhibitor of different processes that are essential for cancer cell growth. Rigosertib has been in clinical trials for a number of other cancers, principally myelodysplastic syndrome (a cancer of the blood) and therefore the company that developed the drug, Onconova, have good experience of using rigosertib in patients. We have identified that in the laboratory rigosertib kills EB cancer cells and doesn't harm normal EB skin cells. This project will test whether rigosertib can kill cancer cells in EB patients and whether the drug can be tolerated by patients. If rigosertib can kill cancer cells in an EB patient we will examine the cancer and the patient to try and identify any features of either which can be used to predict whether future patients can also benefit from this treatment.REC name
London - West London & GTAC Research Ethics Committee
REC reference
19/LO/1166
Date of REC Opinion
29 Jan 2020
REC opinion
Further Information Favourable Opinion