Rigosertib for RDEB-SCC

Status Recruiting

Clinical Trial 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 patients 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, the investigators 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 a polo-like kinase inhibitor interfering with different molecular pathways that are essential for cancer cell growth. Rigosertib was developed by Onconova Therapeutics and is currently tested in several clinical trials for a number of other cancers including myelodysplastic syndrome (a cancer of the blood). The investigators have identified that rigosertib most selectively kills EB cancer cells in vitro while leaving normal EB skin cells unaffected. This project will evaluate whether rigosertib is capable of inducing an anti-cancer response in EB patients and whether the drug is well-tolerated. Mechanisms of molecular targeting of squamous cancer cells by rigosertib will further be investigated in EB patients, also aiming at the identification of biomarkers that may allow the predictive identification of best responders.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT03786237
Study type	Interventional
Source	Salzburger Landeskliniken
Contact	Manager EB Study Center
Phone	+43 5 7255
Email	Clinical-Trial-eb@salk.at
Status	Recruiting
Phase	Phase 1/Phase 2
Start date	April 12, 2021
Completion date	June 2026

View Details

See also
Status	Clinical Trial	Phase
Terminated	`NCT02213133` - Selinexor Treatment of Advanced Relapsed/Refractory Squamous Cell Carcinomas	Phase 2
Not yet recruiting	`NCT04533321` - A Biomarker-implemented Clinical Study Evaluating Mutations in MET and TP53 in a Population of Treatment-refractory Squamous Cell Carcinoma	Phase 2
Terminated	`NCT02890368` - Trial of Intratumoral Injections of TTI-621 in Subjects With Relapsed and Refractory Solid Tumors and Mycosis Fungoides	Phase 1
Active, not recruiting	`NCT01232374` - Nimotuzumab in Combination With Chemoradiation for Local Advanced Esophageal Squamous Cell Carcinoma	Phase 2
Completed	`NCT01208883` - A Feasibility Study On Continuous Adaptive [18f]Fdg-Pet-Guided Radiotherapy For Head and Neck Cancer	Phase 1
Withdrawn	`NCT01148082` - School Response to Families Who Have Children With Cancer	N/A
Completed	`NCT01089803` - Observational Study of Swallowing Function After Treatment of Advanced Laryngeal Cancer	N/A
Terminated	`NCT00707655` - Zalutumumab in Combination With Radiotherapy in Head and Neck Cancer Patients Ineligible for Platinum Based Chemotherapy	Phase 1/Phase 2
Completed	`NCT01127737` - Warning Signs of Squamous Cell Carcinoma and Prevention of SCC by at Risk Organ Transplant Recipients	N/A
Completed	`NCT00793169` - Serum Concentration of Lidocaine After Local Injection During Mohs Micrographic Surgery
Completed	`NCT00586040` - Photochemical Tissue Bonding	Phase 2
Completed	`NCT00409565` - A Phase II Trial of Cetuximab and Bevacizumab in Patients With Recurrent or Metastatic Head and Neck Cancer	Phase 2
Completed	`NCT00176267` - Paclitaxel, Carboplatin And Low Dose Radiation As Induction Therapy In Locally Advanced Head And Neck Cancer	Phase 2
Terminated	`NCT04685798` - Optimized Diffusion-Weighted Imaging for the Evaluation of Post-Treatment Squamous Cell Carcinoma in the Neck: Comparative Study With FDG PET/CT	N/A
Recruiting	`NCT04370587` - A Clinical Study of Intratumoral MVR-T3011 (T3011) Given as a Single Agent and in Combination With Intravenous Pembrolizumab in Participants With Advanced or Metastatic Solid Tumors	Phase 1/Phase 2
Recruiting	`NCT04475952` - Early Diagnosis of Upper Digestive Tract Disease
Recruiting	`NCT04435938` - A Study of SBRT for Squamous Cell Carcinoma of the Head and Neck	Phase 2
Not yet recruiting	`NCT05852665` - Buccal Cancer Resection Ultrasound Guided	N/A
Recruiting	`NCT05048459` - Comparing Two Surveillance Approaches for People Who Have Received Treatment for HPV-associated Head and Neck Cancer and Show No Signs of Disease	N/A
Suspended	`NCT03952585` - De-intensified Radiation Therapy With Chemotherapy (Cisplatin) or Immunotherapy (Nivolumab) in Treating Patients With Early-Stage, HPV-Positive, Non-Smoking Associated Oropharyngeal Cancer	Phase 2/Phase 3