Ignite Creation Date:
2024-05-06 @ 12:32 PM
Last Modification Date:
2024-10-26 @ 1:00 PM
Study NCT ID:
NCT03786237
Status:
RECRUITING
Last Update Posted:
2023-11-27
First Post:
2018-11-14
Brief Title:
Rigosertib for RDEB-SCC
Sponsor:
Prof Johann Bauer
Organization:
Salzburger Landeskliniken
Study Overview
Official Title:
A Phase II Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally AdvancedMetastatic Squamous Cell Carcinoma
Status:
RECRUITING
Status Verified Date:
2023-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 25 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
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 25 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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None