Recessive Dystrophic Epidermolysis bullosa associated Locally Advanced/Metastatic Squamous Cell Carcinoma
Conditions
Brief summary
To determine the Objective Response Rate (ORR) of therapy with Rigosertib in RDEB patients with locally advanced/metastatic squamous cell carcinoma of the skin using Response Criteria in Solid Tumors Version 1.1 (RECIST1.1) per site assessment up to 52 weeks by CT/MR Scan., To evaluate the safety and tolerability of Rigosertib administered either orally as capsules or dissolved capsules daily for three weeks on, one week off or IV as a 72- hr CIV infusion on days 1, 2, and 3 of a 2-week cycle for the first eight 2-week cycles, then on days 1, 2, and 3 of a 4-week cycle thereafter.
Detailed description
Assess impact on quality of life (QOLEB)., Biomarker analysis (to include markers of PI3K/Akt and PLK1 pathways) performed on all archival tissues from all patients.
Interventions
DRUGRigosertib Sodium IV
DRUGRigosertib Sodium Oral
Sponsors
Eb Haus Austria Gemeinnuetzige Salzburger Landeskliniken Betriebsgesellschaft mbH
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To determine the Objective Response Rate (ORR) of therapy with Rigosertib in RDEB patients with locally advanced/metastatic squamous cell carcinoma of the skin using Response Criteria in Solid Tumors Version 1.1 (RECIST1.1) per site assessment up to 52 weeks by CT/MR Scan., To evaluate the safety and tolerability of Rigosertib administered either orally as capsules or dissolved capsules daily for three weeks on, one week off or IV as a 72- hr CIV infusion on days 1, 2, and 3 of a 2-week cycle for the first eight 2-week cycles, then on days 1, 2, and 3 of a 4-week cycle thereafter. | — |
Secondary
| Measure | Time frame |
|---|---|
| Assess impact on quality of life (QOLEB)., Biomarker analysis (to include markers of PI3K/Akt and PLK1 pathways) performed on all archival tissues from all patients. | — |
Countries
Austria
Outcome results
None listed