1. Efficacy: Objective Response Rate (ORR) (Time Frame - 1 year): The primary (efficacy) objective of this trial is 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.
2. Number of Treatment-related adverse events (Time Frame - 1 year): Safety will be evaluated for all treated patients, who receive at least one dose of Rigosertib, using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0 (http://ctep.cancer.gov). Safety assessments will be based on number of treatment related adverse events per grade.
Secondary outcome:
1. Evaluation of quality of life using the "Quality of life in Epidermolysis bullosa questionnaire (QOLEB)" (Time Frame - 1 year): Assess impact on quality of life using an Epidermolysis bullosa (EB) specific questionnaire developed and published by Murrell et al.2009. Total score is reported (range 0-75; 0 means EB has no affect on ones life, the higher the score the more affect and annoyance/handicap)
2. Rate of presence or absence of cancer specific biomarkers (Time Frame - 1 year): Fixed tissue will be assessed using immuno-histochemistry with antibodies raised against phosphorylated AKT (p473 Akt), phosphorylated C-RAF (p-S338 RAF), phosphorylated ERK and cleaved caspase.
Rigosertib Oral Capsules / Rigosertib Intravenous: Patients will take Rigosertib either as oral capsules or will get intravenous infusions depending on the needs of the patients.
Oral Capsules:
Patients will take oral Rigosertib continuously for a total of three weeks, every four week cycle (three weeks on, one week off drug) for up to 13 cycles. Patients will take 560 mg of oral Rigosertib (ie, 2 capsules of 280 mg) in the morning and in the afternoon, total of 1120mg/day.
Intravenous Infusions:
For IV treatment Rigosertib 1800 mg/24 hr is diluted in 0.9% sodium chloride for injection just prior to dosing and is administered 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.
Quelle: ClinicalTrials.gov
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"Rigosertib for RDEB-SCC"
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