NCT04177498

Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC

A Pilot, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Thomas Jefferson University · Academic / Other
• Sex: All
• Age: 18 Years – 79 Years
• Healthy volunteers: Not accepted

Summary

This pilot trial studies how rigsertib sodium works in treating patients with Recessive Dystrophic Epidermolysis bullosa (RDEB) with locally advanced Squamous Cell Carcinoma (SCC). Rigosertib may selectively target Epidermolysis bullosa (EB) cancer cells while leaving normal EB cells unaffected.

Detailed description

PRIMARY OBJECTIVES: I. To estimate the anti-tumor activity of oral or IV rigosertib in RDEB patients with advanced SCC that have failed prior standard of care, by determining the overall response rate (ORR) which is defined as the proportion of patients who achieve either a CR or a PR by RECIST v1.1 II. To evaluate the safety and tolerability of oral rigosertib administered either orally daily three weeks on, one week off or as 72h CIV infusions on day 1-3 of a two week-cycle for 8 cycles and then on day 1-3 of a 4 week cycle thereafter SECONDARY OBJECTIVES: I. Assess impact on quality of life (QoL) II. Biomarker analysis (to include markers of PI3K/Akt and PLK1 pathways) performed on all archival tissue from all patients EXPLORATORY OBJECTIVE: I. Exome sequencing of patient tumors before, during, and after treatment OUTLINE: Patients will receive rigosertib sodium as either oral capsules or IV infusion. Mode of application is determined by the responsible investigator depending on participant's needs, general condition, and possibility of ambulatory treatment or need of hospitalization. Patients will take oral rigosertib continuously for a total of three weeks of a four-week cycle (three weeks on, one week off drug). For IV treatment, patients will receive rigosertib IV administered as a 72-hr continuous 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. Patients will receive treatment over a 52 week period. After completion of study treatment, patients are followed periodically every 3 months over a 12 month period.

Conditions

• Recessive Dystrophic Epidermolysis Bullosa

Interventions

Type	Name	Description
DRUG	Rigosertib Sodium	given PO or 72 hour continuous infusion
OTHER	Quality-of-Life Assessment	Ancillary studies

Timeline

• Start date: 2021-08-24
• Primary completion: 2025-05-05
• Completion: 2025-05-05
• First posted: 2019-11-26
• Last updated: 2025-08-01

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study