Clinical Trials Logo

Clinical Trial 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.


Clinical Trial 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04177498
Study type Interventional
Source Thomas Jefferson University
Contact Neda Nikbakht, MD, PhD
Phone 215-503-4834
Email neda.nikbakht@jefferson.edu
Status Recruiting
Phase Early Phase 1
Start date August 24, 2021
Completion date June 1, 2025

See also
  Status Clinical Trial Phase
Completed NCT02493816 - Safety Study of Gene-modified Autologous Fibroblasts in Recessive Dystrophic Epidermolysis Bullosa Phase 1
Completed NCT04599881 - A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa Phase 2
Completed NCT04520022 - Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB Phase 1/Phase 2
Not yet recruiting NCT03632265 - Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa Phase 3
Recruiting NCT05944250 - A Pilot Study to Evaluate a Temporary Skin Substitute (Spincare® Matrix) for Wound Healing in RDEB Patients N/A
Recruiting NCT04917887 - Long-Term Follow-up Protocol
Completed NCT04917874 - A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa Phase 3
Not yet recruiting NCT04285294 - Molecular Signatures of Cutaneous Squamous Cell Carcinoma During Recessive Dystrophic Epidermolysis Bullosa
Active, not recruiting NCT04213261 - A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa Phase 3
Active, not recruiting NCT02323789 - Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa Phase 1/Phase 2
Recruiting NCT01874769 - Study of Immune Tolerance and Capacity for Wound Healing of Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) N/A
Recruiting NCT03392909 - Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Phase 1/Phase 2
Completed NCT03752905 - A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) Phase 1/Phase 2
Terminated NCT02984085 - Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB. Phase 1/Phase 2
Completed NCT04227106 - Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) Phase 3
Completed NCT02698735 - Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients Phase 1/Phase 2
Completed NCT03012191 - Gentamicin for RDEB Phase 1/Phase 2
Completed NCT03529877 - Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa Phase 1/Phase 2
Completed NCT05143190 - Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01) Phase 2
Completed NCT04491604 - Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB Phase 3