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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04177498
Other study ID # 19G.527
Secondary ID
Status Recruiting
Phase Early Phase 1
First received
Last updated
Start date August 24, 2021
Est. completion date June 1, 2025

Study information

Verified date March 2024
Source Thomas Jefferson University
Contact Neda Nikbakht, MD, PhD
Phone 215-503-4834
Email neda.nikbakht@jefferson.edu
Is FDA regulated No
Health authority
Study type Interventional

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.


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.


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Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Rigosertib Sodium
given PO or 72 hour continuous infusion
Other:
Quality-of-Life Assessment
Ancillary studies

Locations

Country Name City State
United States Sidney Kimmel Cancer Center at Thomas Jefferson University Philadelphia Pennsylvania

Sponsors (2)

Lead Sponsor Collaborator
Thomas Jefferson University Onconova Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Other Exosome sequencing whole exosome sequencing will be done on frozen tumor tissue. The investigators will catalogue somatic mutations identified in each tumor and compare these data with patient response and ex-vivo tumor responses. baseline up to12 months post treatment
Primary Overall Response Rate (ORR) Defined as the proportion of patients who achieve either a complete response (CR) or partial response (PR) baseline up to12 months post treatment
Primary Incidence of treatment-related adverse events Safety assessments will be based on medical review of adverse event reports and the results of vital sign measurements, physical examinations, and clinical laboratory tests. Results will be reported as an aggregate of the adverse events using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), Version 5.0 terminology and grading. baseline up to12 months post treatment
Secondary Quality of Life Epidermolysis Bullosa (QOLEB) questionnaire Descriptive methods used to analyze scores from QOLEB questionnaire. It consists of 17 questions with four response choices from "not at all" to "constant" baseline up to12 months post treatment
Secondary Biomarker Analysis 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. baseline up to12 months post treatment
Secondary Efficacy of rigosertib sodium treatment: The Objective Response Rate The Objective Response Rate (ORR) will be computed with the corresponding exact binomial 90% confidence interval. baseline to end of treatment (52 weeks)
See also
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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
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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
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Completed NCT04227106 - Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) Phase 3
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Completed NCT05143190 - Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01) Phase 2
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