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Small Molecule
Rigosertib for Skin Cancer in Dystrophic Epidermolysis Bullosa
Phase < 1
Recruiting
Led By Neda Nikbakht, MD, PhD
Research Sponsored by Thomas Jefferson University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Not currently receiving any other cancer therapy
Failure to respond to SCC standard of care treatments such as surgical excision, radiotherapy, and conventional chemotherapy with specific agents
Must not have
Pregnant or lactating women and women of childbearing potential not using reliable contraception
Uncontrolled intercurrent illness including symptomatic congestive heart failure or unstable angina pectoris
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to12 months post treatment
Awards & highlights
No Placebo-Only Group
Summary
This trial studies how rigosertib sodium, a drug taken orally or through IV, can help treat patients with a rare skin condition and advanced skin cancer who haven't responded to other treatments. The drug targets and kills cancer cells while leaving healthy cells alone. Rigosertib has shown potential as a therapeutic option for nonresectable cholangiocarcinoma, demonstrating antitumoral and radiosensitizing effects.
Who is the study for?
This trial is for adults aged 18-79 with Recessive Dystrophic Epidermolysis Bullosa (RDEB) who have Squamous Cell Carcinoma (SCC) that hasn't improved after standard treatments. Participants must not be on other cancer therapies, should understand the study and agree to participate, and need measurable SCC based on certain criteria. Pregnant women, those with active infections like HIV or hepatitis, uncontrolled health issues, or abnormal lab results can't join.
What is being tested?
The trial is testing how well rigosertib sodium works in treating patients with advanced skin cancer associated with RDEB. The goal is to see if rigosertib targets cancer cells without harming normal cells. Patients' quality of life will also be assessed during the study.
What are the potential side effects?
While specific side effects of rigosertib are not listed here, common side effects from similar medications may include nausea, fatigue, blood count changes leading to increased infection risk or bleeding problems. Allergic reactions to medication components could occur.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am not on any cancer treatment right now.
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My skin cancer hasn't improved with standard treatments like surgery, radiation, or chemotherapy.
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I am between 18 and 79 years old.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I am not pregnant or breastfeeding and use reliable birth control.
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I do not have any uncontrolled illnesses like severe heart failure.
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I have a health condition that causes abnormal vital signs that cannot be corrected.
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I have an active HIV, hepatitis B, or hepatitis C infection.
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My blood pressure is not controlled by medication.
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I have an ongoing infection that isn't getting better with treatment.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline up to12 months post treatment
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to12 months post treatment
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Incidence of treatment-related adverse events
Overall Response Rate (ORR)
Secondary study objectives
Biomarker Analysis
Efficacy of rigosertib sodium treatment: The Objective Response Rate
Quality of Life Epidermolysis Bullosa (QOLEB) questionnaire
Other study objectives
Exosome sequencing
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: Treatment (rigosertib sodium)Experimental Treatment2 Interventions
Patients receive rigosertib sodium either oral or IV over a 52 week period. Patients will take oral rigosertib continuously for a total of three weeks, every four-week cycle (three weeks on, one week off drug). For IV, rigosertib is 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.
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Dystrophic Epidermolysis Bullosa (DEB) include protein replacement therapy, gene therapy, and cell therapy. Protein replacement therapy involves delivering recombinant collagen type VII to skin wounds, which helps restore the structural integrity of the skin by replacing the defective or missing protein.
Gene therapy, such as the use of viral vectors to deliver functional versions of the COL7A1 gene, aims to correct the genetic defect at the source, promoting the production of type VII collagen. Cell therapy, including the use of genetically modified autologous skin grafts, involves culturing a patient's keratinocytes with a corrected gene and applying them to affected areas to regenerate healthy skin.
These treatments are crucial for DEB patients as they address the underlying genetic and protein deficiencies, potentially reducing blistering and improving skin stability.
Find a Location
Who is running the clinical trial?
Traws Pharma, Inc.Industry Sponsor
32 Previous Clinical Trials
1,724 Total Patients Enrolled
Thomas Jefferson UniversityLead Sponsor
465 Previous Clinical Trials
176,877 Total Patients Enrolled
Onconova Therapeutics, Inc.Industry Sponsor
31 Previous Clinical Trials
1,660 Total Patients Enrolled