What side effects are associated with this type of intervention?

Common treatments for Dystrophic Epidermolysis Bullosa (DEB) include supportive care, surgical interventions, and experimental therapies like Rigosertib Sodium. Supportive care often involves managing chronic wounds and preventing infections, which can lead to pain, scarring, and potential complications like Squamous Cell Carcinoma (SCC). Surgical interventions, such as esophageal dilation and hand surgeries, carry risks of infection, scarring, and anesthesia-related complications. Experimental therapies, including Rigosertib Sodium, aim to selectively target cancer cells while sparing normal cells. Known side effects of similar targeted therapies can include gastrointestinal disturbances, fatigue, and potential hematologic toxicities. Regular monitoring and individualized treatment plans are essential to manage these side effects effectively.

What prior FDA approvals exist?

Currently, there are no FDA-approved treatments specifically for Dystrophic Epidermolysis Bullosa (DEB) that function similarly to Rigsertib Sodium, which selectively targets cancer cells. Most treatments for DEB focus on symptom management and wound care. Rigsertib Sodium is being studied for its potential to selectively target cancer cells in DEB-associated squamous cell carcinoma, a serious complication of the disease.

What other types of research exist?

Promising novel alternatives to Rigsertib Sodium for Dystrophic Epidermolysis Bullosa patients include gene therapy approaches, such as exon skipping therapies (e.g., viltolarsen and casimersen), and targeted treatments like epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (TKIs). These therapies are being actively researched and have shown potential in clinical trials for related conditions.

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Small Molecule

Rigosertib for Skin Cancer in Dystrophic Epidermolysis Bullosa

Q: What is the mechanism of action of this treatment?

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.

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 below

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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:

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to12 months post treatment for reporting.

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.

0 / 9Eligibility criteria met

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.