Study on Rigosertib Sodium for Patients with Recessive Dystrophic Epidermolysis Bullosa and Advanced Squamous Cell Carcinoma

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What is this study about?

This clinical trial is focused on studying a rare skin condition called Recessive Dystrophic Epidermolysis Bullosa (RDEB), which is associated with a type of skin cancer known as Squamous Cell Carcinoma (SCC). The study is testing a treatment called Rigosertib Sodium, which is being evaluated in two forms: as an intravenous solution (Rigosertib Sodium IV) and as oral capsules (Rigosertib Sodium Oral). Rigosertib Sodium is a chemical substance that works by inhibiting certain pathways in the body that are involved in cancer growth.

The purpose of the study is to assess the safety and effectiveness of Rigosertib Sodium in patients with RDEB who have advanced SCC that has not responded to standard treatments. Participants will receive Rigosertib Sodium either orally or through an infusion. The oral treatment involves taking capsules daily for three weeks followed by a one-week break. The infusion treatment involves receiving the medication over a 72-hour period on the first three days of a two-week cycle for the first eight cycles, and then on the first three days of a four-week cycle thereafter.

The study will last for up to 52 weeks, during which the response to the treatment will be monitored using imaging techniques like CT or MR scans. Additionally, the study will look at the impact of the treatment on the quality of life of participants and analyze certain biological markers related to the pathways affected by Rigosertib Sodium. The goal is to determine how well the treatment works and how it affects the patients’ overall health and well-being.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and a physical examination.

A diagnosis of recessive dystrophic epidermolysis bullosa associated with locally advanced or metastatic squamous cell carcinoma is confirmed through genetic testing or a skin biopsy.

2 informed consent

An informed consent document is provided, explaining the purpose and procedures of the study. Signing this document indicates understanding and willingness to participate.

3 treatment initiation

The treatment involves the administration of rigosertib sodium in two forms: intravenous (IV) and oral.

For the IV form, a 72-hour continuous infusion is administered on days 1, 2, and 3 of a two-week cycle for the first eight cycles. Afterward, the infusion occurs on days 1, 2, and 3 of a four-week cycle.

For the oral form, capsules are taken daily for three weeks, followed by one week off.

4 ongoing monitoring

Throughout the study, regular monitoring is conducted to assess the safety and tolerability of the treatment.

This includes routine check-ups, blood tests, and imaging scans such as CT or MRI to evaluate the response to treatment.

5 quality of life assessment

The impact of the treatment on quality of life is assessed periodically using specific questionnaires designed for this purpose.

6 biomarker analysis

Biomarker analysis is performed on tissue samples to study specific pathways involved in the disease.

7 completion of study

The study is expected to continue until December 31, 2025, or until the treatment is no longer beneficial.

Upon completion, a final assessment is conducted to evaluate the overall response to the treatment.

Who Can Join the Study?

Diagnosis of RDEB (Recessive Dystrophic Epidermolysis Bullosa) confirmed by genetic testing or a skin biopsy with a special test called immunofluorescence mapping (IFM).
Age between 18 and 79 years.
Diagnosis of unresectable (cannot be removed by surgery), locally advanced, or metastatic SCC (Squamous Cell Carcinoma) confirmed before the screening visit.
Has not responded to standard treatments for RDEB SCC, such as surgery, radiotherapy, or traditional chemotherapy drugs like cisplatin, carboplatin, 5-fluorouracil, bleomycin, methotrexate, adriamycin, taxanes, gemcitabine, or ifosfamide, either alone or in combination. Also includes failure to respond to newer treatments like epidermal growth factor inhibitors (e.g., cetuximab, panitumumab) or immune checkpoint inhibitors (e.g., nivolumab, pembrolizumab, cemiplimab).
Is not currently receiving any other cancer treatment.
Has a measurable disease based on a specific set of guidelines called RECIST 1.1 (Response Criteria in Solid Tumors Version 1.1).
The patient or their legally authorized representative must have signed a consent form indicating that they understand the purpose and procedures of the study and are willing to participate.

Who Cannot Join the Study?

Patients who have not been diagnosed with Recessive Dystrophic Epidermolysis Bullosa (a rare genetic skin disorder) associated with Locally Advanced/Metastatic Squamous Cell Carcinoma (a type of skin cancer that has spread).
Patients who are not within the age range specified for the study.
Patients who are not able to follow the study procedures or take the study medication as required.
Patients who have other medical conditions that might interfere with the study or pose a risk to their health.
Patients who are pregnant or breastfeeding.
Patients who are participating in another clinical trial at the same time.
Patients who have had a recent major surgery or are planning to have surgery during the study period.
Patients who have a history of allergic reactions to the study medication or similar drugs.
Patients who have not recovered from the side effects of previous treatments.
Patients who have certain infections or diseases that could affect the study results.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Uniklinikum Salzburg	Salzburg	Austria

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	12.04.2021

Trial locations

Investigated drugs:

Rigosertib Sodium

Rigosertib is a medication being studied for its potential to treat a type of skin cancer called squamous cell carcinoma, which can occur in patients with a rare skin condition known as recessive dystrophic epidermolysis bullosa. This medication is being tested to see if it can help shrink the cancer or stop it from growing. Rigosertib can be taken by mouth or given through an infusion, which is a way of delivering the medication directly into the bloodstream over a period of time. The study aims to find out how safe Rigosertib is for patients and how well it works in treating their cancer.

Investigated diseases:

Epidermolysis bullosa

Recessive Dystrophic Epidermolysis Bullosa-associated Locally Advanced/Metastatic Squamous Cell Carcinoma – This condition involves a rare genetic disorder known as Recessive Dystrophic Epidermolysis Bullosa (RDEB), which causes fragile skin that easily blisters and tears. Over time, individuals with RDEB are at increased risk of developing aggressive skin cancers, such as Squamous Cell Carcinoma (SCC). In this context, SCC can become locally advanced or spread to other parts of the body, becoming metastatic. The progression of SCC in RDEB patients is often more rapid compared to the general population. The disease is characterized by the uncontrolled growth of abnormal cells in the skin, leading to tumor formation. As the cancer advances, it can invade deeper tissues and potentially spread to other organs.

Trial ID:

2024-518846-25-00

NCT ID:

NCT03786237

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on Rigosertib Sodium for Patients with Recessive Dystrophic Epidermolysis Bullosa and Advanced Squamous Cell Carcinoma

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