Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients with Recessive Dystrophic Epidermolysis bullosa associated Locally Advanced/Metastatic Squamous Cell Carcinoma

This research study is looking into a new medicine called Rigosertib for people aged 18 and over who have a type of skin cancer called squamous cell carcinoma. This cancer is specifically linked to a rare skin condition called Recessive Dystrophic Epidermolysis Bullosa (RDEB), where the skin is very fragile. The cancer in this study has either spread nearby or to other parts of the body. The main goal of the study is to see if Rigosertib can make the cancer shrink or stop growing. The researchers also want to find out if the medicine is safe and easy for people to take. They will be giving Rigosertib either as capsules to swallow or through a drip into a vein.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Eb Haus Austria Gemeinnuetzige Salzburger Landeskliniken Betriebsgesellschaft mbH

Enrolment target

Start

10 Dec 2024

Recessive Dystrophic Epidermolysis bullosa associated Locally Advanced/Metastatic Squamous Cell Carcinoma

What is this study about?

This study is a Phase II clinical trial, which means it's one of the earlier stages of testing a new medicine in people. The medicine being tested is called Rigosertib, and it's for adults who have a specific, more advanced type of skin cancer. This skin cancer, called squamous cell carcinoma, is related to a very rare and serious genetic skin condition called Recessive Dystrophic Epidermolysis Bullosa, often shortened to RDEB. People with RDEB have extremely fragile skin that blisters and scars easily, and unfortunately, they have a higher risk of developing this type of skin cancer.

The main purpose of this study is to see how well Rigosertib works against this specific type of cancer. Doctors want to know if it can cause the cancer to shrink or stop it from growing. They will be carefully measuring the size of the cancer over a period of time, up to about a year, using scans like CT or MRI. They also want to understand if the medicine is safe to use and what side effects people might experience.

Participants will receive Rigosertib either as capsules they take by mouth, or through a drip into a vein. The schedule for taking the medicine will vary depending on whether it's taken by mouth or through a drip. The study will also look at how the medicine affects a person's quality of life and will examine tissue samples to understand more about how the medicine works at a cellular level.

Key takeaways

This study tests a new medicine, Rigosertib, for a type of skin cancer linked to RDEB.
It aims to see if the medicine can shrink the cancer and if it's safe to use.
Rigosertib can be given as capsules or through a drip.
The study lasts up to a year with regular check-ups and scans.
Your quality of life will also be monitored throughout the study.

Who may be eligible?

To be part of this study, you need to be an adult, aged 18 or older.

You must have a specific type of skin cancer, called squamous cell carcinoma, which is linked to having Recessive Dystrophic Epidermolysis Bullosa (RDEB). This cancer needs to be locally advanced (meaning it has grown into nearby tissues) or metastatic (meaning it has spread to other parts of your body).

There will be other detailed medical checks to make sure the treatment is safe for you and that you will benefit most from participating. These checks will be explained fully by the study team.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have squamous cell carcinoma of the skin related to RDEB?
Has your cancer spread locally or to other parts of your body?
Are you able to travel to the study site for regular appointments?
Are you generally well enough to take part in a clinical trial?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be given the medicine Rigosertib. This can be either as capsules you swallow (or dissolved in liquid), or through a drip into your arm (intravenous, or IV). If you take capsules, you'll take them daily for three weeks, then have one week off, and this cycle will repeat. If you receive it through a drip, it will be a continuous slow infusion for 72 hours (3 days) at the start of a two-week cycle for a few months, and then on the first three days of a four-week cycle afterwards.

You will have regular check-ups, scans (like CT or MRI) to see how the cancer is responding, and blood tests. Doctors will also ask you about your general well-being and how you're feeling, including your quality of life. The study will last for up to 52 weeks (about a year) where your cancer will be regularly assessed. After this, you will still be monitored for safety.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. The potential benefit of this study is that Rigosertib might help to shrink or control your specific type of skin cancer. However, it's really important to remember that this is a new medicine, and we don't know for sure if it will work for everyone, or even if it will work effectively for anyone. You might also experience side effects, which the study team will monitor closely and explain in detail. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Austria

Common questions

What is Rigosertib?

Rigosertib is the new medicine being tested in this study. It's designed to target certain pathways that help cancer cells grow.

What does 'Phase II' mean?

Phase II means this is an early stage of testing where doctors are looking to see if the medicine works and is safe in a small group of people with the specific condition.

What is Recessive Dystrophic Epidermolysis Bullosa (RDEB)?

RDEB is a rare genetic skin condition that makes the skin very fragile, prone to blistering and scarring. People with RDEB have a higher risk of developing certain skin cancers.

How will doctors know if the medicine is working?

Doctors will use scans like CT or MRI to measure the size of your cancer over time and see if it is shrinking or stable.

Will I have to pay to be in the study?

No, you will not have to pay for the study medicine or study-related tests and appointments. However, you should discuss any potential travel costs or other expenses with the study team.