Active not recruitingEARLY_PHASE1INTERVENTIONAL

Self-Assembled Skin Substitute for the Treatment of Epidermolysis Bullosa

This early-stage study, known as an 'early phase 1' trial, was conducted with just one patient. The patient has a severe skin condition called dystrophic epidermolysis bullosa, which causes fragile skin and chronic, open wounds that haven't healed with standard treatments like special dressings, creams, and medicines. The study aimed to test a new type of skin patch, called a Self-Assembled Skin Substitute (SASS). Researchers wanted to see if applying this SASS to the patient's stubborn wounds could help them to close and get better. This was a very early step to understand if this new treatment might be helpful for people with this condition in the future.

At a glance

Status

Active not recruiting

Phase

EARLY_PHASE1

Sponsor

CHU de Quebec-Universite Laval

Enrolment target

Start

10 Dec 2019

Estimated completion

18 Dec 2026

Epidermolysis Bullosa Dystrophica

What is this study about?

This clinical trial is about a very specific and serious skin condition called dystrophic epidermolysis bullosa (EB). People with this condition have extremely fragile skin that easily blisters and tears, leading to long-lasting open wounds. These wounds can be very painful and difficult to heal, even with regular medical care like special dressings, antiseptic creams, antibiotics, and other medicines.

This particular study was designed for just one patient who has these severe, chronic wounds that haven't responded to any of the usual treatments. The main purpose of the study was to try a new approach called a Self-Assembled Skin Substitute, or SASS for short. Think of SASS as a specially made skin patch designed to help these difficult wounds heal more effectively.

The researchers wanted to see if applying these SASS patches directly to the patient's problem wounds could encourage them to close up and improve. This is an 'early phase 1' study, which means it's one of the very first times this treatment is being tested in people. The goal is to learn if it's safe and if it shows any signs of working for this challenging condition.

Key takeaways

This was a very early study (Phase 1) involving only one patient.
It tested a new skin patch (SASS) for severe wounds in Epidermolysis Bullosa.
The patient's wounds were chronic and hadn't responded to other treatments.
The goal was to see if SASS could help these difficult wounds heal.
This kind of study helps researchers learn if new treatments might be safe and effective.

Who may be eligible?

This study was specifically designed for one individual patient. Therefore, it was not open to the general public.

The patient selected had a confirmed diagnosis of dystrophic epidermolysis bullosa. Crucially, they also had chronic, open wounds that had not been healing despite receiving various other treatments like special dressings, creams, and medicines through a vein or by mouth, often used to fight infection or reduce inflammation. They needed to have these very stubborn wounds that weren't getting better with standard care.

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.

You have a difficult skin condition called dystrophic epidermolysis bullosa.
You have long-lasting open wounds that haven't healed with dressings or creams.
Your wounds haven't improved with other medicines, such as antibiotics.
You are the specific patient for whom this custom study was designed.

Answer every question to see your result.

What does participation involve?

This study was uniquely designed for a single patient. The details of participation, including specific visits, assessments, how the SASS was applied, and follow-up, would have been tailored exactly to their needs. Generally, taking part in a study like this would involve regular hospital visits for the SASS application and close monitoring of the wounds. Assessments would likely include photographs, measurements of the wound size, and checks for any changes or side effects. The total duration of the patient’s participation would depend on how long it took for the wounds to heal or if the treatment was stopped for any reason.

Potential risks and benefits

In a study like this, the potential benefit is that the new skin substitute might help to heal severe wounds that haven't responded to other treatments, potentially reducing pain and improving quality of life. However, like any new treatment, there are potential risks, which could include the SASS not working, causing infection, or having other unexpected side effects. The participant would have had the right to withdraw from the study at any time without affecting their usual medical care.

Locations (1)

The Hospital for Sick Children
Verified postcode
Toronto, Canada

Common questions

What is Epidermolysis Bullosa Dystrophica?

It's a rare genetic skin condition that makes skin very fragile, leading to painful blisters and open wounds that struggle to heal.

What is a 'Self-Assembled Skin Substitute' (SASS)?

It's a special type of skin patch or material designed to cover and help severe wounds heal, especially when other treatments haven't worked.

Was this study for many people?

No, this was a very early-stage study specifically designed for just one patient to test this new treatment for the first time.

What does 'early phase 1' mean?

It means it's one of the very first steps in testing a new treatment in people, mainly to check if it's safe and to see if it shows any early signs of working.

Did this study involve children or adults?

The study was open to anyone, regardless of age, as long as they met the very specific criteria for this one-patient trial.