RecruitingPHASE1, PHASE2INTERVENTIONAL

GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment

This study is exploring a new way to treat Recessive Dystrophic Epidermolysis Bullosa (RDEB), a condition where the skin is very fragile and easily forms painful blisters. The treatment is a special skin graft called GMEB-SASS. This graft is made from a small sample of the patient's own skin cells. In the lab, these cells are given new instructions to produce a vital protein missing in RDEB, which helps skin stick together. Once modified, these cells are grown into a usable skin graft. The aim is for this permanent graft to replace damaged skin and improve wound healing. This trial will see if this new approach is safe and effective for patients, both adults and children, with RDEB.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

CHU de Quebec-Universite Laval

Enrolment target

Start

07 Jan 2026

Estimated completion

01 Dec 2035

RDEB Recessive Dystrophic Epidermolysis Bullosa Epidermolysis Bullosa Dystrophica, Recessive

What is this study about?

This study is about a new treatment for a rare skin condition called Recessive Dystrophic Epidermolysis Bullosa, or RDEB. People with RDEB have incredibly fragile skin that easily blisters and breaks, leading to painful, long-lasting wounds. This happens because their bodies don't make a protein called type VII collagen, which acts like glue to hold different layers of skin together.

The new treatment being tested is a special type of skin graft named GMEB-SASS. This graft is made uniquely for each patient using a tiny sample of their own skin. In a lab, scientists take the patient's skin cells and, using a clever technique, teach them how to make the missing type VII collagen protein. These 're-educated' cells are then grown into new skin patches. When these patches are put onto a wound, the hope is that they will become a permanent part of the patient's skin, helping to heal the wounds and stop new ones from forming.

This study is the first time this specific GMEB-SASS graft is being used in people. It's designed in two stages: first, they will carefully test it in a small number of adults to make sure it's safe. Once they have a better understanding of its safety, they will then slowly include younger patients, including teenagers and children. The main goal is to find out if this specially grown skin can safely and effectively improve wound healing and strengthen the skin in people with RDEB.

Key takeaways

This study tests a new special skin graft for RDEB.
The graft uses your own modified skin cells to make missing protein.
It aims to permanently improve wound healing and skin strength.
Safety will be checked carefully, starting with adults, then including children.
Participation involves minor surgeries and regular check-ups.

Who may be eligible?

This study is for people who have been diagnosed with Recessive Dystrophic Epidermolysis Bullosa (RDEB) and have a specific gene fault in the COL7A1 gene. To join, you (or your parents/carers if you're a child) must be able to understand what the study involves and agree to follow the study plan. You also need to have wounds on your body or limbs that are large enough for the special skin grafts.

The study will start with adults. First, people aged 18 and over will be included. Then, the study will expand to include those aged 12 and over, and finally, children aged 7 and over. Everyone taking part must be well enough to travel to the study center and have surgery under anesthetic.

There are certain reasons why someone might not be able to join. These include being generally unwell, having serious infections, a history of certain skin cancers at the wound site, or being pregnant or breastfeeding. You also can't have had certain strong medications like chemotherapy or high doses of steroids recently, as these could affect the study results or your safety.

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.

Do I have a confirmed diagnosis of RDEB with a specific gene change?
Am I (or my child) aged 7 or older?
Do I have wounds on my body or limbs that need grafting?
Am I generally well enough to have a minor operation and travel for visits?
Am I pregnant or breastfeeding (if female)? (Exclusion)

Answer every question to see your result.

What does participation involve?

Taking part in this study involves several steps. First, doctors will take a small skin sample from you. This sample will be sent to a lab where your cells will be used to grow the special GMEB-SASS skin grafts. Before the main graft, you'll have a minor operation where a temporary skin graft (from a donor) might be used to prepare the wound area. A few days later, you'll have another operation where the GMEB-SASS grafts will be carefully applied to your chosen wounds.

After the grafts, you will need to attend regular follow-up visits with the study team. These visits will check how your wounds are healing, look for any side effects, and make sure the grafts are settling in well. The maximum amount of skin grafted at one time will cover no more than 9% of your body surface, which helps manage risk.

The study will involve careful monitoring for an extended period to understand the long-term effects of the grafts. The exact number of visits and the total duration aren't fully detailed here, but expect a commitment to regular assessments and monitoring over time.

Potential risks and benefits

As this is a new treatment, there are potential benefits and potential risks. The main potential benefit is improved wound healing and stronger skin, which could significantly improve the quality of life for people with RDEB. However, there are also risks, like with any surgical procedure or new medication. These could include issues with the graft taking, infection, or unexpected side effects. The study is carefully designed to monitor these closely, starting with adults to gather safety information before including children. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

The Hospital for Sick Children (SickKids)
Verified postcode
Toronto, Canada· Recruiting

Common questions

What is RDEB?

RDEB is a rare genetic skin condition where the skin is very fragile, easily blisters, and forms wounds because a key protein is missing.

What is GMEB-SASS made of?

It's a special skin graft made from your own skin cells, which are modified in a lab to produce the missing protein that makes skin stronger.

Will this be a permanent change?

The GMEB-SASS graft is designed to be permanent, aiming to replace damaged skin with healthy, stronger skin for the long term.

Will children be in this study?

Yes, children from age 7 will be included, but only after some safety information is gathered from adult participants first.

Is GMEB-SASS a cure for RDEB?

This study is testing if GMEB-SASS can help heal wounds and improve skin strength. It's an important step in finding better treatments, but it's not described as a cure in this information.

How to find out more

Lucie Germain, PhD

lucie.germain@fmed.ulaval.ca 418-525-4444 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.