Active not recruitingPHASE3INTERVENTIONAL

Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over)

This research study is looking at a new treatment called allo-APZ2-OTS for people with a rare genetic skin condition called Epidermolysis Bullosa (EB). Specifically, we are focusing on two types: recessive dystrophic EB (RDEB) and Junctional EB (JEB). The main goal is to see if this new cell therapy, given into the bloodstream, is safe and effective in helping the skin of those with RDEB, compared to a placebo. We will also study its effects in very young children with RDEB (under 1 year old) and in people with JEB. This is a larger-scale study (Phase 3) hoping to find better ways to manage this challenging condition.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

RHEACELL GmbH & Co. KG

Enrolment target

Start

13 Jun 2023

Estimated completion

01 Jan 2027

Epidermolysis Bullosa

What is this study about?

This study is investigating a new treatment called allo-APZ2-OTS for a group of rare genetic skin conditions known as Epidermolysis Bullosa (EB). People with EB have very fragile skin that easily blisters and tears, both from minor injury and sometimes even spontaneously. This can lead to chronic wounds and significant pain. The current treatments mainly focus on managing symptoms, so new and more effective therapies are greatly needed.

Allo-APZ2-OTS is a type of cell therapy, meaning it uses specially prepared cells to try and help the body heal. In this study, we are particularly interested in whether these cells can improve the condition of the skin in people with recessive dystrophic EB (RDEB) and Junctional EB (JEB). We want to find out if it's safe to give this treatment into the bloodstream and if it can help wounds heal better and reduce blistering.

The study involves comparing allo-APZ2-OTS against a 'placebo', which looks identical to the actual treatment but contains no active ingredients. This helps us objectively see if any improvements are genuinely due to the new treatment rather than other factors. There's also a part of the study for very young children with RDEB (under one year old) and for people with JEB, where everyone will receive the active treatment. This research is important because it could potentially lead to a new treatment option for people living with EB, improving their quality of life.

Key takeaways

This study is testing a new cell therapy for Epidermolysis Bullosa (EB).
It aims to see if the treatment is safe and can help improve skin and wound healing.
Some participants will receive the active treatment, others a placebo (dummy treatment), for comparison.
There's a special part of the study for very young children with RDEB and for those with JEB.
Participation involves regular clinic visits for checks, treatment, and follow-up.
You have the right to leave the study at any time.

Who may be eligible?

To join this study, you need a confirmed diagnosis of recessive dystrophic Epidermolysis Bullosa (RDEB) or Junctional Epidermolysis Bullosa (JEB), verified by genetic tests or a special skin biopsy. You also need to be at least 5 kg (about 11 pounds) in weight and generally healthy enough to take part. We'll be looking at a specific wound on your body that's between 5 and 50 square centimetres in size, is less than 9 months old, and doesn't show signs of infection.

There are also some reasons why you wouldn't be able to join. These include having any skin cancers or pre-cancerous spots, chronic lung conditions, or a history of blood clots. We also can't include you if you've had certain serious immune reactions in the past, or if you have allergies to any of the study medications. If you're pregnant or breastfeeding, or if you've recently taken part in another clinical trial, you won't be able to join this one.

All participants, or their legal guardians, must understand the study and be able to provide written permission. If you are a woman who could become pregnant, you'll need a negative pregnancy test at the start and agree to use effective contraception throughout the study. Male participants whose partners could become pregnant will also need to use effective contraception.

Quick self-check

Do you have a confirmed diagnosis of RDEB or JEB?
Are you at least 5 kg (about 11 lbs) in weight?
Do you have a target wound that is not infected, is between 5-50 cm², and less than 9 months old?
Are you generally in good health?
Are you pregnant or breastfeeding?
Do you have a history of skin cancer or chronic lung disease?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, you'll have regular visits to the clinic. At these visits, doctors and nurses will check your overall health, examine your skin and wounds, and take blood and urine samples. You might also have further tests depending on what the study doctors need to monitor. The study treatment (either the active cell therapy or the placebo) will be given directly into your bloodstream through a drip.

Because this study has a 'cross-over' design for some people, it means that if you initially receive the placebo, you might later have the opportunity to receive the active treatment. You will also have follow-up appointments after your treatment to see how you are doing and track any changes. The total duration of your involvement in the study, including all treatments and follow-up, will be explained in detail by the study team.

Potential risks and benefits

There is a potential for benefit if the new treatment proves to be effective in improving your skin condition and wound healing. However, as with any new treatment, there are also potential risks and side effects, some of which may not yet be known. The study team will explain all known risks to you before you agree to take part. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (16)

Sanatorio Mater Dei
Buenos Aires, Argentina
EB-Haus Austria; Salzburger Landeskliniken (SALK)
Salzburg, Austria
Fundacion Debra Chile
Santiago, Chile
Universitaetsklinik fuer Dermatologie und Allergologie
Nice, France
Hospital Necker-Enfants Malades
Paris, France
Service de Dermatologie Hospital Saint-Louis
Paris, France
Andreas Syggros Hospital of Cutaneous Venereal Diseases
Athens, Greece
Venereal & Skin Diseases Hospital of Thessaloniki
Thessaloniki, Greece
Semmelweis Egyetem
Budapest, Hungary
UOSD Pediatria-Alta Intensita di Curà, Ospedale Maggiore
Milan, Italy
Ospedale Pediatrico Bambin Gesu
Roma, Italy
Medical Concierge Centrum Medyczne
Warsaw, Poland

+4 more sites — see the official record for the full list.

Common questions

What is Epidermolysis Bullosa (EB)?

EB is a group of rare genetic skin conditions where the skin is very fragile and blisters easily, often from minor bumps or friction.

What is a 'cell therapy'?

A cell therapy uses special cells to help repair or replace damaged parts of the body, which in this case, means trying to improve skin healing.

What is a 'placebo'?

A placebo is a dummy treatment that looks just like the real treatment but doesn't contain any active medicine. It helps us compare the new treatment's effects.

Will I know if I'm getting the active treatment or a placebo?

For part of the study, it's 'blinded,' meaning neither you nor the study team will know who is getting which treatment until a later stage. This helps keep the results fair.

Can I stop participating in the study at any time?

Yes, you are free to leave the study at any point, and this will not affect your routine medical care.