Active not recruitingPHASE2, PHASE3INTERVENTIONAL

An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study With Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS)

This international study is looking into a new cream called AC-203 (which contains a medicine called diacerein 1%) for people with a skin condition called Epidermolysis Bullosa Simplex (EBS). EBS causes fragile skin and painful blisters. The cream is designed to help reduce inflammation and improve skin strength. Researchers want to find out how effective and safe this cream is for people, including young children, who have widespread EBS. Half of the participants will use the AC-203 cream, and the other half will use a cream that looks the same but doesn't have the active medicine (called a ‘vehicle’). This helps researchers compare the results fairly. The study also has an ‘open-label extension’ phase, meaning everyone will eventually get the active medicine if they choose to continue.

At a glance

Status

Active not recruiting

Phase

PHASE2, PHASE3

Sponsor

TWi Biotechnology, Inc.

Enrolment target

Start

04 Apr 2024

Estimated completion

01 Mar 2027

Generalized Epidermolysis Bullosa Simplex

What is this study about?

This study is about a skin condition called Epidermolysis Bullosa Simplex (EBS). If you or someone you know has EBS, you’ll know it makes your skin very fragile, leading to painful blisters and sores, sometimes all over the body. This happens because of a problem with certain proteins in the skin that act like a scaffold, holding it together. When these proteins aren't quite right, the skin breaks down easily.

Researchers have noticed that along with the skin fragility, there's often a lot of inflammation in people with EBS. This inflammation, which is the body's natural response to injury or irritation, seems to make the condition worse. That's why scientists are exploring whether tackling this inflammation could help improve EBS symptoms. They're also looking at ways to strengthen the skin's structure.

This study, therefore, is testing a new cream called AC-203. This cream contains a medicine named diacerein, which is known for its ability to calm down inflammation. It also appears to help correct the skin protein problems that cause the fragility in EBS. By using this cream, doctors hope to reduce blistering and improve the overall skin condition for people with widespread EBS. This research is important because it could lead to new ways to manage this challenging condition, offering more comfortable lives for those affected.

Key takeaways

Tests a new cream (AC-203) for widespread EBS.
Aims to reduce blisters and inflammation in fragile skin.
Involves a 'blinded' comparison to a dummy cream initially.
Includes children aged 6 months and older.
Could offer a new treatment option for EBS.
Regular check-ups and cream application required.

Who may be eligible?

To be part of this study, you or the person you care for must be at least 6 months old and have a confirmed diagnosis of severe or intermediate Epidermolysis Bullosa Simplex (EBS). This diagnosis needs to be backed up by a genetic test showing specific changes in certain genes (KRT5 or KRT14).

Your skin must show blistering or sores from EBS covering at least 3% of your body, not including your palms and soles. Also, the areas of skin chosen for treatment in the study must have a certain level of severity, as assessed by the study doctor. You or your caregiver must be willing to follow instructions for applying the cream, report all other medicines and creams you use, and attend all study appointments.

There are also some reasons why someone might not be able to join. For example, if you have other serious skin problems (like severe eczema or psoriasis) or other significant health conditions that might interfere with the study or make assessments difficult. If you are a female who could become pregnant, you would need to have a negative pregnancy test and agree to use effective birth control throughout the study.

Quick self-check

Are you or the person you care for at least 6 months old?
Do you have a genetics-confirmed diagnosis of severe or intermediate EBS?
Do you have active EBS blistering/sores on at least 3% of your body (excluding hands/feet)?
Are you able to attend all study visits and apply the cream as directed?
Are you willing to report all other medicines and creams you use?
If you are a female who can get pregnant, are you willing to use effective birth control?

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 be involved in a "double-blind" study. This means that for a while, neither you nor your study doctor will know if you're getting the active AC-203 cream or a 'vehicle' cream (which looks the same but doesn't contain the active medicine). This helps make sure the results are fair. You'll apply the assigned cream as instructed. Your skin and overall health will be regularly checked by the study team through clinic visits, assessments, and questionnaires. You'll also need to keep a record of all other medicines, creams, and treatments you use. After this initial period, there's an 'open-label extension' phase, which means if you continue, everyone will know they are receiving the AC-203 cream. The total duration of your participation, including follow-up, will be explained fully by the study team, but generally, these studies involve several visits over many months.

Potential risks and benefits

Participating in this study might offer the potential benefit of improving your EBS symptoms through a new treatment that targets inflammation and skin strength. However, as with any medical study, there are potential risks. The AC-203 cream might cause side effects, although the study aims to assess its safety. You might also experience discomfort from study procedures, or the cream might not work for you. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (30)

Mission dermatology Center
Rancho Santa Margarita, United States
Stanford University
Stanford, United States
Children's Hospital Colorado
Aurora, United States
Northwestern University - Lurie Childrens's Hospital
Chicago, United States
Cincinnati Childrens Hospital
Cincinnati, United States
Medical University of South Carolina
Charleston, United States
Texas Dermatology and Laser Specialists
San Antonio, United States
Premier Specialists
Kogarah, Australia
Sydney Children's Hospital
Randwick, Australia
Universitaetsklinik fuer Dermatologie und Allergologie
Salzburg, Austria
UZ Leuven
Leuven, Belgium
Dermatology Hospital of Southern Medical University
Guangzhou, China

+18 more sites — see the official record for the full list.

Common questions

What is Epidermolysis Bullosa Simplex (EBS)?

EBS is a genetic skin condition that makes your skin very delicate and prone to blistering, often with painful sores, because of a problem with skin-strengthening proteins.

What is AC-203 cream supposed to do?

AC-203 cream aims to reduce the inflammation (swelling and redness) that can make EBS worse, and it may also help strengthen the fragile skin structure.

Will I definitely get the new cream if I join?

Not at first. In the 'double-blind' part of the study, you'll either get the active AC-203 cream or a 'vehicle' cream (a dummy cream). After this, if you continue, everyone will get the active cream in the 'open-label' phase.

Who can take part in this study?

People aged 6 months and older with a confirmed diagnosis of severe or intermediate EBS and a certain amount of blistering on their body are generally eligible.

What does a 'double-blind' study mean?

It means for part of the study, neither you nor your doctor will know if you're receiving the active AC-203 cream or the dummy cream. This helps ensure unbiased results.