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Study on Diacerein 1% Ointment for Treating Generalized Epidermolysis Bullosa Simplex in Patients Aged 6 Months and Older

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a skin condition called Generalized Epidermolysis Bullosa Simplex (EBS). EBS is a rare genetic disorder that causes the skin to be very fragile, leading to blisters and wounds from minor injuries or friction. The study is testing a treatment called Diacerein 1% Ointment, which is applied directly to the skin. Diacerein is a chemical substance that may help reduce inflammation and improve skin healing in people with EBS.

The purpose of the study is to evaluate how effective and safe the Diacerein 1% Ointment is for treating EBS. Participants in the study will be randomly assigned to receive either the Diacerein ointment or a vehicle ointment, which is a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. The study will last for several weeks, during which participants will apply the ointment to their skin and attend regular check-ups to monitor their progress and any side effects.

Throughout the study, participants will be asked to report any changes in their symptoms, such as pain or itching, and any other health issues they experience. The study aims to determine if the Diacerein 1% Ointment can successfully reduce the severity of EBS symptoms and improve the quality of life for those affected by this condition. Participants will also have the opportunity to continue using the ointment in an open-label extension phase, where everyone will receive the actual treatment, to further assess its long-term benefits and safety.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed. This includes being at least 6 months old, having a clinical diagnosis of severe or intermediate generalized epidermolysis bullosa simplex (EBS), and meeting other specific criteria.

    A negative pregnancy test is required for female patients of childbearing potential before starting the trial.

  2. Step 2

    Baseline assessment

    At the baseline visit, the extent of EBS lesions is assessed. This includes measuring the percentage of body surface area (BSA) affected and evaluating the severity of lesions using an Investigator's Global Assessment (IGA) score.

  3. Step 3

    Treatment phase

    The treatment involves applying diacerein 1% ointment or a placebo ointment to the affected areas. The ointment is applied topically, meaning it is applied directly to the skin.

    The treatment lasts for 8 weeks, with the ointment applied as instructed by the study protocol.

  4. Step 4

    Follow-up visits

    During the 8-week treatment period, regular follow-up visits are scheduled to monitor progress and any side effects.

    These visits include assessments of lesion improvement, changes in pain and itchiness, and overall quality of life.

  5. Step 5

    End of treatment assessment

    At the end of the 8-week treatment period, a final assessment is conducted. This includes evaluating the success of the treatment based on the IGA score and other measures.

    The primary goal is to achieve a significant reduction in lesion severity and improvement in symptoms.

  6. Step 6

    Safety monitoring

    Throughout the study, safety is closely monitored. This includes tracking any adverse events, changes in laboratory results, and vital signs.

    The study aims to ensure that the treatment is both effective and safe for participants.

Who can join the trial?

10 criteria

  • The patient must be at least 6 months old at the start of the study.
  • If the patient is a female who can have children, she must agree to use a very reliable method to prevent pregnancy during the study.
  • The patient must have been diagnosed with severe or intermediate **Epidermolysis Bullosa Simplex (EBS)**, confirmed by a genetic test showing specific changes in the **KRT5** or **KRT14** genes.
  • The patient must have EBS lesions covering at least 5% of their body, not including the hands and feet, at the start of the study.
  • The EBS lesions in the treatment area must have a severity score of 3 or higher at the start of the study.
  • The patient or their caregiver must agree to follow the instructions for applying the study medication.
  • The patient and their caregiver or legal guardian must agree to report all medications they use, including those applied to the skin, like medical cleansers or antiseptics, during the study.
  • The patient and their caregiver or legal guardian must be willing and able to attend all study visits and follow all study requirements, including filling out questionnaires.
  • The patient and their caregiver or legal guardian must be able to provide written consent to participate in the study. If the patient is a child, they may need to give their agreement based on their age.
  • If the patient is a female who can have children, she must have a negative pregnancy test before being randomly assigned to a treatment group in the study.

Who cannot join the trial?

8 criteria

  • Patients who have a different type of skin condition other than Generalized Epidermolysis Bullosa Simplex (EBS) cannot participate.
  • Patients who are younger than 6 months old are not eligible.
  • Patients who have any other serious health issues that might interfere with the study cannot join.
  • Patients who are currently using other treatments that might affect the study results are excluded.
  • Patients who are unable to follow the study procedures or instructions cannot participate.
  • Patients who have allergies to the study medication or its ingredients are not eligible.
  • Patients who are pregnant or breastfeeding cannot take part in the study.
  • Patients who have participated in another clinical trial recently may be excluded.
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Investigated drugs

Diacerein 1% Ointment is being tested in this clinical trial. It is an ointment applied to the skin, specifically designed to help treat a condition called generalized epidermolysis bullosa simplex (EBS). This condition causes the skin to be very fragile and to blister easily. The ointment is being studied to see if it can effectively reduce the symptoms of EBS, such as blistering and skin irritation, over an 8-week treatment period. The trial aims to determine if this ointment can provide relief and improve the quality of life for both adults and children aged 6 months and older who suffer from this condition.

What is already known about the treatment

Diacerein 1% Ointment – This medication is applied topically as an ointment and is currently being studied in clinical trials for its effectiveness in treating Generalized Epidermolysis Bullosa Simplex (EBS). It is in the Phase 2/3 stage of clinical trials, indicating it is being evaluated for both efficacy and safety. The main therapeutic indication is for the treatment of EBS, a skin condition characterized by blistering. Diacerein works by inhibiting the production of certain inflammatory substances in the body, which may help reduce skin inflammation and blistering. It is classified as an anti-inflammatory agent.

Investigated diseases

Generalized Epidermolysis Bullosa Simplex (EBS) – This is a rare genetic skin disorder characterized by fragile skin that easily blisters and tears from minor friction or trauma. The condition primarily affects the outer layer of the skin, leading to painful blisters and erosions. It can vary in severity, with some individuals experiencing widespread blistering while others have more localized symptoms. The blisters typically heal without scarring, but repeated blistering can lead to thickened skin and other complications. EBS is caused by mutations in genes responsible for skin integrity, affecting the proteins that help bind skin layers together. The condition is usually present from birth or early childhood and can impact daily activities due to skin fragility.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2023-508818-42-00Protocol codeAC-203-EBS-007Estimated enrolment102 patientsSponsorTwi Biotechnology Inc.

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