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Evaluating Diacerein Ointment for Treating Generalized Epidermolysis Bullosa Simplex

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) [EBShield Study]

Phase2; Phase3 Interventional TWi Biotechnology, Inc. · NCT06073132

This study is testing a new ointment to see if it can help people with severe or intermediate generalized epidermolysis bullosa simplex by reducing skin inflammation and blistering.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment80 (estimated)
Ages6 Months and up
SexAll
SponsorTWi Biotechnology, Inc. Industry-sponsored
Drugs / interventionschemotherapy
Locations35 sites (Rancho Santa Margarita, California and 34 other locations)
Trial IDNCT06073132 on ClinicalTrials.gov

What this trial studies

This international, multicenter trial aims to assess the efficacy and safety of diacerein 1% ointment in patients with generalized epidermolysis bullosa simplex (EBS). The study includes a randomized, double-blind phase followed by an open-label extension, focusing on patients with severe or intermediate EBS characterized by significant skin fragility and blistering. The treatment targets inflammation and keratin aggregation, which are key factors in the disease's pathogenesis. Participants will apply the ointment and report their experiences throughout the study.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 6 months and older with a confirmed genetic diagnosis of severe or intermediate EBS and significant skin lesions.

Not a fit: Patients with localized EBS or those without documented genetic mutations in KRT5 or KRT14 may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this treatment could significantly improve skin integrity and reduce blister formation in patients with generalized EBS.

How similar studies have performed: While there have been studies targeting inflammation in EBS, the specific use of diacerein 1% ointment represents a novel approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Patient is at least 6 months old at Visit 2 (Day 1/Baseline A). (Note: Enrollment of patients under 4 in the EU is pending approval of the safety review for 10 patients aged 4-12.)
2. Patients has been clinically diagnosed with severe EBS or intermediate EBS, confirmed by documented genetic diagnosis to have autosomal dominant mutations in KRT5 or KRT14 gene.
3. Patient with ≥ 3% BSA of EBS lesions excluding palms and soles at Visit 2 (Day 1/Baseline A).
4. Patient's EBS lesions within the Treatment Area have an IGA score of ≥3 at Visit 2 (Day 1/Baseline A).
5. Patient/caregiver agrees to follow study medication application instructions.
6. Patient (and caregiver/legal guardian) agrees to report use of all prescription and over-the-counter medications, including topical therapies applied to the body, e.g., medical cleansers, bleach cleansers, bleach baths, topical antiseptics, topical disinfectants, etc. for the duration of the study.
7. Patient (and caregiver/legal guardian) is willing and able to comply with all study visits and all the protocol requirements, including completing questionnaires.
8. Patient (and caregiver/legal guardian) is able to provide written informed consent; assent based on age.
9. Female patient of childbearing potential must have a negative pregnancy test prior to randomization.
10. Female patient of childbearing potential is willing to practice highly effective contraception (i.e., pregnancy prevention method with a failure rate of \< 1% per year) from Screening throughout the end of the study.

Exclusion Criteria:

1. Patient has a clinically significant skin disease other than EBS (e.g., psoriasis, atopic dermatitis, eczema, sun damage, etc.), or a vascular disorder associated with cutaneous erosions/ulcerations, that may confound assessments of efficacy or safety.
2. Patient has a clinically significant underlying medical condition, psychiatric condition (such as major depressive or psychotic disorder, severe intellectual disability, or alcohol or drug use disorder), or requires concomitant medication that based on the investigator's judgement may impair evaluation of the Treatment Area or exposes the patient to an unacceptable risk by study participation.
3. Patient has used any diacerein-containing product within 6 months prior to Visit 2 (Day 1/Baseline A).
4. Patient has had a cutaneous infection in the Treatment Area or use systemic antibiotics within 7 days prior to Visit 2 (Day 1/Baseline A).
5. Patient has uncontrolled diabetes mellitus (HbA1c ≥ 6.5%), hepatic enzyme abnormalities (alanine aminotransferase or aspartate aminotransferase \>2.5 the upper limit of normal (ULN), or total bilirubin \>2.0x ULN), or renal abnormalities (estimated glomerular filtration rate \[eGFR\]\< 30 ml/min/1.73 m2) during the Screening period.
6. Patient has a current malignancy, or a history of treatment for a malignancy within 5 years (with the exception of treated non-melanoma cutaneous malignancy e.g., surgically resected with clear margins) prior to Visit 2 (Day 1/Baseline A).
7. Patient is treated with protocol-excluded topical therapies other than steroids within 2 weeks prior to Visit 2 (Day 1/Baseline A) that might influence the assessment of the Treatment Area throughout the study period.
8. Patient has been treated with topical steroids on the EBS lesions within 2 weeks or systemic steroids within 4 weeks. prior to Visit 2 (Day 1/Baseline A). (Note: inhaled and ophthalmic products containing steroids are allowed.)
9. Patient has been treated with: (a) an approved biologic anti-inflammatory therapy (such as monoclonal antibodies that target to modulate the immune responses) and (b) other immunosuppressive/immunomodulatory therapies or chemotherapy within 8 weeks prior to Visit 2 (Day 1/Baseline A).
10. Patient has been treated with any investigational drug or device within 30 days or 5 half-lives, whichever is longer, prior to Visit 2 (Day 1/Baseline A).
11. Patient has a history of allergy or hypersensitivity to any component of study medications, including diacerein or rhein.
12. Patient is pregnant or breastfeeding/lactating.
13. Patient has a planned or anticipated major surgical procedure or other activity that would interfere with their ability to comply with protocol requirements.

Where this trial is running

Rancho Santa Margarita, California and 34 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Generalized Epidermolysis Bullosa SimplexEpidermolysis Bullosa SimplexEBS
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.