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)

Running, not enrolling · Phase 2/Phase 3

Conditions studied: Generalized Epidermolysis Bullosa Simplex

In brief

The proposed Phase 2/3 trial with double-blind and open-label extension phases is an international, multicenter study designed to assess the efficacy and safety of diacerein 1% ointment in patients with generalized EBS.

Key facts

Study ID: NCT06073132
Run by: TWi Biotechnology, Inc.
People needed: 80
Starts: 2024-04-04
Expected to finish: 2027-03-01
Last updated by the study team: 2026-07-09

Who can join

Age: 1 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Patient is at least 6 months old at Visit 2 (Day 1/Baseline A).
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.
Patient with ≥ 3% BSA of EBS lesions excluding palms and soles at Visit 2 (Day 1/Baseline A).
Patient's EBS lesions within the Treatment Area have an IGA score of ≥3 at Visit 2 (Day 1/Baseline A).
Patient/caregiver agrees to follow study medication application instructions.
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.
Patient (and caregiver/legal guardian) is willing and able to comply with all study visits and all the protocol requirements, including completing questionnaires.
Patient (and caregiver/legal guardian) is able to provide written informed consent; assent based on age.
Female patient of childbearing potential must have a negative pregnancy test prior to randomization.
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.

You may not qualify if…

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.
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.
Patient has used any diacerein-containing product within 6 months prior to Visit 2 (Day 1/Baseline A).
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).
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.
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).
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.
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.)
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).
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).
Patient has a history of allergy or hypersensitivity to any component of study medications, including diacerein or rhein.
Patient is pregnant or breastfeeding/lactating.
Patient has a planned or anticipated major surgical procedure or other activity that would interfere with their ability to comply with protocol requirements.

Where it is running

Mission dermatology Center — Rancho Santa Margarita, California, United States
Stanford University — Stanford, California, United States
Children's Hospital Colorado — Aurora, Colorado, United States
Northwestern University - Lurie Childrens's Hospital — Chicago, Illinois, United States
Cincinnati Childrens Hospital — Cincinnati, Ohio, United States
Medical University of South Carolina — Charleston, South Carolina, United States
Texas Dermatology and Laser Specialists — San Antonio, Texas, 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, Guangdong, China
Hospital of Skin and Venereal Diseases of Thessaloniki — Thessaloniki, Greece
Postgraduate Institute of Medical Education and Research (PGIMER) — Chandigarh, India
Children's Health Ireland — Dublin, Ireland
Tel Aviv Sourasky Medical Center — Tel Aviv, Israel
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milano — Milan, Italy
Università degli Studi di Modena e Reggio Emilia (UNIMORE) — Modena, Italy
Istituto Dermopatico dell'Immacolata (IDI) - Istituto di — Rome, Italy
UOS "Centro delle Dermatosi Croniche Complesse e Genodermatosi" UOC Dermatologia — Rome, Italy
Hospital Tunku Azizah (Hospital Wanita Dan Kanak-kanak Kuala Lumpur) — Kuala Lumpur, Malaysia
Asian Hospital — City of Muntinlupa, Philippines
Iloilo Doctors Hospital — Iloilo City, Philippines
Health Cube Medical Clinics — Mandaluyong, Philippines
OT.CO Clinic Osipowicz & Turkowski — Warsaw, Poland

Full record on ClinicalTrials.gov

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