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)
NCT ID: NCT06073132
Last Updated: 2025-11-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2/PHASE3
80 participants
INTERVENTIONAL
2024-04-04
2027-01-31
Brief Summary
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Detailed Description
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Disruption of the keratin 5/14 filament network in basal keratinocytes is a key factor in EBS pathogenesis, compromising skin integrity. The severity of EBS is linked to the extent of keratin mutations disrupting this network, particularly resulting in keratin aggregates in severe cases. Recent studies suggest that mutated keratin proteins can trigger inflammation, exacerbating EBS. Elevated proinflammatory cytokines, like IL-1β and IL-6, are observed in EBS patients, and IFN-γ may mediate inflammation, promoting keratin aggregations. As a result, targeting inflammation is considered a potential therapeutic approach in EBS.
AC-203 (diacerein 1% ointment) is a topical formulation of diacerein, well-known for its ability to inhibit IL-1β and other proinflammatory cytokines. Moreover, diacerein and its active metabolite, rhein, have demonstrated ability in reducing keratin aggregates in keratinocytes derived from severe EBS. Taken together, with its anti-inflammatory property and ability to diminish keratin aggregation, AC-203 shows promise in reducing the clinical severity of EBS.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Part A AC-203
Double-blind, AC-203 Diacerein 1% ointment, QD
AC-203
The investigational product is formulated as 1% topical ointment
Part A Vehicle ointment
Double-blind, Vehicle ointment, QD
Vehicle
Vehicle-only control study medication is the same formulation as investigational product without active ingredient
Part B AC-203
Open-label extension phase, AC-203 Diacerein 1% ointment, QD
AC-203
The investigational product is formulated as 1% topical ointment
Interventions
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AC-203
The investigational product is formulated as 1% topical ointment
Vehicle
Vehicle-only control study medication is the same formulation as investigational product without active ingredient
Eligibility Criteria
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Inclusion Criteria
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
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.
6 Months
ALL
No
Sponsors
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TWi Biotechnology, Inc.
INDUSTRY
Responsible Party
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Locations
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Mission dermatology Center
Rancho Santa Margarita, California, United States
Stanford University
Stanford, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Pediatric Skin Research, LLC
Miami, Florida, United States
Northwestern University - Lurie Childrens's Hospital
Chicago, Illinois, United States
Stony Brook Dermatology
Stony Brook, New York, 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
Royal Melbourne Hospital
Melbourne, Victoria, Australia
Premier Specialists
Kogarah, , Australia
Sydney Children's Hospital
Randwick, , Australia
Universitaetsklinik fuer Dermatologie und Allergologie
Salzburg, , Austria
UZ Leuven
Leuven, , Belgium
Hopital Necker-Enfants Malades
Paris, , France
Andreas Syggros Hospital 1st University Clinic of Skin and Venereal Diseases of Athens
Athens, , Greece
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
IRCCS, AOUBO, Policlinico Sant'Orsola
Bologna, , Italy
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
Gangnam Severane Hospital
Seoul, , South Korea
Hospital Universitario La Paz
Madrid, , Spain
National Cheng Kung University Hospital
Tainan, , Taiwan
Sheikh Khalifa Medical City (SKMC)
Abu Dhabi, , United Arab Emirates
Great Ormond Street Hospital (GOSH) for Children NHS Foundation Trust - Somers Clinical Research Facility (CRF)
London, , United Kingdom
University Hospitals Birmingham NHS Foundation Trust (UHB)
Solihull, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Shireen Guide, MD
Role: primary
Joyce Teng, MD PhD
Role: primary
Anna Bruckner, MD
Role: primary
Mercedes Gonzalez, MD
Role: primary
Amy S. Paller, MD
Role: primary
Jordan Slutsky, MD
Role: primary
Kalyani Marathe, MD, MPH
Role: primary
Lara Wine Lee, MD PhD
Role: primary
John C. Browning, MD
Role: backup
Laura Scardamaglia, MD
Role: primary
Dedee Murrell, MD
Role: primary
Artiene Tatian, MD
Role: primary
Martin Laimer, MD
Role: primary
Caroline Colmant, MD
Role: primary
Christine Bodemer, MD, PhD
Role: primary
Alexandros Stratigos, MD
Role: primary
Dimitra Kyritsi, MD
Role: primary
Rahul Mahajan, MD
Role: primary
Fiona Browne, MD
Role: primary
Mor Pavlosky, MD
Role: primary
Iria Neri, MD
Role: primary
Sophie Guez, MD
Role: primary
Cristina Magnoni, MD
Role: primary
Biagio Didona, MD
Role: primary
Andrea Diociaiuti, MD
Role: primary
Kin Fon Leong, MD
Role: primary
Emerson Vista, MD
Role: primary
Anjuli Jaen, MD
Role: primary
Mae Quizon, MD
Role: primary
Katarzyna Osipowicz, MD
Role: primary
Sang Eun Lee, MD
Role: primary
Rocio Maseda Pedrero, MD
Role: primary
Chao-Kai Hsu, MD PhD
Role: primary
Shaden Mohammad Abdel Hadi, MD
Role: primary
Anna Martinez, MD
Role: primary
Ajoy Bardhan, MD
Role: primary
Other Identifiers
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AC-203-EBS-007
Identifier Type: -
Identifier Source: org_study_id
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