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]

Status: Recruiting
Location: See all (35) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6 months
Healthy Volunteers: f
View:

• 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.

Locations
United States
California
Mission dermatology Center
RECRUITING
Rancho Santa Margarita
Stanford University
RECRUITING
Stanford
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Florida
Pediatric Center of Excellence (PCE) - Miami Pediatric Endocrinology, LLC
RECRUITING
Miami
Illinois
Northwestern University - Lurie Childrens's Hospital
RECRUITING
Chicago
New York
Stony Brook Dermatology
RECRUITING
Stony Brook
Ohio
Cincinnati Childrens Hospital
RECRUITING
Cincinnati
South Carolina
Medical University of South Carolina
RECRUITING
Charleston
Texas
Texas Dermatology and Laser Specialists
RECRUITING
San Antonio
Other Locations
Australia
Premier Specialists
RECRUITING
Kogarah
Sydney Children's Hospital
RECRUITING
Randwick
Austria
Universitaetsklinik fuer Dermatologie und Allergologie
RECRUITING
Salzburg
Belgium
UZ Leuven
RECRUITING
Leuven
France
Hopital Necker-Enfants Malades
RECRUITING
Paris
Greece
Hospital of Skin and Venereal Diseases of Thessaloniki
RECRUITING
Thessaloniki
India
Postgraduate Institute of Medical Education and Research (PGIMER)
NOT_YET_RECRUITING
Chandigarh
Sri Madhusudan Sai Institute of Medical Science
NOT_YET_RECRUITING
Muddenahalli
Ireland
Children's Health Ireland
RECRUITING
Dublin
Israel
Tel Aviv Sourasky Medical Center
RECRUITING
Tel Aviv
Italy
IRCCS, AOUBO, Policlinico Sant'Orsola
RECRUITING
Bologna
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milano
RECRUITING
Milan
Università degli Studi di Modena e Reggio Emilia (UNIMORE)
RECRUITING
Modena
Istituto Dermopatico dell'Immacolata (IDI) - Istituto di
RECRUITING
Rome
UOS Centro delle Dermatosi Croniche Complesse e Genodermatosi UOC Dermatologia
RECRUITING
Rome
Malaysia
Hospital Tunku Azizah (Hospital Wanita Dan Kanak-kanak Kuala Lumpur)
RECRUITING
Kuala Lumpur
Philippines
Asian Hospital
RECRUITING
City Of Muntinlupa
Iloilo Doctors Hospital
RECRUITING
Iloilo City
Health Cube Medical Clinics
RECRUITING
Mandaluyong
Poland
OT.CO Clinic Osipowicz & Turkowski
RECRUITING
Warsaw
Republic of Korea
Gangnam Severane Hospital
RECRUITING
Seoul
Spain
Hospital Universitario La Paz
RECRUITING
Madrid
Taiwan
National Cheng Kung University Hospital
RECRUITING
Tainan City
United Arab Emirates
Sheikh Khalifa Medical City (SKMC)
RECRUITING
Abu Dhabi
United Kingdom
Great Ormond Street Hospital (GOSH) for Children NHS Foundation Trust - Somers Clinical Research Facility (CRF)
RECRUITING
London
University Hospitals Birmingham NHS Foundation Trust (UHB)
RECRUITING
Solihull
Contact Information
Primary
Sandy Lin, PhD
sandy.lin@twibiotech.com
+886-2-2657-1788
Backup
TWiB
twib-ebs@twibiotech.com
+886-2-2657-1788
Time Frame
Start Date: 2024-04-04
Estimated Completion Date: 2027-01
Participants
Target number of participants: 80
Treatments
Experimental: Part A AC-203
Double-blind, AC-203 Diacerein 1% ointment, QD
Placebo_comparator: Part A Vehicle ointment
Double-blind, Vehicle ointment, QD
Experimental: Part B AC-203
Open-label extension phase, AC-203 Diacerein 1% ointment, QD
Sponsors
Leads: TWi Biotechnology, Inc.

This content was sourced from clinicaltrials.gov