RECRUITING

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)

Conditions

Generalized Epidermolysis Bullosa Simplex Epidermolysis Bullosa Simplex EBS

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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]

Quick Facts

Study Start:2024-04-04

Study Completion:2026-03

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06073132

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Months

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Patient is at least 6 months old at Visit 2 (Day 1/Baseline A). 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. Patients with ≥ 5% 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.	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.

Inclusion Criteria

Exclusion Criteria

1. Patient is at least 6 months old at Visit 2 (Day 1/Baseline A).
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. Patients with ≥ 5% 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.

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.

Contacts and Locations

Study Contact

Sandy Lin, PhD

CONTACT

+886-2-2657-1788

sandy.lin@twibiotech.com

TWiB

CONTACT

+886-2-2657-1788

twib-ebs@twibiotech.com

Study Locations (Sites)

Mission dermatology Center

Rancho Santa Margarita, California, 92688

United States

Stanford University

Stanford, California, 94304

United States

Children's Hospital Colorado

Aurora, Colorado, 80045

United States

Pediatric Center of Excellence (PCE) - Miami Pediatric Endocrinology, LLC

Miami, Florida, 33146

United States

Northwestern University - Lurie Childrens's Hospital

Chicago, Illinois, 60611

United States

Stony Brook Dermatology

Stony Brook, New York, 11790

United States

Cincinnati Childrens Hospital

Cincinnati, Ohio, 45229

United States

Medical University of South Carolina

Charleston, South Carolina, 29425

United States

Texas Dermatology and Laser Specialists

San Antonio, Texas, 78218

United States

Collaborators and Investigators

Sponsor: TWi Biotechnology, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-04-04

Study Completion Date2026-03

Study Record Updates

Study Start Date2024-04-04

Study Completion Date2026-03

Terms related to this study

Keywords Provided by Researchers

Epidermolysis Bullosa Simplex
EBS

Additional Relevant MeSH Terms

Generalized Epidermolysis Bullosa Simplex