pidermolysis bullosa simplex
Conditions
Interventions
Treatment Groups:Topical application on all EBS lesions and on at least approximately 3?4 inch (2 cm) of uninvolved skin surrounding the site
Control Group:Topical application on all EBS lesions and on at least approximately 3?4 inch (2 cm) of uninvolved skin surrounding the site
Sponsors
Dematology Hospital of Southern Medical University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. The subject is at least 6 months old at Visit 2 (Day 1 / Baseline A); 2. The subject is clinically diagnosed with severe or moderate EBS and confirmed by genetic testing to have an autosomal dominant mutation in the KRT5 or KRT14 gene; 3. The subject has an EBS lesion BSA = 3% at Visit 2 (Day 1 / Baseline A), excluding the palms and soles; 4. The subject is clinically diagnosed with severe or moderate EBS and confirmed by genetic testing to have an autosomal dominant mutation in the KRT5 or KRT14 gene; 5. The subject/caregiver agrees to follow the study drug administration instructions; 6. The subject (and caregiver/legal guardian) agrees to report all prescription and over-the-counter medications used during the study, including topical treatments for the body, such as medical cleansers, bleach-containing cleansers, bleach baths, topical antiseptics, topical disinfectants, etc.; 7. The subject (and caregiver/legal guardian) is willing and able to comply with all study visits and all protocol requirements, including completing questionnaires; 8. The subject (and caregiver/legal guardian) can provide written informed consent; assent is obtained based on the subject's age; 9. Female subjects of childbearing potential must undergo a pregnancy test before randomization, and the result must be negative; 10. Female subjects of childbearing potential are willing to use highly effective contraception (i.e., pregnancy prevention measures with a failure rate of <1% per year) from the screening period until the end of the study.
Exclusion criteria
Exclusion criteria: 1.Patient has a clinically significant skin disease other than EBS (e.g., psoriasis, atopic or other dermatitis, 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 judgment, 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, aspartate aminotransferase, or total bilirubin > 2.5 the upper limit of normal), or renal abnormalities (estimated glomerular filtration rate [eGFR] < 30 ml/min/1.73 m^2) 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 malignancies 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint of this study is the proportion of subjects who achieve treatment success on the IGA of the treated area, where treatment success is defined as a score of 0 or 1 with at least a 2; | — |
Secondary
| Measure | Time frame |
|---|---|
| Incidence and proportion of patients with AEs, including treatment emergent adverse events (TEAEs), and serious adverse events and relationship to the study medication.;Change from Baseline A (Visit 2/Day 1) in clinical laboratory results in hematology, biochemistry, and urinalysis. (If the tests are omitted at Baseline A at the discretion of the investigator, the cl;Change in EBS lesion BSA % in the treatment area from baseline A (Visit 2/Day 1) to Week 8 (Visit 5/EOT);Change in pain intensity scores from Baseline A (Visit 2/Day 1) to Week 8 (Visit 5/EOT).;Change from Baseline A (Visit 2/Day 1) in vital signs, physical examination, and ECG parameters. (If the tests are omitted at Baseline A at the discretion of the investigator, the closest data during;Change in pruritus intensity scores from Baseline A (Visit 2/Day 1) to Week 8 (Visit 5/EOT).;Incidence and proportion of patients with mild, moderate, and severe AEs.;Change in EBDASI score (skin activity) from Baseline A (Visit 2/Day 1) to Week 8 (Visit 5/EOT).;Change in the QOLEB from Baseline A (Visit 2/Day 1) to Week 8 (Visit 5/EOT).; | — |
Countries
China
Contacts
Public ContactZhimiao Lin
Dematology Hospital of Southern Medical University
Outcome results
None listed