Health Condition 1: Q810- Epidermolysis bullosa simplex
Conditions
Interventions
Intervention1: AC-203 diacerein 1 percent ointment: Dose Formulation : Ointment
Dose Strength : 1 percent
Route of Administration :Topical
Dosage Level : To be applied topically once daily to all EBS
Sponsors
TWi Biotechnology, Inc.
Veeda Clinical Research Ltd
Eligibility
Inclusion criteria
Inclusion criteria: 1) Patient is at least 6 months of age at Visit 2 that is Day 1 or Baseline A. 2) Patient 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 more than or equal to 5 percent BSA of EBS lesions excluding palms and soles at Visit 2. 4) EBS lesions of patient within the treatment area have an IGA score of more than or equal to 3 at Visit 2. 5) Patient or caregiver agrees to follow study medication application instructions. 6) Patient and caregiver or legal guardian agrees to report use of all prescription and over-the-counter medications, including topical therapies applied to the body. 7) Patient and caregiver or legal guardian is willing and able to comply with all study visits and all the protocol requirements, including completing questionnaires. 8) Patient and caregiver or legal guardian is able to provide written informed consent or 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 from screening throughout the end of the study.
Exclusion criteria
Exclusion criteria: 1) Patient has a clinically significant skin disease other than EBS or a vascular disorder associated with cutaneous erosions or ulcerations, that may confound assessments of efficacy or safety. 2) Patient has a clinically significant underlying medical condition, psychiatric condition 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. 4) Patient has had a cutaneous infection in the Treatment Area or use systemic antibiotics within 7 days prior to Visit 2. 5) Patient has uncontrolled diabetes mellitus , hepatic enzyme abnormalities or renal abnormalities 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 prior to Visit 2. 7) Patient is treated with protocol-excluded topical therapies other than steroids within 2 weeks prior to Visit 2 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. 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 or immunomodulatory therapies or chemotherapy within 8 weeks prior to Visit 2. 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. 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 |
|---|---|
| To evaluate the efficacy of study drug after 8 weeks of treatment in adult and pediatric patients (aged 6 months and older) with generalized EBS.Timepoint: Proportion of patients achieving treatment success on the IGA of the Treatment Area, in which treatment success is defined as a score of 0 or 1 with at least a 2-point reduction from Baseline A (Visit 2 that is Day 1) to Week 8 (Visit 5 that is EOT). | — |
Secondary
| Measure | Time frame |
|---|---|
| To evaluate the safety & efficacy of study drug in adult & pediatric patients (aged 6 months & older) after 8 weeks of treatment with generalized EBSTimepoint: Efficacy endpoints: Change in percentage BSA of EBS lesions in the Treatment Area, pain intensity, pruritus intensity, EBDASI & QOLEB score from Baseline A Visit 2,3,4 & 5-EOT. Safety endpoints: Incidence & proportion of patients with AEs, SAEs & relationship to the study medication & change in clinical laboratory results in hematology, biochemistry, urinalysis, vital signs, Physical examination & ECG parameters from Baseline A (Visit 2 that is Day 1). | — |
Countries
Australia, Austria, Belgium, France, Greece, India, Ireland, Israel, Italy, Malaysia, Philippines, Poland, Republic of Korea, Spain, Taiwan, United Arab Emirates, United Kingdom, United States of America
Contacts
Public ContactDr Ravi Alamchandani
Veeda Clinical research Limited
Outcome results
None listed