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 percent Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS) [EBShield Study]
试验速览
- 阶段
- 2/3 期
- 状态
- 尚未招募
- 入组人数
- 100
- 试验地点
- 2
- 主要终点
- 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.
研究概览
简要总结
This is a double-blind, randomized, multicenter trial with vehicle-controlled phase (Part A) of the Phase 2 and 3 study , designed to demonstrate the efficacy and safety of AC-203 diacerein 1 percent after 8 weeks of treatment in patients with generalized EBS. After completing the Part A of the study, eligible patients in both groups will continue in the open-label extension phase (Part B) to collect long-term safety data
Part A
7 visits and 1 video phone call, 16 weeks
Visit 1 : Week -10 to Day -1: screening, eligibility assessment
Visit 2 : Day 1: Baseline for Part A (Baseline A), enrollment, randomization, first study medication application
Visit 3 : Week 2: treatment period follow-up
Visit 4 : Week 4: treatment period follow-up
Week 6 : treatment period follow-up via Video phone call
Visit 5 : Week 8: end of treatment for Part A (EOT)
Visit 6 : Week 12: no treatment follow-up
Visit 7 : Week 16: end of no treatment follow-up for Part A (EOF), Baseline for Part B (Baseline B)
Part B
Visit 7 : Week 16: Baseline for Part B (Baseline B)
Visit 8 : Week 24: open-label extension treatment period visit
Visit 9 : Week 32: open-label extension treatment period visit
Visit 10 : Week 40: end of study visit (EOS)
研究设计
- 研究类型
- Interventional
- 分配方式
- Computer generated randomization
- 盲法
- Participant, Investigator, Outcome Assessor and Date-entry Operator Blinded
入排标准
- 年龄范围
- 6.00 Month(s) 至 99.00 Year(s)（—）
- 性别
- All
入选标准
- •Patient is at least 6 months of age at Visit 2 that is Day 1 or Baseline A.
- •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.
- •Patient with more than or equal to 5 percent BSA of EBS lesions excluding palms and soles at Visit
- •EBS lesions of patient within the treatment area have an IGA score of more than or equal to 3 at Visit
- •Patient or caregiver agrees to follow study medication application instructions.
- •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.
- •Patient and caregiver or legal guardian is willing and able to comply with all study visits and all the protocol requirements, including completing questionnaires.
- •Patient and caregiver or legal guardian is able to provide written informed consent or 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 from screening throughout the end of the study.
排除标准
- •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.
- •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.
- •Patient has used any diacerein-containing product within 6 months prior to Visit
- •Patient has had a cutaneous infection in the Treatment Area or use systemic antibiotics within 7 days prior to Visit
- •Patient has uncontrolled diabetes mellitus , hepatic enzyme abnormalities or renal abnormalities during the Screening period.
- •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
- •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.
- •Patient has been treated with topical steroids on the EBS lesions within 2 weeks or systemic steroids within 4 weeks.
- •prior to Visit
- •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
- •Patient has been treated with any investigational drug or device within 30 days or 5 half-lives, whichever is longer, prior to Visit
- •Patient has a history of allergy or hypersensitivity to any component of study medications, including diacerein or rhein.
- •Patient is pregnant or breastfeeding/lactating.
- •Patient has a planned or anticipated major surgical procedure or other activity that would interfere with their ability to comply with protocol requirements.
结局指标
主要结局
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.
时间窗: 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).
次要结局
- To evaluate the safety & efficacy of study drug in adult & pediatric patients (aged 6 months & older) after 8 weeks of treatment with generalized EBS(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.)