jRCT2011240064招募中不适用
Multi-centeR, open-label study to EValuate the safety, efficacy, and pharmacokinetics of FIlsuVEz (Oleogel-S10) in Japanese subjects with epidermolysis bullosa (REVIVE)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 6
- 主要终点
- -
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Non-randomized Controlled Trial
- 干预模型
- Single Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Open(masking Not Used)
入排标准
- 年龄范围
- 3week old over 至 No limit（—）
- 性别
- All
入选标准
- •Male or female aged >= 21 days
- •Confirmed diagnosis of either JEB or DEB
- •Both biological parents and all 4 grandparents of Japanese descent
- •At least 3 EB wounds that meet the following criteria at the time of enrollment:
- •All are located outside of the anogenital region
- •All are distinguishable from any other wounds on the subjects body (separated by a minimum of 5 cm)
- •At least one has been present for a minimum of 21 days but less than 9 months AND is 10 cm2 to 50 cm2 in size
- •At least two have been present for a minimum of 21 days but less than 9 months AND OR are 10 cm2 to 50 cm2 in size
- •Note: wound size will be determined by the ARANZ Silhouette system as described in Protocol.
- •A female subject must meet one of the following criteria:
- •If of childbearing potential, she must:
- •a. Have a negative pregnancy test result at Screening and Baseline Visits, AND
- •b. Agree to use one of the following highly reliable methods of contraception from the day of the informed consent signature until the day after the last Oleogel-S10 application. The following methods are acceptable:
- •Placement of an intrauterine device (IUD) or intrauterine releasing system (IUS)
- •Combined (both oestrogen and progestogen) hormonal contraception (oral) associated with inhibition of ovulation, supplemented with a barrier method (preferably male condom)
- •Bilateral tubal occlusion
- •Sexual abstinence, defined as refraining from heterosexual intercourse during the entire study period
- •Partner vasectomy, provided that the partner is the sole sexual partner and has received medical verification of the surgical success
- •Be of non-childbearing potential, defined as one of the following:
- •Pre-menarche, OR
- •Post-menopausal (12 consecutive months of amenorrhea), OR
- •Permanently sterile following hysterectomy, bilateral salpingectomy, or bilateral oophorectomy (supporting evidence required)
- •Subject and or their legal representative has been informed about the study, has read and understood the information provided, and has given written informed consent
- •Subject and or their legal representative is able and willing to follow all study procedures and instructions
排除标准
- •Hypersensitivity to Oleogel-S10 or any of its excipients
- •Diagnosis of EB subtypes simplex or Kindler EB
- •Receipt of systemic antibiotics for wound-related infections within 7 days prior to enrollment
- •Receipt of systemic or topical (on EB wounds) steroids within 30 days prior to enrollment
- •Exceptions: Inhaled, ophthalmic, or mucosal applications, such as budesonide suspension for esophageal strictures
- •Receipt of immunosuppressive therapy or cytotoxic chemotherapy within 60 days prior to enrollment
- •Note: Dupilumab to manage pruritis is allowed if the subject has been on a stable dose for more than 3 months prior to baseline
- •Receipt of systemic gene therapy for the treatment of inherited EB
- •Receipt of short-acting stem cell therapy (infusion of Muse cells, allogeneic adipocyte-derived MSC sheet (topically applied), TEMCELL (allogeneic MSCs, subcutaneous injection)) within 6 months prior to study enrollment
- •Receipt of topical gene therapy for the treatment of inherited EB (e.g., beremagene geperpavec) within 3 months prior to enrollment
- •Receipt of a JACE skin graft on one of the selected wounds
- •Current and or former malignancy, including skin malignancies such as basal cell carcinoma and squamous cell carcinoma
- •Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until study participation ends
- •Enrollment in any other interventional study or treated with any other investigational drug for any disease within 4 weeks prior to enrollment
- •Presence of any factor in the subject and/or legal representative that could interfere with study compliance, such as the ability to attend the scheduled study visits or to properly manage the dressing changes at home
结局指标
主要结局
-
Incidence of complete closure of the target wounds within 45 days of treatment
次要结局
- Proportion of subjects achieving complete closure of at least one target wound(from baseline through Day 45 and from baseline through Day 90)
- Proportion of subjects achieving complete closure of a target wound that meets both the criteria listed in inclusion criterion #4(from baseline through Day 45 and from baseline through Day 90)
- Mean change from baseline in the surface area of each of the 3 target wounds(Day 45 and Day 90)
- Score on the Patient Global Impression of Improvement (PGI-I)(Day 45 and Day 90)
- Score on the Clinician Global Impression of Improvement (CGI-I)(Day 45 and Day 90)
- Mean change from baseline in the frequency of dressing changes(Day 45 and Day 90)
- The incidence of infections in the target wounds(Day 45 and Day 90)
- Incidence of complete closure of the target wounds within 90 days of treatment(through Day 90)
- Time to first complete closure of any of the 3 target wounds(through Day 90)
- Time to first complete closure of a target wound that meets both the criteria listed in inclusion criterion #4(through Day 90)
- The maximum severity of infection in the target wounds(through Day 90)
研究者
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