A Phase 2b, Multi-Center, Randomized, Double-Blind, Vehicle-Controlled Study of the Effectiveness and Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 48
- 主要终点
- Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment
研究概览
简要总结
The purpose of this study was to assess whether the topical use of SD-101 cream (3% or 6%) was effective in treating wounds in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa (EB).
详细描述
This was a Phase 2b, multi-center, randomized, double-blind, vehicle-controlled study to assess the efficacy and safety of SD-101 dermal cream (3% or 6%) on skin lesions in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
SD-101 cream [containing 3% or 6% or vehicle (0%)] was applied topically, once a day to the entire body for a period of 90 days. Eligible participants had a target wound assessed at baseline. Selected target wound had to be at least a certain age and within a prespecified size range at study entry. Photographic confirmation of the target wound location was collected at baseline, and the picture saved from the first visit was used to confirm location of the target wound at subsequent visits. The participant returned to the study site at Week 2, Month 1, Month 4, and Month 5 (at 14, 30, 60, and 90 days, respectively) to have the target wound previously identified at baseline re-assessed for the level of healing. In addition, changes in itching, pain, body surface area coverage of blisters and lesions, and scaring of the healed target wound were also assessed at each visit.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Informed Consent form signed by the participant or the participant's legal representative; if the participant is under the age of majority but capable of providing assent, signed assent from the participant.
- •Participant (or caretaker) was willing to comply with all protocol requirements.
- •Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
- •Participants 6 months of age and older.
- •Participants had 1 target wound within a prespecified size range at study entry.
- •Target wound was at least 21 days or older.
排除标准
- •Participants who did not meet all the entry criteria outlined in inclusion criteria.
- •Selected target wound had clinical evidence of local infection.
- •Use of any investigational drug within 30 days before enrollment.
- •Use of immunotherapy or cytotoxic chemotherapy within 60 days before enrollment.
- •Use of systemic or topical steroidal therapy within 30 days before enrollment (inhaled steroids and ophthalmic drops containing steroids were allowed).
- •Use of systemic antibiotics within 7 days before enrollment.
- •Current or former malignancy.
- •Arterial or venous disorder resulting in ulcerated wounds.
- •Diabetes mellitus.
- •Pregnancy or breastfeeding during the study (a urine pregnancy test was performed at screening for female participants of childbearing potential).
- •Females of childbearing potential who were not abstinent and not practicing a medically acceptable method of contraception.
- •Known history of cardiac, hepatic, or renal disease.
研究组 & 干预措施
SD-101 Dermal Cream (6%)
SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days.
干预措施: SD-101 Dermal Cream (6%) (Drug)
SD-101 Dermal Cream (3%)
SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days.
干预措施: SD-101 dermal cream (3%) (Drug)
Vehicle (0%)
Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days.
干预措施: Vehicle (SD-101 0%) (Drug)
结局指标
主要结局
Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment
时间窗: Baseline to 1 Month
The ARANZ SilhouetteStar™, a wound imaging, measurement, and documentation system providing accurate wound assessment, was used to measure the target wound at all visits. Information captured included photographic images, quantitative measures, and other target wound assessment data input to the device by the clinician, all obtained with no contact to the participant's skin. Information about the target wound's measurement history was available on this system so that the serial progression of the target wound status could also be calculated and presented.
次要结局
- Participants Experiencing A Change From Baseline In Itching At Day 7(Baseline, Day 7)
- Percentage Change From Baseline In Lesional Skin Based On Body Surface Area Index (BSAI) Measurements At Month 3(Baseline, Month 3)
- Participants With Scarring At Week 2, Month 1, Month 2, And Month 3(Week 2, Month 1, Month 2, and Month 3)
- Participants With Documented Complete Closure Of The Target Wound Within 2 And 3 Months After Initiation Of Treatment(Baseline to Month 2 and Month 3)
- Change From Baseline In Pain At Day 7(Baseline, Day 7)