A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 169
- 主要终点
- Time To Complete Target Wound Closure Within 3 Months
研究概览
简要总结
The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0) cream in the treatment of skin lesions in participants with Epidermolysis Bullosa. Funding Source - United States Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD).
详细描述
This was a Phase 3, multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of SD-101-6.0 cream versus placebo (SD-101-0.0) cream on skin lesions in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa. Epidermolysis Bullosa is a rare group of inherited disorders that typically manifest at birth as blistering and lesion formation on the skin in response to little or no apparent trauma. In this study, SD-101-6.0 cream or placebo (SD-101-0.0) cream was to be applied topically, once a day to the entire body for a period of 90 days. Participants had 1 target wound selected at baseline by the investigator. The selected target wound was required to have been present for at least 21 days. 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 for Visit 2 (approximately 14 days from baseline), Visit 3 (approximately 30 days from baseline), Visit 4 (approximately 60 days from baseline), and Visit 5 (approximately 90 days from baseline) to have the target wound assessed for the level of healing. In addition, itching, pain, body surface area, target wound closure, and scarring of healed target wound were assessed at each visit. The ARANZ SilhouetteStar™ was used to measure the target wound at all visits.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 1 Month 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 participant's legal representative; if the participant was under the age of 18 but capable of providing assent, signed assent from the participant.
- •Participant (or caretaker) must have been willing to comply with all protocol requirements.
- •Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
- •Participant must have had 1 target wound (size 10 to 50 cm^2) at study entry.
- •Participants 1 month and older.
- •Target wound must have been present for at least 21 days.
排除标准
- •Participants who did not meet the entry criteria outlined above.
- •Selected target wound did not have clinical evidence of local infection.
- •Use of any investigational drug within the 30 days before enrollment.
- •Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.
- •Use of systemic or topical steroidal therapy within the 30 days before enrollment. (Inhaled steroids and ophthalmic drops containing steroids were allowed).
- •Use of systemic antibiotics within the 7 days before enrollment.
- •Current or former malignancy.
- •Arterial or venous disorder resulting in ulcerated lesions.
- •Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at screening and every 30 days until the final visit for female participants of childbearing potential).
- •Females of childbearing potential who were not abstinent and not practicing a medically acceptable method of contraception.
研究组 & 干预措施
SD-101-6.0 cream
SD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days
干预措施: SD-101-6.0 cream (Drug)
Placebo (SD-101-0.0) cream
SD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days
干预措施: Placebo (SD-101-0.0) cream (Drug)
结局指标
主要结局
Time To Complete Target Wound Closure Within 3 Months
时间窗: From baseline to Month 3 visit
Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. Time to target wound closure was measured from the date of the first administration of the study drug to the date of target wound closure. Participants were censored if they did not have a response within 3 months, or withdrew earlier before the confirmation of their target wound closing. This primary end point displays the mean time to complete target wound closure, analyzed using a Kaplan-Meier approach.
The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months
时间窗: From baseline to Month 3 visit
Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. Participants were considered responders if they experienced complete wound closure at the Week 2 or Months 1, 2, or 3 visits. If a target wound was documented to have closed at a given visit, it was considered closed at all subsequent visits. This primary end point displays the percentage of participants from the ITT population who had complete target wound closure by the end of the study period (that is, 3 months). Analysis was performed on participants with post-baseline wound closure data.
次要结局
- Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit(Baseline, Month 3 visit)
- Change From Baseline In Itching Score At Day 7(Baseline, Day 7)
- Change From Baseline In Pain Score At Day 7(Baseline, Day 7)
- Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits(From baseline to Month 1 and Month 2 visits)
- Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit(Baseline, Month 3 visit)