An Open Label Extension, Multi-Center, Study to Evaluate the Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 42
- 主要终点
- Number Of Participants With Treatment-Emergent Adverse Events (TEAEs)
研究概览
简要总结
The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB).
Funding Source: FDA Office of Orphan Products Development
详细描述
This was an open-label extension study to assess the continued safety of topically applied SD-101 dermal cream (6%) in participants with Simplex, Recessive Dystrophic, and Junctional non-Herlitz EB.
SD-101 dermal cream (6%) was applied topically, once a day to the entire body for the duration of the study. Participants who successfully completed the entire SD-003 study (NCT02014376) had the option to roll over into the SD-004 study (NCT02090283). The baseline visit occurred at the final visit date for the SD-003 study. The body surface area (BSA) coverage of blisters and lesions assessment made at the final SD-003 study visit were used as the baseline information at the baseline visit for the SD-004 study. Participants returned to the study site for the following 13 visits (36 months) to have BSA assessed. BSA was assessed at all subsequent scheduled study center visits. Scheduled study center visits occurred every 6 months after Month 36 (Month 42, 48, and so on). After completion of Month 36, the next participant visit (Month 39) was a phone call from the site to the participant. Telephone visits occurred every 6 months thereafter (Month 45, 51, and so on) and included assessment of adverse events and concomitant medications only. For female participants of childbearing potential, a urine pregnancy test was performed at Month 6 and every 6 months up to and including the final study visit.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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 participant's legal representative; if the participant is under the age of 18 but capable of providing assent, signed assent from the participant.
- •Participant (or caretaker) must be willing to comply with all protocol requirements.
- •Participant must have successfully completed the SD-003 study.
排除标准
- •Participants who do not meet the inclusion criteria.
- •Pregnancy or breastfeeding during the study. A urine pregnancy test will be performed at the final visit for SD-003 for female participants of childbearing potential.
- •Females of childbearing potential who are not abstinent and not practicing a medically acceptable method of contraception.
研究组 & 干预措施
SD-101 Dermal Cream (6%)
All participants applied SD-101 dermal cream topically, once a day, to the entire body for the duration of the study.
干预措施: SD-101 dermal cream (6%) (Drug)
结局指标
主要结局
Number Of Participants With Treatment-Emergent Adverse Events (TEAEs)
时间窗: From baseline to 30 days after last application of study drug (up to a maximum of 54 months)
Treatment-emergent adverse events were defined as adverse events that started or worsened on or after baseline visit, which occurred at the final visit date for the SD-003 study. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
次要结局
- Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 24(Baseline, Month 24)