An Open Label Multi-Center Extension Study to Evaluate the Long-term Safety of Zorblisa™ (SD-101-6.0) in Patients With Epidermolysis Bullosa
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 152
- 主要终点
- Number Of Participants With Treatment Emergent Adverse Events (TEAEs)
研究概览
简要总结
The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).
详细描述
This was an open label, multi-center extension study to assess the long-term safety of topically applied SD-101-6.0 in participants with simplex, recessive dystrophic, and junctional non-Herlitz EB. SD-101-6.0 was applied topically once a day to the entire body. The planned duration of treatment with SD-101-6.0 for Study SD-006 was up to 48 months, with a safety follow-up period of 30 days; however, the study was terminated early by the sponsor. The maximum study duration completed by at least 1 participant, treatment and safety follow-up, was 37 months.
Participants who successfully completed Study SD-005 had the option to rollover into Study SD-006. The screening/baseline visit (Visit 1) occurred at Visit 5 (approximately 90 days from baseline) of Study SD-005. The Body Surface Area (BSA) assessments of lesional skin and wound burden performed at Visit 5 (approximately 90 days from baseline) for Study SD-005 were utilized as the baseline assessments for Study SD-006. Participants returned for follow-up visits at Month 1 then every 3 months.
At each visit, assessments included BSA of lesional skin and wound burden. For target wounds that are not closed by the end of Study SD-005, the ARANZ picture and calculation of target wound area at the final visit for Study SD-005 was used as the baseline area size of the target wound for Study SD-006. These unhealed target wounds from Study SD-005 were assessed via ARANZ SilhouetteStar™ at each subsequent scheduled visit until the target wound was documented as closed. Closed wounds were assessed for scarring.
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
- 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.
- •Participants who completed the SD-005 study (on study drug at Visit 5, approximately 90 days from baseline).
排除标准
- •Participants who did not meet the entry criteria outlined above.
- •Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at the final visit for Study SD-005 for female participants of childbearing potential and repeated at screening/baseline visit of Study SD-006 if these visits did not occur on the same day).
- •Female participants of childbearing potential who were not abstinent or not practicing a medically acceptable method of contraception.
研究组 & 干预措施
Experimental: SD-101-6.0 cream
All participants (or their caregivers) applied SD-101-6.0 cream topically once a day to the entire body for a period of up to 36 months.
干预措施: SD-101-6.0 cream (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 37 months)
TEAEs were defined as adverse events that started or worsened on or after baseline visit.
次要结局
- Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin Up To Month 30(Baseline, up to Month 30)
- Change From Baseline In BSAI Of Total Body Wound Burden Up To Month 30(Baseline, up to Month 30)