Open Label Treatment of Beremagene Geperpavec (B-VEC)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 47
- 试验地点
- 12
- 主要终点
- To record safety outcomes of participants while on B-VEC.
研究概览
简要总结
This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study is for those who participated in Phase 3 study, as well as, new participants who were unable to participate in the Phase 3 study, who meet all enrollment criteria.
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
- 2 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Willing and able to give consent/assent
- •Clinical diagnosis of epidermolysis bullosa
- •Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A
- •Age: 2 months of age and older at the time of informed consent/assent
- •Women of childbearing age must be willing to use reliable birth control method throughout the treatment duration and for 3 months following the last treatment application
排除标准
- •Diseases or conditions that could interfere with the assessment of safety of the study treatment and compliance of the participant with study visits/procedures, as determined by the Investigator
- •Pregnant or nursing women
- •Active infection in the area that will undergo administration, that the Investigator believes will negatively impact the IP application
- •Known allergy to any of the constituents of the product
- •Concurrent skin transplant or mesh skin grafting; if the participant is currently having transplantation or grafting surgeries they must wait until the transplant has begun to heal and the graft has absorbed prior to initiating B-VEC therapy
- •Participation in an interventional gene therapy clinical trial within the past three (3) months (not including B-VEC administration)
结局指标
主要结局
To record safety outcomes of participants while on B-VEC.
时间窗: Up to 112 weeks
Record reportable Adverse (AE) and Serious Adverse Events (SAEs) during the continued use of B-VEC to participants who have participated in and completed Krystal Biotech's Phase 3 Protocol (A Phase 3 Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)), as an extension of use, upon study completion; as well as, for participants diagnosed with DEB who have not participated in the B-VEC Phase 3 trial.
次要结局
未报告次要终点