A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 22
- 试验地点
- 2
- 主要终点
- Itch Severity
研究概览
简要总结
A Long-Term Extension Study for Participants Previously Treated with EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
详细描述
This is an open-label, long-term, follow-up study in participants from prior interventional trials involving surgical application of gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites. Up to 22 participants will be enrolled in this study, with follow-up through at least 5 years post treatment. Patient-reported outcomes and safety will be assessed throughout the study, and where applicable, compared to Baseline of the preceding interventional EB-101 trial. Upon completion, participants will transfer to an annual monitoring program for additional 10 years of phone visits.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 6 Years 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;
- •If under the age of 18, guardian(s) is/are willing and able to give consent;
- •Prior study treatment with EB-101.
排除标准
- •Inability to properly follow protocol as determined by the Principal Investigator
研究组 & 干预措施
Long-Term Extension
This study will follow one group of participants who were enrolled in a previous EB-101 study.
干预措施: EB-101 (Biological)
结局指标
主要结局
Itch Severity
时间窗: 5 years
Longitudinal change of Itch severity scores assessed at all clinic visits up to Week 54.
Wound Closure
时间窗: 5 years
Proportion of RDEB wounds with healing ≥50%, ≥75%, and 100% at all clinic visits up to Month 54 as determined by direct investigator assessment.
Pain Reduction
时间窗: 5 years
Pain reduction assessed by Wong-Baker FACES scale at all clinic visits up to Month 54.
Wound Infection and Adverse Events
时间窗: 5 years
The number of participants and wounds that have an infection or any related adverse event.
Serious Adverse Events
时间窗: 5 years
The number of participants and wounds that result in hospitalization (serious adverse event \[SAE\]).
Zarit Burden Interview
时间窗: 5 years
Longitudinal change of Zarit Burden Interview Short Form (ZBI-12) for caregiver scores related to wound care, assessed at all clinic visits up to Month 54.
Quality of Life
时间窗: 5 years
Quality of Life in Epidermolysis Bullosa (QOLEB) scores assessed at all clinic visits up to Month 54.
The number of treatment-emergent adverse events.
时间窗: 5 years
The number of treatment-emergent adverse events.
Caregiver Global impression of Pain
时间窗: 5 years
Caregiver Global impression of Pain (CrGI-Pain) scores assessed at all clinic visits up to Month 54.
Incidence of squamous cell carcinoma.
时间窗: 5 years
Incidence of squamous cell carcinoma.
Replication-competent retrovirus (RCR) status
时间窗: 5 years
Replication-competent retrovirus (RCR) status
Pain Severity Scale (Wong-Baker FACES)
时间窗: 5 years
Change in pain assessed by Wong-Baker FACES scale at all clinic visits up to Month 54. The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Itch Severity Scale
时间窗: 5 years
Longitudinal change of Itch severity scores assessed at all clinic visits up to Week 54. The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Caregiver Global Impression of Change for Pain Scale
时间窗: 5 years
Caregiver Global Impression of Change for Pain (CrGI-Pain) scores assessed at all clinic visits up to Month 54. The minimum score value is 1 and the maximum score value is 7, with the higher scores being the worse outcome.
Zarit Burden Interview Scale
时间窗: 5 years
Longitudinal change of Zarit Burden Interview Short Form (ZBI-12) for caregiver scores related to wound care, assessed at all clinic visits up to Month 54. The minimum score value is 0 and the maximum score value is 4, with the higher scores being the worse outcome.
Quality of Life in Epidermolysis Bullosa
时间窗: 5 years
Quality of Life in Epidermolysis Bullosa (QOLEB) responses will be assessed at all clinic visits up to Month 54.
次要结局
未报告次要终点