A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 250
- 试验地点
- 4
- 主要终点
- The number and incidence of treatment-related malignancies.
研究概览
简要总结
The goal of this observational study is to evaluate the long-term safety and wound healing durability (up to 15 years) for participants with Recessive Dystrophic Epidermolysis Bullosa (RDEB) who were treated with pz-cel. The main questions it aims to answer are:
- The long-term safety profile of pz-cel.
- The wound healing durability of pz-cel.
- The occurrence of all malignancies after treatment with pz-cel, regardless of type or location, in individuals who received pz-cel treatment for RDEB in the post-marketing setting.
Participants will then be followed annually up to 15 years post-treatment. Assessments will occur via remote data collection. If the participants provider determines an office visit is necessary after the first year, some of this data can be collected in person as well. Annual assessments will include, but are not limited to:
- Review of Adverse Events (AEs), Serious Adverse Events (SAEs), hospitalizations, and any evidence of malignancies and potential retroviral infection
- Wound-healing durability data collection via the modified Caregiver Global Impression of Change for Wound Healing
- Any changes in concomitant medications and procedures.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 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.
- •Patients who received treatment with prademagene zamikeracel in the post-marketing setting.
排除标准
- •Inability to properly follow the protocol as determined by the Principal Investigator (PI).
研究组 & 干预措施
Patients with RDEB who received pz-cel treatment in the post-marketing setting
干预措施: Prademagene Zamikeracel (Combination Product)
结局指标
主要结局
The number and incidence of treatment-related malignancies.
时间窗: From enrollment to 15 years post-treatment
The number and incidence of treatment-emergent SAEs, including systematic and wound specific SAEs
时间窗: From enrollment to 15 years post-treatment
The number and incidences of treatment-related SAEs related to pz-cel, including systematic and wound-specific SAEs.
时间窗: From enrollment to 15 years post-treatment
The number and incidence of positive RCR testing results required for AEs and SAEs where retroviral infection is a consideration
时间窗: From enrollment to 15 years post-treatment
次要结局
未报告次要终点