Long-term Follow-up Safety and Efficacy Study in Participants With Duchenne Muscular Dystrophy Who Have Received Fordadistrogene Movaparvovec in a Preceding Clinical Study
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- Pfizer
- 入组人数
- 7
- 试验地点
- 12
- 主要终点
- Number of participants with malignancy adverse event
研究概览
简要总结
The purpose of this study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies. We will follow participants' experience in this study for 10 years after the end of their previous study. Participants will have 1 annual onsite visit and a few annual remote visits. The exact number of remote visits will be decided by their study doctor.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participants who received fordadistrogene movaparvovec in a previous Pfizer interventional study.
排除标准
- •Investigator site staff directly involved in the study and their family members
研究组 & 干预措施
All participants
All participants enrolled in the study.
干预措施: fordadistrogene movaparvovec (Biological)
结局指标
主要结局
Number of participants with malignancy adverse event
时间窗: At least annually from 5 through 10 years after dosing in the interventional study
Number of participants with clinically significant findings in echocardiogram parameters
时间窗: Annually from 5 through 10 years after dosing in the interventional study
Number of participants with serious adverse events
时间窗: At least annually from 5 through 10 years after dosing in the interventional study.
Percentage of participants with serious adverse events
时间窗: At least annually from 5 through 10 years after dosing in the interventional study.
Number of participants with clinically significant findings in electrocardiogram (ECG) assessments
时间窗: Annually from 5 through 10 years after dosing in the interventional study.
Number of participants with clinically significant findings in cardiac troponin I laboratory examinations
时间窗: Annually from 5 through 10 years after dosing in the interventional study
Number of participants with adverse events considered related to treatment
时间窗: At least annually from 5 through 10 years after dosing in the interventional study.
Percentage of particpants with adverse events considered related to treatment
时间窗: At least annually from 5 through 10 years after dosing in the interventional study
次要结局
- Age at loss of ambulation(From 5 through 10 years after dosing in the interventional study)
- Cause of death(From 5 through 10 years after dosing in the interventional study)
- Change from pre-dose in the Upper Limb Function Patient Reported Outcome Measure(Annually from 5 through 10 years after dosing in the interventional study)
- Age at death(From 5 through 10 years after dosing in the interventional study)
- Glucocorticoid use dose and frequency(At least annually from 5 through 10 years after dosing in the interventional study)
- Change from pre-dose in the North Star Ambulatory Assessment total score(Annually from 5 through 10 years after dosing in the interventional study)
- Change from pre-dose in the ability to walk 10 meters unassisted(Annually from 5 through 10 years after dosing in the interventional study.)
- Change from pre-dose in the ability to climb stairs.(Annually from 5 through 10 years after dosing in the interventional study)
- Change from pre-dose in the Performance of Upper Limb (PUL) 2.0 entry score(Annually from 5 through 10 years after dosing in the interventional study)
- Change from pre-dose in percent of predicted forced vital capacity (%pFVC) and percent predicted peak expiratory flow (%pPEF)(Annually from 5 through 10 years after dosing in the interventional study)
- Change from pre-dose in left ventricular ejection fraction (LVEF) on echocardiogram(Annually from 5 through 10 years after dosing in the interventional study)
- Change from pre-dose in the Modified Pediatric Outcomes Data Collection Instrument(Annually from 5 through 10 years after dosing in the interventional study)
- Age when percent predicted forced vital capacity <30%(Annually from 5 through 10 years after dosing in the interventional study)