A Low-Interventional Study Of AAV9 Neutralizing Antibody Seroconversion in Household Contacts of Participants Within the C3391003 Clinical Trial
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- Pfizer
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Number of Participants (Previously Seronegative for Neutralizing Antibodies [NAbs] to AAV9) Who Developed NAb to AAV9 at Day 28 After the Interventional Study Patient Was Dosed
研究概览
简要总结
This study will include male and female participants who live or work in the same household as a patient in one of the fordadistrogene movaparvovec interventional studies. Up to 5 participants from the same household may be enrolled. The objective is to estimate the likelihood of NAb seroconversion to AAV9 in household contacts of a patient in one of the interventional studies who is treated with fordadistrogene movaparvovec gene therapy.
详细描述
This single center study will include approximately 50 to 250 participants and is designed to estimate the likelihood of NAb seroconversion to AAV9 because of exposure to shed viral vector material released by a DMD patient treated with fordadistrogene movaparvovec in an interventional study. Eligible participants will undergo a blood draw provided by a Home Health Care Vendor at three home visits.
The total duration of participation in this study is about 4 months, including up to 48 days for the screening/baseline period and about 56 days after the DMD patient of the same household is dosed with the investigational gene therapy in the interventional study.
To maintain the blind in the blinded interventional studies, all C3391007 study participants and investigators as well as the sponsor will remain blinded to the Cohort assignment of the interventional study patient until the interventional study becomes unblinded.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 0 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Males or females who weigh at least 9 kg.
- •Anticipated to be living or working in the same household as a patient in one of the fordadistrogene movaparvovec interventional studies for at least 4 months.
- •Anticipated to have > 10 hours of contact per week and expected to have direct contact with the interventional study patient.
- •The interventional patient is dosed in the interventional study.
排除标准
- •Prior treatment with gene therapy utilizing AAV vectors of any serotype.
结局指标
主要结局
Number of Participants (Previously Seronegative for Neutralizing Antibodies [NAbs] to AAV9) Who Developed NAb to AAV9 at Day 28 After the Interventional Study Patient Was Dosed
时间窗: Day 28 after the interventional study participant was dosed
Development of NAb to AAV9 was defined by an increase of \>=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer \>=1 and \<4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer \<1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer \>=6.
Number of Participants (Previously Seronegative for NAb to AAV9) Who Developed NAb to AAV9 at Day 56 After the Interventional Study Patient Was Dosed
时间窗: Day 56 after the interventional study participant was dosed
Development of NAb to AAV9 was defined by an increase of \>=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer \>=1 and \<4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer \<1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer \>=6.
次要结局
- Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 28 After the Interventional Study Patient Was Dosed(Day 28 after the interventional study participant was dosed)
- Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 56 After the Interventional Study Patient Was Dosed(Day 56 after the interventional study participant was dosed)