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.
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.
Study Type
OBSERVATIONAL
Enrollment
8
Blood Samples for NAb and ADA to AAV9
Kennedy Krieger Institute
Baltimore, Maryland, United States
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
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.
Time frame: Day 28 after the interventional study participant was dosed
Number of Participants (Previously Seronegative for NAb to AAV9) Who Developed NAb to AAV9 at Day 56 After the Interventional Study Patient 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.
Time frame: Day 56 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 28 After the Interventional Study Patient Was Dosed
Development of ADA to AAV9 was defined by a titer ≥300 in participants with a negative test (ie, titer \<50) for ADA to AAV9 at Baseline.
Time frame: 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
Development of ADA to AAV9 was defined by a titer ≥300 in participants with a negative test (ie, titer \<50) for ADA to AAV9 at Baseline.
Time frame: Day 56 after the interventional study participant was dosed
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