Total vector DNA biodistribution and shedding following 6 × 1013 vg/kg valoctocogene roxaparvovec infusion
| . | Participants with detectable vector DNA, n (%) . | Time to first detectable sample∗, wk . | Peak concentration†, vg/mL . | Time to peak concentration, wk . | Time to first BLOQ/negative sample‡, wk . | Participants with 3 consecutive BLOQ/negative samples, n (%) . | Time to first negative sample‡,§, wk . | Participants with 3 consecutive negative samples, n (%) . |
|---|---|---|---|---|---|---|---|---|
| Blood | ||||||||
| n | 134 (100) | 134 | 134 | 134 | 18 | 18 (13.4) | 0 | 0 (0) |
| Median | 0.1 | 4.7 × 1010 | 0.1 | 123.0 | 0 | |||
| Min | 0.1 | 1.6 × 108 | 0.1 | 31.9 | 0 | |||
| Max | 1.0 | 2.0 × 1011 | 1.0 | 182.0 | 0 | |||
| Saliva | ||||||||
| n | 134 (100) | 134 | 134 | 134 | 134 | 134 (100) | 111 | 111 (82.8) |
| Median | 0.1 | 6.6 × 107 | 0.1 | 6.6 | 48.0 | |||
| Min | 0.1 | 1.3 × 106 | 0.1 | 3.1 | 7.9 | |||
| Max | 1.1 | 4.3×109 | 2.3 | 52.0 | 122.0 | |||
| Semen | ||||||||
| n | 133‖ (100) | 133 | 133 | 133 | 133 | 133 (100) | 122 | 122 (91.7) |
| Median | 0.1 | 1.8 × 106 | 1.0 | 6.1 | 20.6 | |||
| Min | 0.1 | 1.2 × 104 | 0.1 | 0.6 | 6.1 | |||
| Max | 2.1 | 1.0 × 1010 | 12.1 | 36.1 | 94.0 | |||
| Stool | ||||||||
| n | 134 (100) | 134 | 134 | 134 | 119 | 119 (88.8) | 7 | 7 (5.2) |
| Median | 0.1 | 2.7 × 105 | 1.1 | 44.1 | 105 | |||
| Min | 0.1 | 2.1 × 102 | 0.1 | 12.0 | 68.4 | |||
| Max | 6.3 | 5.7 × 106 | 6.3 | 131.0 | 144.0 | |||
| Urine | ||||||||
| n | 134 (100) | 134 | 134 | 134 | 134 | 134 (100) | 132 | 132¶ (98.5) |
| Median | 0.1 | 8.9 × 104 | 0.2 | 2.3 | 12.1 | |||
| Min | 0.1 | 7.7 × 103 | 0.1 | 0.3 | 3.0 | |||
| Max | 0.9 | 3.7 × 107 | 2.3 | 8.1 | 48.0 |
| . | Participants with detectable vector DNA, n (%) . | Time to first detectable sample∗, wk . | Peak concentration†, vg/mL . | Time to peak concentration, wk . | Time to first BLOQ/negative sample‡, wk . | Participants with 3 consecutive BLOQ/negative samples, n (%) . | Time to first negative sample‡,§, wk . | Participants with 3 consecutive negative samples, n (%) . |
|---|---|---|---|---|---|---|---|---|
| Blood | ||||||||
| n | 134 (100) | 134 | 134 | 134 | 18 | 18 (13.4) | 0 | 0 (0) |
| Median | 0.1 | 4.7 × 1010 | 0.1 | 123.0 | 0 | |||
| Min | 0.1 | 1.6 × 108 | 0.1 | 31.9 | 0 | |||
| Max | 1.0 | 2.0 × 1011 | 1.0 | 182.0 | 0 | |||
| Saliva | ||||||||
| n | 134 (100) | 134 | 134 | 134 | 134 | 134 (100) | 111 | 111 (82.8) |
| Median | 0.1 | 6.6 × 107 | 0.1 | 6.6 | 48.0 | |||
| Min | 0.1 | 1.3 × 106 | 0.1 | 3.1 | 7.9 | |||
| Max | 1.1 | 4.3×109 | 2.3 | 52.0 | 122.0 | |||
| Semen | ||||||||
| n | 133‖ (100) | 133 | 133 | 133 | 133 | 133 (100) | 122 | 122 (91.7) |
| Median | 0.1 | 1.8 × 106 | 1.0 | 6.1 | 20.6 | |||
| Min | 0.1 | 1.2 × 104 | 0.1 | 0.6 | 6.1 | |||
| Max | 2.1 | 1.0 × 1010 | 12.1 | 36.1 | 94.0 | |||
| Stool | ||||||||
| n | 134 (100) | 134 | 134 | 134 | 119 | 119 (88.8) | 7 | 7 (5.2) |
| Median | 0.1 | 2.7 × 105 | 1.1 | 44.1 | 105 | |||
| Min | 0.1 | 2.1 × 102 | 0.1 | 12.0 | 68.4 | |||
| Max | 6.3 | 5.7 × 106 | 6.3 | 131.0 | 144.0 | |||
| Urine | ||||||||
| n | 134 (100) | 134 | 134 | 134 | 134 | 134 (100) | 132 | 132¶ (98.5) |
| Median | 0.1 | 8.9 × 104 | 0.2 | 2.3 | 12.1 | |||
| Min | 0.1 | 7.7 × 103 | 0.1 | 0.3 | 3.0 | |||
| Max | 0.9 | 3.7 × 107 | 2.3 | 8.1 | 48.0 |
Defined as time to first positive shedding sample.
Units for stool reported as vg/mg.
Confirmed by 2 consecutive samples.
FDA definition of time to clearance.
One participant did not have available semen shedding assessments.
Three consecutive negatives were confirmed by week 24 in 1 participant after the data snapshot, and another participant achieved clearance defined by 3 consecutive BLOD and/or negative samples by week 32.