TABLE 4.
Change from baseline in biologic outcomes for all patients.
| A) Part 1 | |||||||
| Delandistrogene moxeparvovec (n = 20) a | Placebo (n = 21) b | p-value | |||||
|---|---|---|---|---|---|---|---|
| CFBL to Week 12 | CFBL to Week 12 | ||||||
| Western blot adjusted for muscle content, % normal | Mean (SD) | 23.82 (39.76) | 0.14 (1.24) | <0.0001 | |||
| Vector genome copy number | Mean (SD) | 1.56 (1.51) | 0.00 | <0.0001 | |||
| Fiber intensity, % control | Mean (SD) | 25.81 (46.23) | −0.48 (6.29) | 0.0002 | |||
| PDPF, % | Mean (SD) | 23.88 (25.58) | 5.09 (12.96) | 0.0056 | |||
| B) Part 2 Week 12 (crossover) | |||||||
| Placebo/delandistrogene moxeparvovec (n = 21)† | |||||||
| CFBL to Week 12 | |||||||
| Western blot adjusted for muscle content, % normal | Mean (SD) | 39.64 (31.79) | |||||
| p-value | <0.0001 | ||||||
| Vector genome copy number | Mean (SD) | 3.43 (2.02) | |||||
| p-value | <0.0001 | ||||||
| Fiber intensity, % control | Mean (SD) | 74.09 (47.69) | |||||
| p-value | <0.0001 | ||||||
| PDPF, % | Mean (SD) | 78.92 (22.47) | |||||
| p-value | <0.0001 | ||||||
| C) Part 2 Week 12 (Part 1 Week 60) (crossover) | |||||||
| Delandistrogene moxeparvovec/placebo (n = 18) a | |||||||
| CFBL to Week 12 (Part 1 Week 60) | |||||||
| Western blot adjusted for muscle content, % normal | Mean (SD) | 19.10 (36.43) | |||||
| p-value | 0.0048 | ||||||
| Vector genome copy number | Mean (SD) | 0.94 (1.25) | |||||
| p-value | <0.0001 | ||||||
| Fiber intensity, % control | Mean (SD) | 38.30 (62.83) | |||||
| p-value | 0.0014 | ||||||
| PDPF, % | Mean (SD) | 57.12 (30.63) | |||||
| p-value | <0.0001 | ||||||
a
Patients who received delandistrogene moxeparvovec at all doses in Part 1 and placebo in Part 2. Two patients did not receive placebo in Part 2 and did not have the Part 2 (Week 60) biopsy: one patient had to be tapered off of steroids due to a steroid-related AE and one patient required elective surgery for a femur fracture that required significant recovery time; both patients continue to be followed for efficacy and safety.
b
Patients who received placebo in Part 1 and delandistrogene moxeparvovec at all doses in Part 2. AE, adverse event; CFBL, change from baseline; PDPF, percent dystrophin-positive fibers; SD, standard deviation.