TABLE 5.
Summary of AEs.
| A) Part 1 | ||
| Delandistrogene moxeparvovec a (n = 20) | Placebo b (n = 21) | |
|---|---|---|
| Total number of AEs | 308 | 230 |
| Patients with at least one AE, n (%) | 20 (100.0) | 21 (100.0) |
| Total number of TEAEs | 285 | 209 |
| Patients with at least one TEAE, n (%) | 20 (100.0) | 21 (100.0) |
| Treatment-related TEAE, n (%) | 17 (85.0) | 9 (42.9) |
| Total number of SAEs | 4 | 2 |
| Patients with at least one SAE, n (%) | 3 (15.0) | 2 (9.5) |
| Treatment-related SAE, n (%) | 3 (15.0) | 1 (4.8) |
| Patients with an AE leading to study discontinuation, n | 0 | 0 |
| Deaths, n | 0 | 0 |
| Treatment-related SAEs, n (%) | ||
| Rhabdomyolysis | 2 (10.0) | 1 (4.8) |
| Increased transaminases | 1 (5.0) | 0 |
| Liver injury | 1 (5.0) | 0 |
| Treatment-related TEAEs, n (%) c | ||
| Vomiting | 12 (60.0) | 4 (19.0) |
| Decreased appetite | 6 (30.0) | 0 |
| Nausea | 6 (30.0) | 2 (9.5) |
| Gamma-glutamyl transferase increased | 5 (25.0) | 0 |
| Abdominal pain upper | 3 (15.0) | 1 (4.8) |
| Abdominal pain | 3 (15.0) | 0 |
| Blood bilirubin increased | 2 (10.0) | 0 |
| Pain in extremity | 2 (10.0) | 1 (4.8) |
| Rhabdomyolysis | 2 (10.0) | 1 (4.8) |
| Pyrexia | 1 (5.0) | 0 |
| B) Part 2 (crossover) | ||
| Placebo a (n = 20) | Delandistrogene moxeparvovec b (n = 21) | |
|---|---|---|
| Total number of AEs | 157 | 278 |
| Patients with at least one AE, n (%) | 19 (95.0) | 21 (100.0) |
| Total number of TEAEs | 131 | 262 |
| Patients with at least one TEAE, n (%) | 19 (95.0) | 21 (100.0) |
| Treatment-related TEAE, n (%) | 4 (20.0) | 20 (95.2) |
| Total number of SAEs | 2 | 1 |
| Patients with at least one SAE, n (%) | 2 (10.0) | 1 (4.8) |
| Treatment-related SAE, n (%) | 0 | 0 |
| Patients with an AE leading to study discontinuation, n | 0 | 0 |
| Deaths, n | 0 | 0 |
| Treatment-related TEAEs, n (%) c | ||
| Vomiting | 0 | 16 (76.2) |
| Decreased appetite | 0 | 15 (71.4) |
| Nausea | 1 (5.0) | 10 (47.6) |
| Gamma-glutamyl transferase increased | 0 | 6 (28.6) |
| Abdominal pain upper | 1 (5.0) | 8 (38.1) |
| Abdominal pain | 0 | 1 (4.8) |
| Blood bilirubin increased | 0 | 2 (9.5) |
| Pyrexia | 0 | 4 (19.0) |
| Thrombocytopenia | 0 | 5 (23.8) |
| Glutamate dehydrogenase increased | 0 | 3 (14.3) |
| Fatigue | 0 | 2 (9.5) |
| Headache | 0 | 2 (9.5) |
| Ketonuria | 1 (5.0) | 1 (4.8) |
| Lethargy | 0 | 2 (9.5) |
| Myalgia | 0 | 2 (9.5) |
| White blood cell count decreased | 0 | 2 (9.5) |
a
Patients who received delandistrogene moxeparvovec in Part 1 and placebo in Part 2. Two patients did not receive placebo in Part 2: 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. Data represent AEs experienced in second year post-treatment.
b
Patients who received placebo in Part 1 and delandistrogene moxeparvovec in Part 2.
c
Treatment-related TEAEs reported in at least two patients in Part 1 or Part 2.
AE, adverse event; SAE, serious AE; TEAE, treatment-emergent AE.