Table 3.
Micro-dystrophin clinical trials in DMD patients. NSAA: NorthStar Ambulatory Assessment score, SM: Skeletal muscle.
| Trial Promoter/Product Name/Reference | Vector | Promoter | Micro-Dystrophin Domains | Dose vg/kg of Body Weight | Expression in SM | NSAA | Serious Adverse Events |
|---|---|---|---|---|---|---|---|
| Sarepta SRP-9001-101 [177] |
AAVrh74 | MHCK7 (SM and cardiac) |
coΔR4-R23/ΔCT | 2 × 1014 | 95.8% of normal | 5.5 points increase after 1 year | |
| Pfizer PF-06939926 |
AAV9 | Human muscle specific | - | 1 × 1014 3 × 1014 |
23.6% of normal 29.5% of normal |
2 points increase after 1 year | In 1 patient at 3 × 1014 vg/kg: complement activation, acute kidney failure, thrombocytopenia |
| Solid SGT-001 |
AAV9 | CK8 | ΔR2-R15/ΔR18-R22/ΔCT | 2 × 1014 2 doses |
Complement activation, acute kidney failure, thrombocytopenia (2 SAEs in 6th patient) |