Duchenne Muscular Dystrophy
Conditions
Brief summary
Safety 1. Incidence and severity of local (foot) and systemic adverse events (any grade) in DMD patients treated with intra- muscular injections of genetically corrected auto-MABS for one year from the injection., Efficacy 2.Presence of dystrophin (≥ to 1:10 dilution of a control muscle for WB; ≥ 10% of gene expression of skipped dystrophin at ddPCR; ≥ 10% of dystrophin positive fibers for IF respect to a control muscle) on muscle biopsy of the EDB muscle transplanted with genetically corrected auto-MABS, in at least two out of the three assays (Quantitative IF , ddPCR, and Western Blot) at two months after injection.
Interventions
DRUGDMD06-MAB
Sponsors
Ospedale San Raffaele S.r.l., University Of Manchester
Eligibility
Sex/Gender
Male
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety 1. Incidence and severity of local (foot) and systemic adverse events (any grade) in DMD patients treated with intra- muscular injections of genetically corrected auto-MABS for one year from the injection., Efficacy 2.Presence of dystrophin (≥ to 1:10 dilution of a control muscle for WB; ≥ 10% of gene expression of skipped dystrophin at ddPCR; ≥ 10% of dystrophin positive fibers for IF respect to a control muscle) on muscle biopsy of the EDB muscle transplanted with genetically corrected auto-MABS, in at least two out of the three assays (Quantitative IF , ddPCR, and Western Blot) at two months after injection. | — |
Countries
Italy
Outcome results
None listed