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- W4205748548 abstract "We designed an AAV vector (rAAVrh74) containing a codon-optimized human micro-dystrophin transgene with a muscle-specific promoter and a cardiac enhancer (MHCK7); we tested the safety and efficacy of this investigational gene transfer therapy (rAAVrh74.MHCK7 micro-dystrophin [SRP-9001]) in a three-part, multicenter, Phase 2 clinical trial (Study 102; NCT03769116). Part 1 is a 48-week, randomized, double-blind, placebo-controlled period. Part 2 is a 48-week period in which Part 1 placebo-treated patients receive SRP-9001 (blinded crossover). Part 3 is an open-label follow-up period (up to 212 weeks). Participants (ambulatory boys 4–7 years old, confirmed DMD mutation, stable steroid dosing) received SRP-9001 (n=20) or placebo (n=21). The intended dose was 2.0 × 1014 vg/kg (supercoiled qPCR, linear plasmid standard equivalent of 1.33 × 1014 vg/kg). Randomization was stratified by age (4–5 and 6–7 years). Primary endpoints were change in micro-dystrophin expression (western blot; baseline to Week 12) and change in North Star Ambulatory Assessment (NSAA; baseline to Week 48). Safety endpoints included serious adverse events (SAEs) and treatment-emergent AEs. In Part 1, the primary biological endpoint (change in micro-dystrophin expression) was met. At Week 48, NSAA change from baseline was not statistically different between groups. Pre-specified subgroup analysis of 4–5 year olds showed a statistically significant difference on NSAA change between SRP-9001 and placebo groups (+2.5, P=0.0172). Baseline NSAA score of the 4–5 year olds was balanced across arms but was significantly imbalanced in the 6–7 year olds. We observed no clinically relevant complement activation. Treatment-related AEs were transient and manageable. Four patients had treatment-related SAEs, which resolved. Additional Part 2 data will be presented. These data suggest that SRP-9001 has a biological effect that may be clinically relevant in people with DMD. Results reinforce a favorable benefit-risk profile." @default.
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- W4205748548 date "2021-10-01" @default.
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- W4205748548 title "CLINICAL TRIAL HIGHLIGHTS" @default.
- W4205748548 doi "https://doi.org/10.1016/j.nmd.2021.07.383" @default.
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