Duchenne muscular dystrophy
Conditions
Brief summary
Number and proportion of participants with treatment-emergent adverse events (TEAEs), treatment emergent serious adverse events (TESAEs), TEAEs considered related to study drug, and TEAEs leading to discontinuation from study drug and discontinuation from the study., Change from Baseline in dystrophin protein levels in muscle tissue as determined by Western blot analysis at Week 25
Detailed description
Muscle Tissue Endpoints: Cohorts dosed Q4W or Q8W with a second biopsy at Week 25, Change from Baseline at Week 25: -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissue, Change from baseline up to Week 145: -blood CK levels, Cohorts dosed Q8W with a second biopsy at Week 49 Change from Baseline at Week 49 in: -dystrophin protein levels in muscle tissue -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissue, Change from Baseline up to Week 145 in: -blood CK level, Functional Endpoints: All cohorts, Change from Baseline up to Week 145 in: -PUL scale V 2.0 score -%pFVC -NSAA total score, TTR, 10MRW, and SV95C in ambulatory participants, Plasma Endpoints: All cohorts -Cmax -tmax -AUCtlast -AUC∞ -λZ -t½ -CL -Vz and Vss, if appropriate, Immunogenicity Endpoint: All cohorts -Incidence of ADAs
Interventions
DRUGThe placebo is commercially available 5% dextrose solution intended for IV
DRUGDYNE-251
Sponsors
Dyne Therapeutics Inc.
Eligibility
Sex/Gender
Male
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number and proportion of participants with treatment-emergent adverse events (TEAEs), treatment emergent serious adverse events (TESAEs), TEAEs considered related to study drug, and TEAEs leading to discontinuation from study drug and discontinuation from the study., Change from Baseline in dystrophin protein levels in muscle tissue as determined by Western blot analysis at Week 25 | — |
Secondary
| Measure | Time frame |
|---|---|
| Muscle Tissue Endpoints: Cohorts dosed Q4W or Q8W with a second biopsy at Week 25, Change from Baseline at Week 25: -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissue, Change from baseline up to Week 145: -blood CK levels, Cohorts dosed Q8W with a second biopsy at Week 49 Change from Baseline at Week 49 in: -dystrophin protein levels in muscle tissue -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissue, Change from Baseline up to Week 145 in: -blood CK level, Functional Endpoints: All cohorts, Change from Baseline up to Week 145 in: -PUL scale V 2.0 score -%pFVC -NSAA total score, TTR, 10MRW, and SV95C in ambulatory participants, Plasma Endpoints: All cohorts -Cmax -tmax -AUCtlast -AUC∞ -λZ -t½ -CL -Vz and Vss, if appropriate, Immunogenicity Endpoint: All cohorts -Incidence of ADAs | — |
Countries
Belgium, Ireland, Italy, Spain
Outcome results
None listed