Duchenne muscular dystrophy
Conditions
Interventions
investigational material(s)
Generic name etc : DS-5141b
INN of investigational material : -
Therapeutic category code : 49- Other agents affecting cellular function
Dosage and Administration for Inves
Sponsors
DAIICHISANKYO Co.,Ltd.
Orphan Disease Treatment Institute Co., Ltd.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1)Confirmation of out-of-frame deletion(s) that could be corrected by dystrophin gene exon 45 skipping. 2)Intact muscles of adequate quality for biopsy to allow evaluation of the efficacy of the study drug. 3)Boys aged from 5 years to <11 years. 4)Patients able to walk at least 325 meters in the 6-minutes walk test. 5)Glucocorticoid-naive patients, or patients who have used glucocorticoids for at least 6 months prior to enrollment in this study with no dose changes for at least 3 months prior to enrollment.
Exclusion criteria
Exclusion criteria: 1)A genetic mutation that can not be expected the expression of dystrophin protein by dystrophin gene exon 45 skipping. 2)A concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function. 3)Current or history of severe disorder. 4)Left ventricular ejection fraction (LEVF) 0.45 sec.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| safety efficacy pharmacokinetics Exploratory evaluation of the safety, tolerability, efficacy, and PK profile. Safety: Adverse events, Labolatory tests, etc. Efficacy: Dystrophin protein expression in muscle tissue PK profile: PK parameters (Cmax, AUC, Tmax, T1/2, etc.) | — |
Secondary
| Measure | Time frame |
|---|---|
| efficacy Efficacy: Production of exon 45-skipped dystrophin mRNA in muscle tissue | — |
Countries
Japan
Outcome results
None listed