Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04179409 | A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. | PHASE2 | COMPLETED | 3 | — | — | Feb 18, 2020 | Sep 1, 2023 | Oct 26, 2023 | 1 | United States |
This will be assessed by the comparison of quantification of protein in muscle biopsy tissue by western blot from baseline to 1 year post-initiation of treatment.
This will be measured by capturing and reviewing Adverse Events as defined by CTCAE v4.0.
| Arm | Type | Description |
|---|---|---|
| AMONDYS 45 | EXPERIMENTAL | This arm will involve the treatment of boys with DMD who have a duplication of exon 45, for which AMONDYS 45 will target skipping of this exon. |
| EXONDYS 51 | EXPERIMENTAL | This arm will involve the treatment of boys with DMD who have a duplication of exon 51, for which EXONDYS 51 will target skipping of this exon. |
| VYONDYS 53 | EXPERIMENTAL | This arm will involve the treatment of boys with DMD who have a duplication of exon 53, for which VYONDYS 53 will target skipping of this exon. |
| Name | Type | Description |
|---|---|---|
| Amondys 45 | DRUG | This drug is used to target skipping of exon 45 of the dystrophin gene. |
| Exondys 51 | DRUG | This drug is used to target skipping of exon 51 of the dystrophin gene. |
| Vyondys 53 | DRUG | This drug is used to target skipping of exon 53 of the dystrophin gene. |
Inclusion Criteria: * Is a male with DMD and has an out-of-frame duplication of either exon 45, 51, or 53, with a normal copy number of all other DMD exons. * Is above age 6 months of age. * Has sufficient muscle mass in a pair of bilateral muscles that will allow for pre- and post-treatment muscle...