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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00159250 | Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy | PHASE1 | COMPLETED | 7 | — | — | Oct 26, 2007 | Mar 31, 2009 | Dec 5, 2019 | 1 | United Kingdom |
Number of Subjects with Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs
Assessed by light microscopy and immunocytochemistry to detect the differences in inflammatory infiltrates between the AVI-4568 and placebo-treated EDB muscles
| Arm | Type | Description |
|---|---|---|
| Low dose | EXPERIMENTAL | Low dose of AVI-4658 |
| High dose | EXPERIMENTAL | High dose of AVI-4658 |
| Name | Type | Description |
|---|---|---|
| AVI-4658 (PMO) | DRUG | morpholino antisense oligonucleotide |
Inclusion Criteria: 1. Subject is male ≥ 10 years and ≤ 17 years of age at the time of study drug administration. 2. Subject has clinical diagnosis compatible with Duchenne's Muscular Dystrophy (DMD) and evidence of mutational and dystrophin defects from muscle biopsy consistent with DMD (out-of fr...