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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01396239 | Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients | PHASE2 | COMPLETED | 12 | — | — | Jul 1, 2011 | Jun 1, 2012 | Mar 30, 2020 | 1 | United States |
| NCT00844597 | Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients | PHASE1 | COMPLETED | 19 | — | — | Jan 1, 2009 | Dec 1, 2010 | Oct 6, 2015 | 2 | United Kingdom |
The primary efficacy endpoint will be based on the pre-treatment and post-treatment change in the number (%) of dystrophin positive fibers as measured in the muscle biopsy tissue on immunohistochemistry (IHC).
Number of subjects with 1 or more Treatment Emergent Adverse Event that are possibly related to the investigational drug
Number of Patients with Treatment Emergent Adverse Events
| Arm | Type | Description |
|---|---|---|
| AVI-4658 (Eteplirsen) | EXPERIMENTAL | 1. 50 mg/kg eteplirsen for 28 weeks 2. 30 mg/kg eteplirsen for 28 weeks |
| Placebo / Delayed Treatment | PLACEBO_COMPARATOR | 3a. Placebo 50 mg/kg phosphate buffered saline solution identical in appearance to eteplirsen for 24 weeks followed by 50 mg/kg of eteplirsen for 4 weeks. 3b. Placebo 30 mg/kg phosphate buffered saline solution identical in appearance to eteplirsen for 24 weeks followed by 30 mg/kg of eteplirsen for 4 weeks. |
| Cohort 1 - 0.5 mg/kg/wk | EXPERIMENTAL | Subjects in this group will receive a 0.5 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period |
| Cohort 2 - 1.0 mg/kg/wk | EXPERIMENTAL | Subjects in this group will receive a 1.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period |
| Cohort 3 - 2.0 mg/kg/wk | EXPERIMENTAL | Subjects in this group will receive a 2.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period |
| Cohort 4 - 4.0 mg/kg/wk | EXPERIMENTAL | Subjects in this group will receive a 4.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period |
| Cohort 5 - 10.0 mg/kg/wk | EXPERIMENTAL | Subjects in this group will receive a 10.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period |
| Cohort 6 - 20.0 mg/kg/wk | EXPERIMENTAL | Subjects in this group will receive a 20.0 mg/kg/wk dose of AVI-4658 over 12 weekly IV infusions in 50 mL of normal saline solution over a 60-minute period |
| Name | Type | Description |
|---|---|---|
| AVI-4658 (Eteplirsen) | DRUG | Treatment group 1 (n=4): 50.0 mg/kg eteplirsen once weekly x 24 weeks via a 60-minute i.v. infusion Treatment group 2 (n=4): 30.0 mg/kg eteplirsen once weekly x 24 weeks via a 60-minute i.v. infusion Treatment group 3 (n=4): matching placebo once weekly x 24 weeks via a 60-minute i.v. infusion; treatment group 3a will match two placebo subjects to 50.0 mg/kg eteplirsen; treatment group 3b will match two placebo subjects to 30.0 mg/kg eteplirsen |
| Placebo | OTHER | sterile, isotonic, clear, colorless phosphate buffered saline solution of eteplirsen at a concentration of 100 mg/mL in single-use vials containing a nominal volume of 1.0 mL without preservatives. |
| AVI-4658 for Injection | DRUG | AVI-4658 for Injection, is packaged as 100 mg/mL in phosphate buffered saline with 1 mL per vial. Study dosages will be infused over a 1 hour period with Normal saline in 6 dose cohorts. |
Major Inclusion and Exclusion Criteria: Inclusion Criteria: A subject must meet all of the following criteria to be eligible for this study. * Be a male with DMD and have an out-of-frame deletion(s) that may be corrected by skipping exon 51 \[e.g., deletions of exons 45-50, 47-50, 48-50, 49-50, 5...