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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01037309 | Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | PHASE1 | COMPLETED | 18 | — | — | Dec 1, 2009 | Oct 1, 2013 | Oct 16, 2018 | 4 | Belgium, Italy +2 |
number of subjects with 1 or more treatment emergent adverse events following SC or IV PRO044
| Arm | Type | Description |
|---|---|---|
| PRO044, cohort 1 | EXPERIMENTAL | Subcutaneous injection of 0.5 mg/kg on day 1, 8, 15, 22 and 29. |
| PRO044, cohort 2 | EXPERIMENTAL | Subcutaneous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29. |
| PRO044, cohort 3 | EXPERIMENTAL | Subcutaneous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29. |
| PRO044, cohort 4 | EXPERIMENTAL | Subcutaneous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29. |
| PRO044, cohort 5 | EXPERIMENTAL | Subcutaneous injection of maximally 10 mg/kg on day 1, 8, 15, 22 and 29 |
| PRO044, cohort 6 | EXPERIMENTAL | Subcutaneous injection of maximally 12 mg/kg on day 1, 8, 15, 22 and 29 |
| PRO044, cohort 7 | EXPERIMENTAL | Intravenous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29 |
| PRO044, cohort 8 | EXPERIMENTAL | Intravenous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29 |
| PRO044, cohort 9 | EXPERIMENTAL | Intravenous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29 |
| Name | Type | Description |
|---|---|---|
| PRO044 SC | DRUG | Subcutaneous injection, once a week, for five weeks |
| PRO044 IV | DRUG | Intravenous injection, once a week, for five weeks |
Inclusion Criteria: 1. Boys aged between 5 and 16 years inclusive. 2. Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO044. 3. Life expectancy of at least 6 months. 4. No previous treatment with investigational medicinal treatment within 6 months prior to the s...