Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PRO044 · 1 trial · 1 indication
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...
PRO044 is an investigational small molecule being studied for the treatment of Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. It is being developed by BioMarin Pharmaceutical Inc. (BMRN) and has completed a Phase 1/2 clinical trial in patients with DMD.
PRO044 is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. The company is conducting clinical research on this investigational small molecule for Duchenne Muscular Dystrophy.
PRO044 is in Phase 1 of clinical development. A Phase 1/2 study of PRO044 in Duchenne Muscular Dystrophy has been completed. The drug is investigational and has not been approved by regulatory authorities, as it is still in the early stages of clinical testing.
PRO044 has one completed clinical trial, identified as NCT01037309, titled "Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)." This trial enrolled 18 male participants aged 5 years and older across Belgium, Italy, Netherlands, and Sweden. The study was controlled but not randomized or double-blinded.
No alternative names for PRO044 have been disclosed. The drug is identified solely by its development code PRO044 and is being investigated by BioMarin Pharmaceutical Inc. for Duchenne Muscular Dystrophy.