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Also known as PRO044 SC
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 being developed for the treatment of Duchenne Muscular Dystrophy (DMD), a genetic neuromuscular disorder. It is an investigational small molecule that has been studied in a Phase 1/2 clinical trial in male patients aged 5 years and older. It is not approved for any indication.
PRO044 is being developed by BioMarin Pharmaceutical Inc., which trades on the Nasdaq under the ticker BMRN. BioMarin is the sponsor of the clinical program evaluating PRO044 in Duchenne Muscular Dystrophy.
PRO044 is in Phase 1 development. A Phase 1/2 study of PRO044 in Duchenne Muscular Dystrophy has been completed, and the program remains investigational. PRO044 has not been approved by the FDA or any other regulatory agency for any indication.
PRO044 has been evaluated in one clinical trial, NCT01037309, titled "Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)." The trial enrolled 18 male participants aged 5 years and older across Belgium, Italy, the Netherlands, and Sweden. It was a controlled study that has since been completed.
Yes, PRO044 SC is another name for PRO044, the investigational small molecule being developed by BioMarin Pharmaceutical Inc. for Duchenne Muscular Dystrophy. Both names refer to the same drug candidate studied in the Phase 1/2 trial NCT01037309.