A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Summary
Protocol PTR-01-001 is a Phase 1/2 study of PTR-01. The study is divided into an up to 4-week Screening Period, a 10-week Treatment Period and an 8-week Follow-up Period. Cohorts 1, 2, 3 and 4 will consist of 2, 4, 3 and 3 patients respectively. Each cohort will consist of patients divided into two groups (Group 1 and Group 2) randomized in a 1:1 ratio. Patients in Group 1 will receive three doses of active drug followed by 3 doses of saline control. Patients in Group 2 will receive three doses of saline control followed by 3 doses of active drug. Cohort 1 patients randomized to Group 1 will receive 3 doses of active treatment (PTR-01) at a dose of 0.1 mg/kg followed by 3 doses of saline control for a total of 6 doses. Cohort 1 patients randomized to Group 2 will receive 3 doses of saline control followed by 3 doses of active treatment (PTR-01) at a dose of 0.1 mg/kg for a total of 6 doses.
Timeline
- Start
- 2019-01-09
- Primary completion
- 2020-11-30
- Completion
- 2020-11-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | PTR-01 | Protein / enzyme biologic | 0.1 mg/kg | Intravenous |
| Subject | PTR-01 | Protein / enzyme biologic | 0.3 mg/kg | Intravenous |
| Subject | PTR-01 | Protein / enzyme biologic | 1 mg/kg | Intravenous |
| Subject | PTR-01 | Protein / enzyme biologic | 3 mg/kg | Intravenous |