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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05143190 | Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01) | PHASE2 | COMPLETED | 2 | — | — | Dec 2, 2021 | Aug 9, 2022 | Nov 21, 2022 | 2 | United States |
Change in a majority of target lesions of at least 2 levels using a 7-point (1-7) Global Impression of Change instrument (7 being the worst)
Safety and tolerability, as assessed by treatment-emergent adverse events
| Arm | Type | Description |
|---|---|---|
| PTR-01 | EXPERIMENTAL | All patients will receive a PTR-01 dose of 3.0 mg/kg once weekly every week for a total of 4 doses, followed by a dose of 3.0 mg/kg once monthly for a total of 5 doses. |
| Name | Type | Description |
|---|---|---|
| PTR-01 | DRUG | Intravenous recombinant collagen 7 |
Inclusion Criteria: 1. Willing to provide informed consent form, or if 12 to \<18 years of age, legal guardian has provided informed consent form and the minor has signed an assent form acknowledging that they understand and agree to study procedures. 2. Has satisfactorily completed participation i...