| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04917874 | A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa | PHASE3 | COMPLETED | 47 | — | — | May 25, 2021 | Jul 31, 2023 | Apr 9, 2024 | 6 | United States |
| NCT04491604 | Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB | PHASE3 | COMPLETED | 31 | — | — | Aug 17, 2020 | Jan 14, 2022 | Feb 17, 2023 | 3 | United States |
| NCT03536143 | A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients | PHASE1 | COMPLETED | 12 | — | — | May 6, 2018 | Nov 1, 2019 | Jan 31, 2023 | 1 | United States |
Record reportable Adverse (AE) and Serious Adverse Events (SAEs) during the continued use of B-VEC to participants who have participated in and completed Krystal Biotech's Phase 3 Protocol (A Phase 3 Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)), as an extension of use, upon study completion; as well as, for participants diagnosed with DEB who have not participated in the B-VEC Phase 3 trial.
The primary wound was defined as a responder wound that met either of the following conditions: * Complete wound healing on Week 22 and Week 24, or * Complete wound healing on Week 24 and Week 26. For subjects with missing primary wound healing data, a multiple imputation approach (10 repliates) was used. The total numbers of primary wounds with complete healing for B-VEC and Placebo presented below were the average of those from the multiple imputation replicates, and therefore, they would not be whole numbers (integers).
Safety assessments included evaluation of medical and medication history, physical / skin examination, vital signs, adverse events, and laboratory evaluations. Due to the 'split-person' intrasubject design, the safety assessments were reported at subject level, but not per intervention.
Safety assessments included evaluation of medical and medication history, physical / skin examination, vital signs, adverse events, and laboratory evaluations. Due to the 'split-person' intrasubject design, the safety assessments were reported at subject level, but not per intervention.
One wound is a responder if the reduction from baseline in wound surface is ≥90%.
Time to wound closure was defined as the time from the first treatment to Complete Wound Closure (≥90% reduction in wound surface area from baseline)
Duration of wound closure
| Arm | Type | Description |
|---|---|---|
| B-VEC | EXPERIMENTAL | Open label B-VEC topical treatment of DEB wounds. |
| Placebo | PLACEBO_COMPARATOR | Matching masked inactive topical gel |
| Topical beremagene geperpavec | EXPERIMENTAL | HSV1-COL7A1 vector (KB103) |
| Name | Type | Description |
|---|---|---|
| Open Label Topical Beremagene Geperpavec (B-VEC) | BIOLOGICAL | Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein |
| Topical Beremagene Geperpavec | BIOLOGICAL | Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein |
| Placebo | OTHER | Matching masked inactive topical gel |
| Placebo gel | BIOLOGICAL | Placebo gel |
Inclusion Criteria: * Willing and able to give consent/assent * Clinical diagnosis of epidermolysis bullosa * Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A1. * Age: 2 months of age and older at the time of informed consent/assent * Women of childbearing age must...