Trials / Completed
CompletedNCT04917874
A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
Open Label Treatment of Beremagene Geperpavec (B-VEC)
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 47 (actual)
- Sponsor
- Krystal Biotech, Inc. · Industry
- Sex
- All
- Age
- 2 Months
- Healthy volunteers
- Not accepted
Summary
This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study is for those who participated in Phase 3 study, as well as, new participants who were unable to participate in the Phase 3 study, who meet all enrollment criteria.
Conditions
- Dystrophic Epidermolysis Bullosa
- DEB - Dystrophic Epidermolysis Bullosa
- Recessive Dystrophic Epidermolysis Bullosa
- Dominant Dystrophic Epidermolysis Bullosa
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | Open Label Topical Beremagene Geperpavec (B-VEC) | Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein |
Timeline
- Start date
- 2021-05-25
- Primary completion
- 2023-07-31
- Completion
- 2023-07-31
- First posted
- 2021-06-08
- Last updated
- 2024-04-09
Locations
6 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04917874. Inclusion in this directory is not an endorsement.