A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
Completed · Phase 3
Conditions studied: Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
In brief
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.
Key facts
- Study ID
- NCT04917874
- Run by
- Krystal Biotech, Inc.
- People needed
- 47
- Starts
- 2021-05-25
- Expected to finish
- 2023-07-31
- Last updated by the study team
- 2024-04-09
Who can join
Age: 0 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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 be willing to use reliable birth control method throughout the treatment duration and for 3 months following the last treatment application
You may not qualify if…
- Diseases or conditions that could interfere with the assessment of safety of the study treatment and compliance of the participant with study visits/procedures, as determined by the Investigator
- Pregnant or nursing women
- Active infection in the area that will undergo administration, that the Investigator believes will negatively impact the IP application
- Known allergy to any of the constituents of the product
- Concurrent skin transplant or mesh skin grafting; if the participant is currently having transplantation or grafting surgeries they must wait until the transplant has begun to heal and the graft has absorbed prior to initiating B-VEC therapy
- Participation in an interventional gene therapy clinical trial within the past three (3) months (not including B-VEC administration)
Where it is running
- Mission Dermatology Center — Rancho Santa Margarita, California, United States
- Stanford University — Redwood City, California, United States
- Pediatric Skin Research — Coral Gables, Florida, United States
- Northwestern University — Chicago, Illinois, United States
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
- Ascension Seton- Dell's Children Medical Group — Austin, Texas, United States
Full record on ClinicalTrials.gov
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