DEB - Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Conditions
Keywords
DEB, Krystal Biotech
Brief 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.
Interventions
BIOLOGICALOpen Label Topical Beremagene Geperpavec (B-VEC)
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
Sponsors
Krystal Biotech, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Months to No maximum
Healthy volunteers
No
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 be willing to use reliable birth control method throughout the treatment duration and for 3 months following the last treatment application
Exclusion criteria
* 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)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To record safety outcomes of participants while on B-VEC. | Up to 112 weeks | 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. |
Countries
United States
Outcome results
None listed