Clinical trial NCT04917874: A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
Official title: Open Label Treatment of Beremagene Geperpavec (B-VEC)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Dystrophic Epidermolysis Bullosa
DEB - Dystrophic Epidermolysis Bullosa
Recessive Dystrophic Epidermolysis Bullosa
Dominant Dystrophic Epidermolysis Bullosa
Keywords: DEB
Krystal Biotech
Lead sponsor: Krystal Biotech, Inc.
Sponsor class: INDUSTRY
First posted: 2021-06-08
Enrollment: 47
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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)

Primary outcomes:
measure: To record safety outcomes of participants while on B-VEC.
timeFrame: Up to 112 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Rancho Santa Margarita
country: United States
facility: Mission Dermatology Center
state: California
city: Redwood City
country: United States
facility: Stanford University
state: California
city: Coral Gables
country: United States
facility: Pediatric Skin Research
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Austin
country: United States
facility: Ascension Seton- Dell's Children Medical Group
state: Texas
Study officials:
affiliation: Senior Vice President of Clinical Development
name: David Chien, MD
role: STUDY_DIRECTOR
References: