Back to resultsA Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
Primary Purpose
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Open Label Topical Beremagene Geperpavec (B-VEC)
Sponsored by
About this trial
This is an interventional treatment trial for Dystrophic Epidermolysis Bullosa focused on measuring DEB, Krystal Biotech
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: 6 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)
Sites / Locations
- Mission Dermatology Center
- Stanford University
- Pediatric Skin Research
- Northwestern University
- Cincinnati Children's Hospital Medical Center
- Ascension Seton- Dell's Children Medical Group
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
B-VEC
Arm Description
Open label B-VEC topical treatment of DEB wounds.
Outcomes
Primary Outcome Measures
To record safety outcomes of participants while on B-VEC.
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.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04917874
Brief Title
A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
Official Title
Open Label Treatment of Beremagene Geperpavec (B-VEC)
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
May 25, 2021 (Actual)
Primary Completion Date
July 31, 2023 (Actual)
Study Completion Date
July 31, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Krystal Biotech, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
Keywords
DEB, Krystal Biotech
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
47 (Actual)
8. Arms, Groups, and Interventions
Arm Title
B-VEC
Arm Type
Experimental
Arm Description
Open label B-VEC topical treatment of DEB wounds.
Intervention Type
Biological
Intervention Name(s)
Open Label Topical Beremagene Geperpavec (B-VEC)
Intervention Description
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
Primary Outcome Measure Information:
Title
To record safety outcomes of participants while on B-VEC.
Description
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.
Time Frame
Up to 112 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Months
Accepts Healthy Volunteers
No
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hubert Chen, MD
Organizational Affiliation
Senior Vice President of Clinical Development
Official's Role
Study Director
Facility Information:
Facility Name
Mission Dermatology Center
City
Rancho Santa Margarita
State/Province
California
ZIP/Postal Code
92688
Country
United States
Facility Name
Stanford University
City
Redwood City
State/Province
California
ZIP/Postal Code
94063
Country
United States
Facility Name
Pediatric Skin Research
City
Coral Gables
State/Province
Florida
ZIP/Postal Code
33146
Country
United States
Facility Name
Northwestern University
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Ascension Seton- Dell's Children Medical Group
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
12. IPD Sharing Statement
Learn more about this trial
A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
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