A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04917874 |
Recruitment Status :
Enrolling by invitation
First Posted : June 8, 2021
Last Update Posted : April 18, 2023
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Condition or disease | Intervention/treatment | Phase |
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Dystrophic Epidermolysis Bullosa DEB - Dystrophic Epidermolysis Bullosa Recessive Dystrophic Epidermolysis Bullosa Dominant Dystrophic Epidermolysis Bullosa | Biological: Open Label Topical Beremagene Geperpavec (B-VEC) | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 45 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Open Label Treatment of Beremagene Geperpavec (B-VEC) |
Actual Study Start Date : | May 25, 2021 |
Estimated Primary Completion Date : | December 31, 2023 |
Estimated Study Completion Date : | December 31, 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: B-VEC
Open label B-VEC topical treatment of DEB wounds.
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Biological: Open Label Topical Beremagene Geperpavec (B-VEC)
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein |
- To record safety outcomes of participants while on B-VEC. [ Time Frame: 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.

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Ages Eligible for Study: | 2 Months and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts 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)

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04917874
United States, California | |
Mission Dermatology Center | |
Rancho Santa Margarita, California, United States, 92688 | |
Stanford University | |
Redwood City, California, United States, 94063 | |
United States, Florida | |
Pediatric Skin Research | |
Coral Gables, Florida, United States, 33146 | |
United States, Illinois | |
Northwestern University | |
Chicago, Illinois, United States, 60611 | |
United States, Ohio | |
Cincinnati Children's Hospital Medical Center | |
Cincinnati, Ohio, United States, 45229 | |
United States, Texas | |
Ascension Seton- Dell's Children Medical Group | |
Austin, Texas, United States, 78723 |
Responsible Party: | Krystal Biotech, Inc. |
ClinicalTrials.gov Identifier: | NCT04917874 |
Other Study ID Numbers: |
B-VEC-EX-02 |
First Posted: | June 8, 2021 Key Record Dates |
Last Update Posted: | April 18, 2023 |
Last Verified: | April 2023 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
DEB Krystal Biotech |
Epidermolysis Bullosa Epidermolysis Bullosa Dystrophica Skin Abnormalities Congenital Abnormalities Skin Diseases, Genetic |
Genetic Diseases, Inborn Skin Diseases Skin Diseases, Vesiculobullous Collagen Diseases Connective Tissue Diseases |