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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04917874 |
Recruitment Status :
Recruiting
First Posted : June 8, 2021
Last Update Posted : June 9, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
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 : | 30 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, 2022 |
Estimated Study Completion Date : | December 31, 2022 |

- Biological: Open Label Topical Beremagene Geperpavec (B-VEC)
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
- Number of participants with adverse event (AE) or serious adverse events (SAE) [ Time Frame: Up to 78 weeks ]An Adverse Event (AE) is any untoward medical occurrence in a clinical investigation participant administered an investigational product and which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign, symptom, disease or exacerbation of a pre-existing condition temporally associated with the use of an investigational product.

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Ages Eligible for Study: | 6 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: 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)

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
Contact: Brittani Agostini | 4125865830 | bagostini@krystalbio.com |
United States, California | |
Mission Dermatology Center | Recruiting |
Rancho Santa Margarita, California, United States, 92688 | |
Contact: Guida Pinto 949-858-3376 ext 108 gpinto.missionderm@gmail.com | |
Principal Investigator: Shireen Guide, MD | |
Stanford University | Not yet recruiting |
Redwood City, California, United States, 94063 | |
Contact: Sinem Bagci 650-484-6878 isbagci@stanford.edu | |
Principal Investigator: Peter Marinkovich, MD | |
United States, Florida | |
Pediatric Skin Research | Not yet recruiting |
Coral Gables, Florida, United States, 33146 | |
Contact: Edilmary Urdaneta | |
Principal Investigator: Mercedes Gonzalez, MD | |
United States, Illinois | |
Northwestern University | Not yet recruiting |
Chicago, Illinois, United States, 60611 | |
Principal Investigator: Amy Paller, MD | |
United States, Ohio | |
Cincinnati Children's Hospital Medical Center | Not yet recruiting |
Cincinnati, Ohio, United States, 45229 | |
Principal Investigator: Anne Lucky, MD | |
United States, Texas | |
Ascension Seton- Dell's Children Medical Group | Not yet recruiting |
Austin, Texas, United States, 78723 | |
Principal Investigator: Moise Levy, MD |
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: | June 9, 2021 |
Last Verified: | June 2021 |
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 |